Hemophilia A, Hemophilia A or B With Inhibitors, Hemophilia B
Conditions
Keywords
Hemophilia A, Factor VIII Inhibitor, Factor IX Inhibitor, Hemophilia B
Brief summary
The purpose of the study was to determine the efficacy, safety, and health-related quality of life benefits with FEIBA NF prophylactic treatment as compared with on-demand treatment.
Interventions
BIOLOGICALFactor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated)
85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Sponsors
Baxalta now part of Shire
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
4 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* Signed and dated informed consent form by the participant or the participant's legally authorized representative * The participant is ≥ 4 to ≤ 65 years of age * The participant has a Karnofsky performance score of ≥ 60 * Hemophilia A and B of any severity, with documented history of high-titer inhibitor (\> 5 Bethesda unit (BU)) for at least 12 months; or, if inhibitor titer is ≤ 5 BU, and the participant is refractory with increased dosing of either factor VIII (FVIII) or factor IX (FIX), as demonstrated from the participant's medical history * Currently being treated on an on-demand basis for treatment of bleeding episodes * Adequate venous access, with or without central venous device * ≥ 12 bleeding episodes requiring treatment with by-passing agents in the past 12 months, based on medical history * Competent in-home treatment and infusion therapy * Currently using bypassing agents (activated prothrombin complex concentrate (APCC) or recombinant activated factor VII (rFVIIa)) for treatment of bleeding episodes * HCV-, either by antibody testing or polymerase chain reaction (PCR); or HCV+ with stable hepatic disease * HIV-, or HIV+ with stable disease and CD4 count \> 200 cells/mm3 at screening * Female participant of childbearing potential, presents with a negative serum pregnancy test, and agrees to employ adequate birth control measures for the duration of the study
Exclusion criteria
* Currently receiving immune tolerance induction (ITI) * Currently on regular prophylactic therapy to prevent bleeding episodes * Clinically symptomatic liver disease (e.g. diagnosis of cirrhosis \[confirmed by liver biopsy\], portal vein hypertension, ascites, prothrombin time (PT) 5 seconds above upper limit of normal) * Platelet count \< 100,000/ml * Planned elective surgery during participation in this study * Participant is currently participating in another clinical study and has received an investigational product or device within 30 days prior to study entry * Planned use of pegylated or non-pegylated alpha-interferon with or without ribavirin for HCV infected participants or planned use of a protease inhibitor for HIV infected participants. Participants currently taking any of these medications for a 30-day course are eligible. * Clinically significant increase in D-dimer levels from historical baseline and/or associated with chronic liver disease or clinically evident thromboembolic event * Known hypersensitivity to anti-inhibitor coagulant complexes (AICCs) * Currently treated with a systemic immunomodulating drug * Prior history of thromboembolic event: acute myocardial infarction, deep vein thrombosis, or pulmonary embolism * Diagnosis of advanced atherosclerosis, malignancy and/or other diseases that may increase the participant's risk of thromboembolic complications * Clinically significant medical, psychiatric, or cognitive illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant safety or compliance
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Reduction in Annualized Bleeding Episode Rate (ABR) Among Participants Receiving Prophylactic Treatment as Compared to Those Treated On-demand | 12 months ± 14 days | Participants were Randomized to Receive 1 of the 2 Following Treatment Regimens: 1.On-Demand: FEIBA NF dose & dosing interval as prescribed by treating physician 2.Prophylaxis: 85 ± 15 U/kg of FEIBA NF every other day during 12-month prophylactic period Annualized rate of bleeding episodes was calculated as: (Number of bleeding episodes/observed treatment period in days) \* 365.25 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Rate of Related Adverse Events (AEs) Per Year | 12 months ± 14 days | — |
| Rate of Related Adverse Events (AEs) During or Within 1 Hour of Infusion Per Year | 12 months ± 14 days | — |
| Number of Related Thromboembolic Adverse Events (AEs) | 12 months ± 14 days | — |
| Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type | 12 months ± 14 days | Spontaneous includes unknown/undermined etiology |
| Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens by Bleeding Etiology, and Bleeding Type | 12 months ± 14 days | Annualized bleed rates were transformed using the square root of the number of bleeding episodes observed (X bleeds/year), X' = √(X + 0.5). This transformation was performed to stabilize the variance and align the sample distribution with the assumption of normality inherent in using the t-test. The difference in mean transformed ABRs was used to perform statistical tests and generate p-values at a significance level of 5% Participants were Randomized to Receive 1 of the 2 Following Treatment Regimens: 1.On-Demand: FEIBA NF dose & dosing interval as prescribed by treating physician 2.Prophylaxis: 85 ± 15 U/kg of FEIBA NF every other day during 12-month prophylactic period |
| Annualized Bleeding Rate for New Target Joints | 12 months ± 14 days | Target joints are ≥4 bleeds/6 months in any one of the following joints: ankles, knees, elbows, and hips; a target joint bleeding episode refers to an individual anatomical location. |
| Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens: New Target Joints | 12 months ± 14 days | Annualized bleed rates (ABRs) were transformed using the square root of the number of bleeding episodes observed (X bleeds/year), X' = √(X + 0.5). This transformation was performed to stabilize the variance and align the sample distribution with the assumption of normality inherent in using a two-sample, two-sided t-test. The difference in mean transformed ABRs was used to perform statistical tests and generate p-values at a significance level of 5% |
| Number of New Target Joints | 12 months ± 14 days | Target Joints are defined as ≥4 bleeds/6 months in any one of the following joints: ankles, knees, elbows and hips |
| Assessment of Objective Clinical Symptoms- Visual Analog Scale (VAS): Pain in Adolescents and Adults (≥12 Years Old) | Throughout the study period, 12 months ± 14 days | Pain caused by a bleeding episode in adolescents and adults (≥12 years old) was measured at pre-infusion (pre-inf) and at 6 ± 0.5 hours (h) and 24 ± 1 h post-infusion (post-inf) (after the last infusion given to treat a bleeding episode) on the VAS pain scale in millimeters from 0 (no pain) to 100 (worst possible pain). For analysis purposes, if short acting analgesics (duration of activity approximately 6 ± 0.5 h) were used, pain was assigned the highest possible score (100). Pain assessment occurred after each infusion related to single bleeding episodes. In case participants required an additional infusion within 24h, pain was assessed 6 ± 0.5 h and 24 ±1 h following the subsequent infusion. Change in VAS scores at 6 ± 0.5 h and 24 ±1 h post-infusion were also compared relative to pre-infusion VAS scores (ie, (pre-infusion VAS score) - (post-infusion VAS score)). |
| Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | 12 months ± 14 days | Pain caused by a bleeding episode (BE) in pediatric participants (\<12 years old) was measured at pre-infusion (pre-inf) and at 6 ± 0.5 h and 24 ± 1 h post-infusion (post-inf) (after the last infusion given to treat a bleeding episode) using the children's VAS pain scale (a facial expression scale with one end marked as no pain and the opposite end marked as the worst possible pain). For analysis purposes, if short acting analgesics (duration of activity approximately 6 ± 0.5 h) were used, pain was assigned the highest possible score (worst possible pain). Scores on the children's VAS scale are presented as: -No Pain -Mild Pain -Moderate pain -Severe pain -Very severe pain |
| Assessment of Clinical Symptoms - Range of Motion (ROM) | 12 months ± 14 days | ROM was measured using a goniometer for 3 key joints (ie, ankles, knees, and elbows) at screening, month 6, and termination (end of study visit) |
| Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 6 Hours | 6 h ± 30 min post-infusion | Number of rAHF-PFM-treated bleeding episodes with an assessment of hemostasis (4-point ordinal scale): Excellent: Full pain relief & bleeding cessation within \ 6 hours of 1 infusion. Additional infusions may have been given to maintain hemostasis; Good: Definite pain relief and/or improvement in bleeding within \ 6 hours after infusion. Possibly requires \>1 infusion for complete resolution; Fair: Probable or slight relief of pain & slight improvement in bleeding within \ 6 hours after infusion. Requires \>1 infusion for complete resolution; None: No improvement or condition worsens |
| Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 24 Hours | 24 ± 1 h post-infusion | Number of rAHF-PFM-treated bleeding episodes with an assessment of hemostasis (4-point ordinal scale): Excellent: Full pain relief & bleeding cessation within \ 24 hours of 1 infusion. Additional infusions may have been given to maintain hemostasis; Good: Definite pain relief and/or improvement in bleeding within \ 24 hours after infusion. Possibly requires \>1 infusion for complete resolution; Fair: Probable or slight relief of pain & slight improvement in bleeding within \ 24 hours after infusion. Requires \>1 infusion for complete resolution; None: No improvement or condition worsens |
| The Number of Bleeding Episode (BE) Which Required 1, 2, 3, or ≥4 Infusions to Control Bleeding | 12 months ± 14 days | — |
| Abnormal Activated Partial Thromboplastin Time (aPTT) Assay Results | Screening visit, Month 3, Month 6, Month 9, and Termination visit | The normal reference range of values for aPTT is 22.8 - 31 seconds. |
| Abnormal D-Dimer Assay Results | Screening visit, Month 3, Month 6, Month 9, and Termination visit | The normal reference range of values for D-dimers is \<500 ng/mL. |
| Abnormal Fibrinogen Assay Results | Screening visit, Month 3, Month 6, Month 9, and Termination visit | The normal reference range of values for fibrinogen is 200-400 mg/dL. |
| Abnormal Fibrin Degradation Products (FDP) Assay Results | Screening visit, Month 3, Month 6, Month 9, and Termination visit | The normal reference range of values for FDP is 0-5 ug/mL. |
| Abnormal Prothrombin Fragment F 1.2 Assay Results | Screening visit, Month 3, Month 6, Month 9, and Termination visit | The normal reference range of values for prothrombin fragment F 1.2 is 69-229 pmol/L. |
| Abnormal Prothrombin Time Assay Results | Screening visit, Month 3, Month 6, Month 9, and Termination visit | The normal reference range of values for PT is 9.7-12.3 sec. |
| Abnormal Thrombin-Antithrombin III (TAT) Assay Results | Screening visit, Month 3, Month 6, Month 9, and Termination visit | The normal reference range of values for TAT is 1-4.1 ug/L. |
| Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | 12 months ± 14 days | -Hepatitis A Virus Antibody (HAV Ab) -Hepatitis B Virus Core Antibody (HBcAb) -Hepatitis B Virus Surface Antibody (HBsAb) -Hepatitis B Virus Surface Antigen (HBsAg) -Hepatitis C Virus (HCV) |
| Viral Serology From Screening Visit and Study Termination Visit: HIV-1/2 Antibody (Ab) | 12 months ± 14 days | — |
| Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgG Antibody [IV] | 12 months ± 14 days | Normal range (0 - 0.89 IV); High (\> 0.89 IV) - Parvovirus B19 IgG Antibody \[IV\] (Parvo IgG Ab) |
| Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgM Antibody [IV] | 12 months ± 14 days | Normal range (0 - 0.89 IV); High (\> 0.89 IV) - Parvovirus B19 IgM Antibody \[IV\] (Parvo IgM Ab) |
| Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels | 12 months ± 14 days | Absolute Changes in Inhibitor Titer (or no change in low or high titer status): -Inhibitor Titer went from Low (≤5 BU) to Low (≤5 BU) -Inhibitor Titer went from Low (≤5 BU) to High (\>5 BU) -Inhibitor Titer went from High (\>5 BU) to Low (≤5 BU) -Inhibitor Titer went from High (\>5 BU) to High (\>5 BU) |
| Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | 12 months ± 14 days | Absolute Changes in Inhibitor Titer (or no change in low or high titer status): -Inhibitor Titer went from Low (≤5 BU) to Low (≤5 BU) -Inhibitor Titer went from Low (≤5 BU) to High (\>5 BU) -Inhibitor Titer went from High (\>5 BU) to Low (≤5 BU) -Inhibitor Titer went from High (\>5 BU) to High (\>5 BU) |
| Pharmacoeconomics: Annual Days Lost Due to Bleeding (Work or School) | 12 months ± 14 days | — |
| Pharmacoeconomics: Annual Number of Hospitalizations for Bleeding | 12 months ± 14 days | — |
| Pharmacoeconomics: Annual Number of Hospitalizations for Indwelling Line | 12 months ± 14 days | — |
| Pharmacoeconomics: Annual Number of Emergency Room Visits | 12 months ± 14 days | — |
| Pharmacoeconomics: Annual Number of Physician's Office Visits | 12 months ± 14 days | — |
| Pharmacoeconomics: Annual Total Length of Hospitalization for Bleeding | 12 months ± 14 days | — |
| Pharmacoeconomics: Annual Total Length of Hospitalization for Indwelling Line | 12 months ± 14 days | — |
| Pharmacoeconomics: Annual Total Number of Days Lost (Work or School) | 12 months ± 14 days | — |
| Health-Related Quality of Life (HRQoL): EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Index Scores | 12 months ± 14 days | EQ-5D is a participant answered questionnaire scoring 5 dimensions - mobility, self-care, usual activities, pain/discomfort and anxiety/depression. The EQ-5D total score ranges from 0 (worst health state) to 1 (perfect health state) and 1 reflects the best outcome. EQ-5D Index scores based on EQ-5D questionnaire were calculated for participants ≥14 years of age, at screening, 6 months, and at termination visit. Changes in scores at 6 months and termination were also calculated. A relatively higher score represents better quality of life. |
| Total Weight Adjusted Dose to Control a Bleeding Episode | 12 months ± 14 days | — |
| Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | 12 months ± 14 days | The Haemo-QoL is a quality of life (QoL) assessment instrument for children and adolescents with haemophilia. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. The areas covered by this instrument are: Physical Health (PH), Sports & School (S&S), Dealing with Hemophilia (Dealing), Family, Feeling, Relationships (R'ships), Treatment, View, Outlook for the Future (Future), Friends, Others, and Support. A Haemo-QoL Total Score (Total) was also calculated. For the Haemo-QoL, higher scores indicate a worse quality of life. Scores on a scale range between 0 and 100. Haemo-QoL scores at screening, 6 months, and at termination visit were collected. Changes in scores at 6 months and termination were also calculated. |
| Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | 12 months ± 14 days | The Haemo-QoL is a quality of life (QoL) assessment instrument for children and adolescents with haemophilia. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. The areas covered by this instrument are: Physical Health (PH), Sports & School (S&S), Dealing with Hemophilia (Dealing), Family, Feeling, Relationships (R'ships), Treatment, View, Outlook for the Future (Future), Friends, Others, and Support. A Haemo-QoL Total Score (Total) was also calculated. For the Haemo-QoL, higher scores indicate a worse quality of life. Scores on a scale range between 0 and 100. Haemo-QoL scores at screening, 6 months, and at termination visit were collected. Changes in scores at 6 months and termination were also calculated. |
| Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Adults and Adolescents ≥12 Years Old | Baseline, 6 months and 12 months ± 14 days | General pain was assessed using a VAS pain scale at screening, 6 months, and at termination. Unlike the VAS pain assessment for pain of bleeding episodes (Outcome above), this general pain assessment did not take use of analgesics into account. For the pain scale, a higher number indicates worse pain. The visual analog scale ranges from 0 to 100 where the endpoints are labeled 'Worst imaginable health state' (=0) and 'Best imaginable health state' (=100). A positive change from baseline indicates improvement. Change in VAS scores at 6 months and study termination were also compared relative to Baseline/Screening scores (ie, (Baseline/Screening VAS score) - (VAS score at 6 months and study termination). |
| Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Baseline, 6 months and 12 months ± 14 days | General pain was assessed using the children's VAS pain scale (a facial expression scale with one end marked as no pain and the opposite end marked as the worst possible pain). Assessments were done at the screening, 6 months, and termination visits. Scores on the children's VAS scale are presented as: -No Pain -Mild Pain -Moderate pain -Severe pain -Very severe pain Unlike the VAS pain assessment for pain of bleeding episodes (Outcome above), this general pain assessment did not take use of analgesics into account. Change in VAS scores at 6 months and study termination were also compared relative to Baseline/Screening scores (ie, (Baseline/Screening VAS score) - (VAS score at 6 months and study termination). |
| Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | 12 months ± 14 days | The Haem-A-QoL instrument has been developed and used in Hemophilia A patients. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. The areas covered by this instrument are: Physical Health (PH), Sports & Leisure (S&L), School & Work (W&S), Dealing with Hemophilia (Dealing), Family Planning (FP), Feeling, Relationships (R'ships), Treatment, View, and Outlook for the Future (Future). A Haem-A-QoL Total Score (Total) was also calculated. For the Haem-A-QoL, higher scores indicate a worse quality of life. Scores on a scale range between 0 and 100. Haem-A-QoL scores at screening, 6 months, and at termination visit were collected. Changes in scores at 6 months and termination were also calculated. |
Countries
Brazil, Bulgaria, Croatia, Japan, New Zealand, Poland, Romania, Russia, Ukraine, United States
Participant flow
Recruitment details
Enrollment was conducted in Europe, North America, Asia-Pacific, and South America at 17 clinical sites beginning in March 2009.
Pre-assignment details
52 participants were enrolled. Sixteen participants discontinued, (ten were screen failures and six were withdrawn before randomization (2 sponsor's decision- inhibitors, 3 withdrew consent, and 1 due to investigator decision to have participant on prophylaxis). Therefore 36 participants were randomized.
Participants by arm
| Arm | Count |
|---|---|
| On-demand Arm Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : Standard FEIBA NF dose and dosing interval as prescribed by the treating physician | 19 |
| Prophylaxis Arm Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period | 17 |
| Total | 36 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 0 | 1 |
| Overall Study | Death | 1 | 0 |
| Overall Study | planned surgery | 1 | 0 |
Baseline characteristics
| Characteristic | On-demand Arm | Prophylaxis Arm | Total |
|---|---|---|---|
| Age, Continuous | 29.1 years STANDARD_DEVIATION 15.2 | 25.6 years STANDARD_DEVIATION 15.4 | 27.4 years STANDARD_DEVIATION 15.2 |
| Region of Enrollment Brazil | 3 Participants | 2 Participants | 5 Participants |
| Region of Enrollment Bulgaria | 0 Participants | 2 Participants | 2 Participants |
| Region of Enrollment Croatia | 0 Participants | 1 Participants | 1 Participants |
| Region of Enrollment Japan | 1 Participants | 1 Participants | 2 Participants |
| Region of Enrollment New Zealand | 1 Participants | 1 Participants | 2 Participants |
| Region of Enrollment Poland | 1 Participants | 1 Participants | 2 Participants |
| Region of Enrollment Romania | 0 Participants | 1 Participants | 1 Participants |
| Region of Enrollment Russian Federation | 8 Participants | 3 Participants | 11 Participants |
| Region of Enrollment Ukraine | 4 Participants | 4 Participants | 8 Participants |
| Region of Enrollment United States | 1 Participants | 1 Participants | 2 Participants |
| Sex: Female, Male Female | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Male | 19 Participants | 17 Participants | 36 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 4 / 19 | 3 / 17 |
| serious Total, serious adverse events | 7 / 19 | 6 / 17 |
Outcome results
Primary
Reduction in Annualized Bleeding Episode Rate (ABR) Among Participants Receiving Prophylactic Treatment as Compared to Those Treated On-demand
Participants were Randomized to Receive 1 of the 2 Following Treatment Regimens: 1.On-Demand: FEIBA NF dose & dosing interval as prescribed by treating physician 2.Prophylaxis: 85 ± 15 U/kg of FEIBA NF every other day during 12-month prophylactic period Annualized rate of bleeding episodes was calculated as: (Number of bleeding episodes/observed treatment period in days) \* 365.25
Time frame: 12 months ± 14 days
Population: Efficacy Intent to Treat Analysis Dataset: Dataset consists of data from all randomized participants. Dataset includes those who discontinued FEIBA NF but did not withdraw informed consent and were willing to continue providing data. For these participants, only the data collected before FEIBA NF discontinuation is included.
| Arm | Measure | Value (MEDIAN) | Dispersion |
|---|---|---|---|
| On-demand Arm | Reduction in Annualized Bleeding Episode Rate (ABR) Among Participants Receiving Prophylactic Treatment as Compared to Those Treated On-demand | 28.7 bleeds/year | Inter-Quartile Range 1.8 |
| Prophylaxis Arm | Reduction in Annualized Bleeding Episode Rate (ABR) Among Participants Receiving Prophylactic Treatment as Compared to Those Treated On-demand | 7.9 bleeds/year | Inter-Quartile Range 1.8 |
Comparison: H0: μ(on-demand) = μ(prophylaxis) Versus H1: μ(on-demand) ≠ μ(prophylaxis) (Where H0 implies no difference in mean bleeding episode rate between prophylaxis and on-demand treatment arms and H1 implies otherwise. This test was performed at a significance level of 5%, two-sided, two sample)p-value: 0.0003Two-sample, two-sided t-test
Secondary
Abnormal Activated Partial Thromboplastin Time (aPTT) Assay Results
The normal reference range of values for aPTT is 22.8 - 31 seconds.
Time frame: Screening visit, Month 3, Month 6, Month 9, and Termination visit
Population: Safety Analysis Set Participants with Clinically Significant Laboratory Results
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| On-demand Arm | Abnormal Activated Partial Thromboplastin Time (aPTT) Assay Results | Month 3 (n= 14, 15) | 68.0 seconds |
| On-demand Arm | Abnormal Activated Partial Thromboplastin Time (aPTT) Assay Results | Month 9 (n= 17, 15) | 70.0 seconds |
| On-demand Arm | Abnormal Activated Partial Thromboplastin Time (aPTT) Assay Results | Month 6 (n= 17, 15) | 68.2 seconds |
| On-demand Arm | Abnormal Activated Partial Thromboplastin Time (aPTT) Assay Results | Termination visit (n= 18, 16) | 69.8 seconds |
| On-demand Arm | Abnormal Activated Partial Thromboplastin Time (aPTT) Assay Results | Screening visit (n= 19, 16) | 67.3 seconds |
| Prophylaxis Arm | Abnormal Activated Partial Thromboplastin Time (aPTT) Assay Results | Termination visit (n= 18, 16) | 69.4 seconds |
| Prophylaxis Arm | Abnormal Activated Partial Thromboplastin Time (aPTT) Assay Results | Screening visit (n= 19, 16) | 68.5 seconds |
| Prophylaxis Arm | Abnormal Activated Partial Thromboplastin Time (aPTT) Assay Results | Month 3 (n= 14, 15) | 62.7 seconds |
| Prophylaxis Arm | Abnormal Activated Partial Thromboplastin Time (aPTT) Assay Results | Month 6 (n= 17, 15) | 65.6 seconds |
| Prophylaxis Arm | Abnormal Activated Partial Thromboplastin Time (aPTT) Assay Results | Month 9 (n= 17, 15) | 68.1 seconds |
Secondary
Abnormal D-Dimer Assay Results
The normal reference range of values for D-dimers is \<500 ng/mL.
Time frame: Screening visit, Month 3, Month 6, Month 9, and Termination visit
Population: Safety Analysis Set Participants with Clinically Significant Laboratory Results
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| On-demand Arm | Abnormal D-Dimer Assay Results | Month 3 (n= 7, 8) | 690.0 ng/mL |
| On-demand Arm | Abnormal D-Dimer Assay Results | Month 9 (n= 6, 8) | 731.5 ng/mL |
| On-demand Arm | Abnormal D-Dimer Assay Results | Month 6 (n= 9, 8) | 969.0 ng/mL |
| On-demand Arm | Abnormal D-Dimer Assay Results | Termination visit (n= 5, 9) | 813.0 ng/mL |
| On-demand Arm | Abnormal D-Dimer Assay Results | Screening visit (n= 7, 6) | 862.0 ng/mL |
| Prophylaxis Arm | Abnormal D-Dimer Assay Results | Termination visit (n= 5, 9) | 1031.0 ng/mL |
| Prophylaxis Arm | Abnormal D-Dimer Assay Results | Screening visit (n= 7, 6) | 833.5 ng/mL |
| Prophylaxis Arm | Abnormal D-Dimer Assay Results | Month 3 (n= 7, 8) | 1021.5 ng/mL |
| Prophylaxis Arm | Abnormal D-Dimer Assay Results | Month 6 (n= 9, 8) | 1032.0 ng/mL |
| Prophylaxis Arm | Abnormal D-Dimer Assay Results | Month 9 (n= 6, 8) | 1121.0 ng/mL |
Secondary
Abnormal Fibrin Degradation Products (FDP) Assay Results
The normal reference range of values for FDP is 0-5 ug/mL.
Time frame: Screening visit, Month 3, Month 6, Month 9, and Termination visit
Population: Safety Analysis Set Participants with Clinically Significant Laboratory Results
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| On-demand Arm | Abnormal Fibrin Degradation Products (FDP) Assay Results | Month 3 (n= 0, 2) | NA ug/mL |
| On-demand Arm | Abnormal Fibrin Degradation Products (FDP) Assay Results | Month 9 (n= 1, 2) | 8.0 ug/mL |
| On-demand Arm | Abnormal Fibrin Degradation Products (FDP) Assay Results | Month 6 (n= 1, 1) | 8.0 ug/mL |
| On-demand Arm | Abnormal Fibrin Degradation Products (FDP) Assay Results | Termination visit (n= 3, 1) | 8.0 ug/mL |
| On-demand Arm | Abnormal Fibrin Degradation Products (FDP) Assay Results | Screening visit (n= 1, 2) | 16.0 ug/mL |
| Prophylaxis Arm | Abnormal Fibrin Degradation Products (FDP) Assay Results | Termination visit (n= 3, 1) | 8.0 ug/mL |
| Prophylaxis Arm | Abnormal Fibrin Degradation Products (FDP) Assay Results | Screening visit (n= 1, 2) | 12.0 ug/mL |
| Prophylaxis Arm | Abnormal Fibrin Degradation Products (FDP) Assay Results | Month 3 (n= 0, 2) | 8.0 ug/mL |
| Prophylaxis Arm | Abnormal Fibrin Degradation Products (FDP) Assay Results | Month 6 (n= 1, 1) | 8.0 ug/mL |
| Prophylaxis Arm | Abnormal Fibrin Degradation Products (FDP) Assay Results | Month 9 (n= 1, 2) | 8.0 ug/mL |
Secondary
Abnormal Fibrinogen Assay Results
The normal reference range of values for fibrinogen is 200-400 mg/dL.
Time frame: Screening visit, Month 3, Month 6, Month 9, and Termination visit
Population: Safety Analysis Set Participants with Clinically Significant Laboratory Results
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| On-demand Arm | Abnormal Fibrinogen Assay Results | Month 3 (n= 0, 2) | NA mg/dL |
| On-demand Arm | Abnormal Fibrinogen Assay Results | Month 9 (n= 1, 4) | 508.0 mg/dL |
| On-demand Arm | Abnormal Fibrinogen Assay Results | Month 6 (n= 3, 1) | 415.0 mg/dL |
| On-demand Arm | Abnormal Fibrinogen Assay Results | Termination visit (n= 2, 2) | 456.5 mg/dL |
| On-demand Arm | Abnormal Fibrinogen Assay Results | Screening visit (n= 4, 6) | 428.0 mg/dL |
| Prophylaxis Arm | Abnormal Fibrinogen Assay Results | Termination visit (n= 2, 2) | 522.5 mg/dL |
| Prophylaxis Arm | Abnormal Fibrinogen Assay Results | Screening visit (n= 4, 6) | 422.0 mg/dL |
| Prophylaxis Arm | Abnormal Fibrinogen Assay Results | Month 3 (n= 0, 2) | 402.0 mg/dL |
| Prophylaxis Arm | Abnormal Fibrinogen Assay Results | Month 6 (n= 3, 1) | 759.0 mg/dL |
| Prophylaxis Arm | Abnormal Fibrinogen Assay Results | Month 9 (n= 1, 4) | 326.5 mg/dL |
Secondary
Abnormal Prothrombin Fragment F 1.2 Assay Results
The normal reference range of values for prothrombin fragment F 1.2 is 69-229 pmol/L.
Time frame: Screening visit, Month 3, Month 6, Month 9, and Termination visit
Population: Safety Analysis Set Participants with Clinically Significant Laboratory Results
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| On-demand Arm | Abnormal Prothrombin Fragment F 1.2 Assay Results | Month 3 (n= 6, 7) | 336.5 pmol/L |
| On-demand Arm | Abnormal Prothrombin Fragment F 1.2 Assay Results | Month 9 (n= 4, 9) | 382.0 pmol/L |
| On-demand Arm | Abnormal Prothrombin Fragment F 1.2 Assay Results | Month 6 (n= 3, 9) | 448.0 pmol/L |
| On-demand Arm | Abnormal Prothrombin Fragment F 1.2 Assay Results | Termination visit (n= 4, 7) | 252.5 pmol/L |
| On-demand Arm | Abnormal Prothrombin Fragment F 1.2 Assay Results | Screening visit (n= 2, 1) | 284.0 pmol/L |
| Prophylaxis Arm | Abnormal Prothrombin Fragment F 1.2 Assay Results | Termination visit (n= 4, 7) | 643.0 pmol/L |
| Prophylaxis Arm | Abnormal Prothrombin Fragment F 1.2 Assay Results | Screening visit (n= 2, 1) | 308.0 pmol/L |
| Prophylaxis Arm | Abnormal Prothrombin Fragment F 1.2 Assay Results | Month 3 (n= 6, 7) | 579.0 pmol/L |
| Prophylaxis Arm | Abnormal Prothrombin Fragment F 1.2 Assay Results | Month 6 (n= 3, 9) | 430.0 pmol/L |
| Prophylaxis Arm | Abnormal Prothrombin Fragment F 1.2 Assay Results | Month 9 (n= 4, 9) | 415.0 pmol/L |
Secondary
Abnormal Prothrombin Time Assay Results
The normal reference range of values for PT is 9.7-12.3 sec.
Time frame: Screening visit, Month 3, Month 6, Month 9, and Termination visit
Population: Safety Analysis Set Participants with Clinically Significant Laboratory Results
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| On-demand Arm | Abnormal Prothrombin Time Assay Results | Month 3 (n= 1, 0) | 9.5 seconds |
| On-demand Arm | Abnormal Prothrombin Time Assay Results | Month 9 (n= 1, 1) | 12.7 seconds |
| On-demand Arm | Abnormal Prothrombin Time Assay Results | Month 6 (n= 0, 1) | NA seconds |
| On-demand Arm | Abnormal Prothrombin Time Assay Results | Termination visit (n= 0, 1) | NA seconds |
| On-demand Arm | Abnormal Prothrombin Time Assay Results | Screening visit (n= 1, 1) | 9.4 seconds |
| Prophylaxis Arm | Abnormal Prothrombin Time Assay Results | Termination visit (n= 0, 1) | 13.4 seconds |
| Prophylaxis Arm | Abnormal Prothrombin Time Assay Results | Screening visit (n= 1, 1) | 9.6 seconds |
| Prophylaxis Arm | Abnormal Prothrombin Time Assay Results | Month 3 (n= 1, 0) | NA seconds |
| Prophylaxis Arm | Abnormal Prothrombin Time Assay Results | Month 6 (n= 0, 1) | 12.5 seconds |
| Prophylaxis Arm | Abnormal Prothrombin Time Assay Results | Month 9 (n= 1, 1) | 13.6 seconds |
Secondary
Abnormal Thrombin-Antithrombin III (TAT) Assay Results
The normal reference range of values for TAT is 1-4.1 ug/L.
Time frame: Screening visit, Month 3, Month 6, Month 9, and Termination visit
Population: Safety Analysis Set Participants with Clinically Significant Laboratory Results
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| On-demand Arm | Abnormal Thrombin-Antithrombin III (TAT) Assay Results | Termination visit (n= 2, 5) | 10.3 ug/L |
| On-demand Arm | Abnormal Thrombin-Antithrombin III (TAT) Assay Results | Screening visit (n= 4, 1) | 5.1 ug/L |
| On-demand Arm | Abnormal Thrombin-Antithrombin III (TAT) Assay Results | Month 3 (n= 3, 7) | 4.7 ug/L |
| On-demand Arm | Abnormal Thrombin-Antithrombin III (TAT) Assay Results | Month 6 (n= 3, 6) | 7.9 ug/L |
| On-demand Arm | Abnormal Thrombin-Antithrombin III (TAT) Assay Results | Month 9 (n= 0, 3) | NA ug/L |
| Prophylaxis Arm | Abnormal Thrombin-Antithrombin III (TAT) Assay Results | Month 9 (n= 0, 3) | 6.0 ug/L |
| Prophylaxis Arm | Abnormal Thrombin-Antithrombin III (TAT) Assay Results | Month 6 (n= 3, 6) | 5.6 ug/L |
| Prophylaxis Arm | Abnormal Thrombin-Antithrombin III (TAT) Assay Results | Screening visit (n= 4, 1) | 5.6 ug/L |
| Prophylaxis Arm | Abnormal Thrombin-Antithrombin III (TAT) Assay Results | Termination visit (n= 2, 5) | 5.6 ug/L |
| Prophylaxis Arm | Abnormal Thrombin-Antithrombin III (TAT) Assay Results | Month 3 (n= 3, 7) | 12.3 ug/L |
Secondary
Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels
Absolute Changes in Inhibitor Titer (or no change in low or high titer status): -Inhibitor Titer went from Low (≤5 BU) to Low (≤5 BU) -Inhibitor Titer went from Low (≤5 BU) to High (\>5 BU) -Inhibitor Titer went from High (\>5 BU) to Low (≤5 BU) -Inhibitor Titer went from High (\>5 BU) to High (\>5 BU)
Time frame: 12 months ± 14 days
Population: Safety Analysis Set - Hemophilia A study participants
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels | Screening to 3 Month- Low to High (N=2, 3) | 12.1 Bethesda Units (BU) |
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels | Screening to 9 Month- Low to High (N=2, 1) | 5.0 Bethesda Units (BU) |
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels | Screening to 6 Month- Low to High (N=2, 1) | 12.9 Bethesda Units (BU) |
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels | Screening to 3 Month- High to Low (N=1, 0) | 2.0 Bethesda Units (BU) |
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels | Screening to Termination- Low to High (N=2, 1) | 39.2 Bethesda Units (BU) |
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels | Screening to 6 Month- High to Low (N=1, 0) | 3.4 Bethesda Units (BU) |
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels | Screening to Termination- High to Low (N=1, 2) | 14.6 Bethesda Units (BU) |
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels | Screening to 9 Month- High to Low (N=0, 2) | NA Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels | Screening to Termination- High to Low (N=1, 2) | 3.3 Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels | Screening to 3 Month- Low to High (N=2, 3) | 4.0 Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels | Screening to 3 Month- High to Low (N=1, 0) | NA Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels | Screening to 6 Month- Low to High (N=2, 1) | 5.3 Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels | Screening to 6 Month- High to Low (N=1, 0) | NA Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels | Screening to 9 Month- Low to High (N=2, 1) | 3.7 Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels | Screening to 9 Month- High to Low (N=0, 2) | 2.1 Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels | Screening to Termination- Low to High (N=2, 1) | 5.8 Bethesda Units (BU) |
Secondary
Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels
Absolute Changes in Inhibitor Titer (or no change in low or high titer status): -Inhibitor Titer went from Low (≤5 BU) to Low (≤5 BU) -Inhibitor Titer went from Low (≤5 BU) to High (\>5 BU) -Inhibitor Titer went from High (\>5 BU) to Low (≤5 BU) -Inhibitor Titer went from High (\>5 BU) to High (\>5 BU)
Time frame: 12 months ± 14 days
Population: Safety Analysis Set - Hemophilia B study participants
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 3 Month- Low to Low (N=1, 0) | 3.2 Bethesda Units (BU) |
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 3 Month- Low to High (N=0, 0) | NA Bethesda Units (BU) |
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 3 Month- High to Low (N=0, 0) | NA Bethesda Units (BU) |
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 3 Month- High to High (N=1, 1) | 0.3 Bethesda Units (BU) |
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 6 Month- Low to Low (N=1, 0) | 1.5 Bethesda Units (BU) |
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 6 Month- Low to High (N=0, 0) | NA Bethesda Units (BU) |
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 6 Month- High to Low (N=0, 0) | NA Bethesda Units (BU) |
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 6 Month- High to High (N=1, 1) | 1.7 Bethesda Units (BU) |
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 9 Month- Low to Low (N=1, 0) | 3.2 Bethesda Units (BU) |
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 9 Month- Low to High (N=0, 0) | NA Bethesda Units (BU) |
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 9 Month- High to Low (N=0, 0) | NA Bethesda Units (BU) |
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 9 Month- High to High (N=0, 1) | NA Bethesda Units (BU) |
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to Termination - Low to Low (N=0, 0) | NA Bethesda Units (BU) |
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to Termination- Low to High (N=0, 0) | NA Bethesda Units (BU) |
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to Termination- High to Low (N=0, 0) | NA Bethesda Units (BU) |
| On-demand Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to Termination - High to High (N=1, 1) | 11.3 Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to Termination - High to High (N=1, 1) | 21.4 Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 3 Month- Low to Low (N=1, 0) | NA Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 9 Month- Low to Low (N=1, 0) | NA Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 3 Month- Low to High (N=0, 0) | NA Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to Termination - Low to Low (N=0, 0) | NA Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 3 Month- High to Low (N=0, 0) | NA Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 9 Month- Low to High (N=0, 0) | NA Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 3 Month- High to High (N=1, 1) | 10.7 Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to Termination- High to Low (N=0, 0) | NA Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 6 Month- Low to Low (N=1, 0) | NA Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 9 Month- High to Low (N=0, 0) | NA Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 6 Month- Low to High (N=0, 0) | NA Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to Termination- Low to High (N=0, 0) | NA Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 6 Month- High to Low (N=0, 0) | NA Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 9 Month- High to High (N=0, 1) | 37.0 Bethesda Units (BU) |
| Prophylaxis Arm | Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels | Screening to 6 Month- High to High (N=1, 1) | 23.2 Bethesda Units (BU) |
Secondary
Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type
Spontaneous includes unknown/undermined etiology
Time frame: 12 months ± 14 days
Population: Efficacy Intent to Treat Analysis Dataset: Dataset consists of data from all randomized participants. Dataset includes those who discontinued FEIBA NF but did not withdraw informed consent and were willing to continue providing data. For these participants, only the data collected before FEIBA NF discontinuation is included.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| On-demand Arm | Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type | Spontaneous | 18.9 Bleeds per year |
| On-demand Arm | Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type | Spontaneous Non-Joint | 1.0 Bleeds per year |
| On-demand Arm | Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type | Non-Joint | 2.9 Bleeds per year |
| On-demand Arm | Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type | Traumatic Joint | 4.0 Bleeds per year |
| On-demand Arm | Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type | Joint | 22.9 Bleeds per year |
| On-demand Arm | Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type | Traumatic Non-Joint | 0.0 Bleeds per year |
| On-demand Arm | Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type | Spontaneous Joint | 16.6 Bleeds per year |
| On-demand Arm | Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type | All Bleeding Etiologies, and Bleed Types | 28.7 Bleeds per year |
| On-demand Arm | Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type | Traumatic | 4.7 Bleeds per year |
| Prophylaxis Arm | Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type | All Bleeding Etiologies, and Bleed Types | 7.9 Bleeds per year |
| Prophylaxis Arm | Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type | Traumatic | 2.5 Bleeds per year |
| Prophylaxis Arm | Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type | Spontaneous | 5.6 Bleeds per year |
| Prophylaxis Arm | Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type | Joint | 6.0 Bleeds per year |
| Prophylaxis Arm | Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type | Non-Joint | 0.5 Bleeds per year |
| Prophylaxis Arm | Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type | Spontaneous Joint | 4.5 Bleeds per year |
| Prophylaxis Arm | Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type | Spontaneous Non-Joint | 0.0 Bleeds per year |
| Prophylaxis Arm | Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type | Traumatic Joint | 1.0 Bleeds per year |
| Prophylaxis Arm | Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type | Traumatic Non-Joint | 0.0 Bleeds per year |
Secondary
Annualized Bleeding Rate for New Target Joints
Target joints are ≥4 bleeds/6 months in any one of the following joints: ankles, knees, elbows, and hips; a target joint bleeding episode refers to an individual anatomical location.
Time frame: 12 months ± 14 days
Population: Efficacy Intent to Treat Analysis Dataset: Dataset consists of data from all randomized participants. Dataset includes those who discontinued FEIBA NF but did not withdraw informed consent and were willing to continue providing data. For these participants, only the data collected before FEIBA NF discontinuation is included.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| On-demand Arm | Annualized Bleeding Rate for New Target Joints | 5.9 Bleeds per year |
| Prophylaxis Arm | Annualized Bleeding Rate for New Target Joints | 0.0 Bleeds per year |
Secondary
Assessment of Clinical Symptoms - Range of Motion (ROM)
ROM was measured using a goniometer for 3 key joints (ie, ankles, knees, and elbows) at screening, month 6, and termination (end of study visit)
Time frame: 12 months ± 14 days
Population: Safety Analysis Set
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Knee - Flexion: Termination (n = 18,16) | 105.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Pronation: Month 6 (n = 17,14) | 80.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Knee - Extension: Screening (n = 18,16) | 35.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Flexion: Screening (n = 19,17) | 120.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Knee - Extension: Month 6 (n = 17,14) | 20.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Extension: Month 6 (n = 18,16) | 10.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Knee - Extension: Termination (n = 18,15) | 17.5 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Flexion: Month 6 (n = 18,16) | 122.5 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Knee - Flexion: Screening (n = 19,17) | 90.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Pronation: Termination (n = 17,15) | 68.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Knee - Flexion: Month 6 (n = 18,16) | 90.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Flexion: Termination (n = 18,16) | 121.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Knee - Flexion: Termination (n = 18,16) | 85.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Flexion: Month 6 (n = 18,16) | 130.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Dorsiflexion: Screening (n = 18,16) | 20.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Pronation: Screening (n = 18,16) | 75.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Dorsiflexion: Month 6 (n = 17,14) | 20.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Supination: Screening (n = 18,16) | 75.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Dorsiflexion: Termination (n = 17,15) | 15.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Pronation: Month 6 (n = 17,14) | 80.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Plantarflexion: Screening (n = 18,16) | 34.5 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Extension: Screening | 15.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Plantarflexion: Month 6 (n = 17,14) | 34.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Pronation: Termination (n = 18,15) | 67.5 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Plantarflexion: Termination (n=17,15) | 35.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Supination: Month 6 (n = 17,14) | 80.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Pronation: Screening (n = 19,17) | 14.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Supination: Screening (n = 18,16) | 75.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Pronation: Month 6 (n = 18,15) | 15.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Flexion: Termination (n = 17,16) | 110.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Pronation: Termination (n = 18,16) | 15.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Supination: Month 6 (n = 17,14) | 70.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Supination: Screening (n = 19,17) | 25.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Supination: Termination (n = 17,15) | 70.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Supination: Month 6 (n = 18,15) | 25.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Supination: Termination (n = 18,15) | 75.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Supination: Termination (n = 18,16) | 25.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Extension: Termination (n = 17,16) | 20.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Dorsiflexion: Screening (n = 18,16) | 15.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Knee - Extension: Screening (n = 18,16) | 15.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Dorsiflexion: Month 6 (n = 17,14) | 15.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Extension: Screening (n = 19,17) | 20.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Dorsiflexion: Termination (n= 17,15) | 15.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Knee - Extension: Month 6 (n = 17,14) | 15.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Plantarflexion: Screening (n= 18,16) | 35.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Pronation: Screening (n = 18,16) | 64.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Plantarflexion: Termination(n=17,15) | 30.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Knee - Extension: Termination (n = 18,15) | 15.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Pronation: Screening (n = 19,17) | 10.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Extension: Month 6 (n = 18,16) | 17.5 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Pronation: Month 6 (n= 18,15) | 10.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Knee - Flexion: Screening (n = 19,17) | 100.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Pronation: Termination (n= 18,16) | 10.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Flexion: Screening (n = 19,17) | 125.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Supination: Screening (n = 19,17) | 17.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Knee - Flexion: Month 6 (n = 18,16) | 112.5 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Supination: Month 6 (n= 18,15) | 20.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Extension: Termination (n = 18,16) | 22.5 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Supination: Termination (n= 18,16) | 20.0 degrees |
| On-demand Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Plantarflexion: Month 6 (n= 17,14) | 30.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Supination: Termination (n= 18,16) | 20.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Plantarflexion: Month 6 (n= 17,14) | 23.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Extension: Screening | 10.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Extension: Month 6 (n = 18,16) | 8.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Extension: Termination (n = 17,16) | 15.5 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Flexion: Screening (n = 19,17) | 115.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Flexion: Month 6 (n = 18,16) | 125.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Flexion: Termination (n = 17,16) | 125.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Pronation: Screening (n = 18,16) | 55.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Pronation: Month 6 (n = 17,14) | 67.5 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Pronation: Termination (n = 17,15) | 65.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Supination: Screening (n = 18,16) | 42.5 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Supination: Month 6 (n = 17,14) | 60.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Elbow - Supination: Termination (n = 17,15) | 70.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Extension: Screening (n = 19,17) | 10.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Extension: Month 6 (n = 18,16) | 10.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Extension: Termination (n = 18,16) | 20.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Flexion: Screening (n = 19,17) | 100.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Flexion: Month 6 (n = 18,16) | 100.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Flexion: Termination (n = 18,16) | 99.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Pronation: Screening (n = 18,16) | 57.5 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Pronation: Month 6 (n = 17,14) | 72.5 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Pronation: Termination (n = 18,15) | 70.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Supination: Screening (n = 18,16) | 52.5 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Supination: Month 6 (n = 17,14) | 72.5 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Elbow - Supination: Termination (n = 18,15) | 70.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Knee - Extension: Screening (n = 18,16) | 1.5 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Knee - Extension: Month 6 (n = 17,14) | 0.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Knee - Extension: Termination (n = 18,15) | 0.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Knee - Flexion: Screening (n = 19,17) | 90.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Knee - Flexion: Month 6 (n = 18,16) | 97.5 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Knee - Flexion: Termination (n = 18,16) | 92.5 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Knee - Extension: Screening (n = 18,16) | 5.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Knee - Extension: Month 6 (n = 17,14) | 10.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Knee - Extension: Termination (n = 18,15) | 5.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Knee - Flexion: Screening (n = 19,17) | 80.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Knee - Flexion: Month 6 (n = 18,16) | 82.5 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Knee - Flexion: Termination (n = 18,16) | 87.5 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Dorsiflexion: Screening (n = 18,16) | 17.5 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Dorsiflexion: Month 6 (n = 17,14) | 17.5 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Dorsiflexion: Termination (n = 17,15) | 20.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Plantarflexion: Screening (n = 18,16) | 30.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Plantarflexion: Month 6 (n = 17,14) | 30.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Plantarflexion: Termination (n=17,15) | 30.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Pronation: Screening (n = 19,17) | 15.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Pronation: Month 6 (n = 18,15) | 15.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Pronation: Termination (n = 18,16) | 12.5 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Supination: Screening (n = 19,17) | 15.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Supination: Month 6 (n = 18,15) | 25.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Left Ankle - Supination: Termination (n = 18,16) | 20.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Dorsiflexion: Screening (n = 18,16) | 20.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Dorsiflexion: Month 6 (n = 17,14) | 25.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Dorsiflexion: Termination (n= 17,15) | 20.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Plantarflexion: Screening (n= 18,16) | 20.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Plantarflexion: Termination(n=17,15) | 21.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Pronation: Screening (n = 19,17) | 10.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Pronation: Month 6 (n= 18,15) | 10.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Pronation: Termination (n= 18,16) | 11.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Supination: Screening (n = 19,17) | 20.0 degrees |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Range of Motion (ROM) | Right Ankle - Supination: Month 6 (n= 18,15) | 20.0 degrees |
Secondary
Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old)
Pain caused by a bleeding episode (BE) in pediatric participants (\<12 years old) was measured at pre-infusion (pre-inf) and at 6 ± 0.5 h and 24 ± 1 h post-infusion (post-inf) (after the last infusion given to treat a bleeding episode) using the children's VAS pain scale (a facial expression scale with one end marked as no pain and the opposite end marked as the worst possible pain). For analysis purposes, if short acting analgesics (duration of activity approximately 6 ± 0.5 h) were used, pain was assigned the highest possible score (worst possible pain). Scores on the children's VAS scale are presented as: -No Pain -Mild Pain -Moderate pain -Severe pain -Very severe pain
Time frame: 12 months ± 14 days
Population: Additional evaluations for bleeding episodes in the intent-to-treat analysis dataset: consists of all participants with at least 1 bleeding episode treated with investigational product.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| On-demand Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | Pre-Infusion (N= 54, 8) - Very Severe Pain | 2 Bleeding episodes |
| On-demand Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | ± 0.5 h post-infusion (N= 74, 8) - Severe Pain | 3 Bleeding episodes |
| On-demand Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | Pre-Infusion (N= 54, 8) - Mild Pain | 3 Bleeding episodes |
| On-demand Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | ± 0.5 h post-infusion (N= 74, 8)- Very Severe Pain | 0 Bleeding episodes |
| On-demand Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | 6 ± 0.5 h post-infusion (N= 74, 8) - No Pain | 47 Bleeding episodes |
| On-demand Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | 24 ± 1 h post-infusion (N= 77, 9) - No Pain | 60 Bleeding episodes |
| On-demand Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | Pre-Infusion (N= 54, 8) - Severe Pain | 10 Bleeding episodes |
| On-demand Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | 24 ± 1 h post-infusion (N= 77, 9) - Mild Pain | 11 Bleeding episodes |
| On-demand Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | ± 0.5 h post-infusion (N= 74, 8) - Mild Pain | 9 Bleeding episodes |
| On-demand Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | 24 ± 1 h post-infusion (N= 77, 9) - Moderate Pain | 6 Bleeding episodes |
| On-demand Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | Pre-Infusion (N= 54, 8) - Moderate Pain | 12 Bleeding episodes |
| On-demand Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | 24 ± 1 h post-infusion (N= 77, 9) - Severe Pain | 0 Bleeding episodes |
| On-demand Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | ± 0.5 h post-infusion (N= 74, 8) - Moderate Pain | 15 Bleeding episodes |
| On-demand Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | 24± 1h post-infusion (N = 77, 9) -Very Severe Pain | 0 Bleeding episodes |
| On-demand Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | Pre-Infusion (N= 54, 8) - No Pain | 27 Bleeding episodes |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | 24± 1h post-infusion (N = 77, 9) -Very Severe Pain | 0 Bleeding episodes |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | Pre-Infusion (N= 54, 8) - No Pain | 3 Bleeding episodes |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | Pre-Infusion (N= 54, 8) - Mild Pain | 1 Bleeding episodes |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | Pre-Infusion (N= 54, 8) - Moderate Pain | 3 Bleeding episodes |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | Pre-Infusion (N= 54, 8) - Severe Pain | 1 Bleeding episodes |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | Pre-Infusion (N= 54, 8) - Very Severe Pain | 0 Bleeding episodes |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | 6 ± 0.5 h post-infusion (N= 74, 8) - No Pain | 3 Bleeding episodes |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | ± 0.5 h post-infusion (N= 74, 8) - Mild Pain | 3 Bleeding episodes |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | ± 0.5 h post-infusion (N= 74, 8) - Moderate Pain | 2 Bleeding episodes |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | ± 0.5 h post-infusion (N= 74, 8) - Severe Pain | 0 Bleeding episodes |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | ± 0.5 h post-infusion (N= 74, 8)- Very Severe Pain | 0 Bleeding episodes |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | 24 ± 1 h post-infusion (N= 77, 9) - No Pain | 7 Bleeding episodes |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | 24 ± 1 h post-infusion (N= 77, 9) - Mild Pain | 1 Bleeding episodes |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | 24 ± 1 h post-infusion (N= 77, 9) - Moderate Pain | 1 Bleeding episodes |
| Prophylaxis Arm | Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old) | 24 ± 1 h post-infusion (N= 77, 9) - Severe Pain | 0 Bleeding episodes |
Secondary
Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 24 Hours
Number of rAHF-PFM-treated bleeding episodes with an assessment of hemostasis (4-point ordinal scale): Excellent: Full pain relief & bleeding cessation within \ 24 hours of 1 infusion. Additional infusions may have been given to maintain hemostasis; Good: Definite pain relief and/or improvement in bleeding within \ 24 hours after infusion. Possibly requires \>1 infusion for complete resolution; Fair: Probable or slight relief of pain & slight improvement in bleeding within \ 24 hours after infusion. Requires \>1 infusion for complete resolution; None: No improvement or condition worsens
Time frame: 24 ± 1 h post-infusion
Population: Bleeding Episodes Analysis Set Consists of all participants who experienced a bleeding episode (BE)
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| On-demand Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 24 Hours | Excellent (n = 16, 6) | 282 bleeding episodes |
| On-demand Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 24 Hours | Good (n = 19, 14) | 280 bleeding episodes |
| On-demand Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 24 Hours | Fair (n = 9, 3) | 36 bleeding episodes |
| On-demand Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 24 Hours | None (n = 1,0) | 1 bleeding episodes |
| On-demand Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 24 Hours | Rating Not Done (n = 8, 3) | 24 bleeding episodes |
| On-demand Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 24 Hours | Not Available (n = 0, 1) | 0 bleeding episodes |
| Prophylaxis Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 24 Hours | Rating Not Done (n = 8, 3) | 28 bleeding episodes |
| Prophylaxis Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 24 Hours | Excellent (n = 16, 6) | 42 bleeding episodes |
| Prophylaxis Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 24 Hours | None (n = 1,0) | 0 bleeding episodes |
| Prophylaxis Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 24 Hours | Good (n = 19, 14) | 89 bleeding episodes |
| Prophylaxis Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 24 Hours | Not Available (n = 0, 1) | 2 bleeding episodes |
| Prophylaxis Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 24 Hours | Fair (n = 9, 3) | 12 bleeding episodes |
Secondary
Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 6 Hours
Number of rAHF-PFM-treated bleeding episodes with an assessment of hemostasis (4-point ordinal scale): Excellent: Full pain relief & bleeding cessation within \ 6 hours of 1 infusion. Additional infusions may have been given to maintain hemostasis; Good: Definite pain relief and/or improvement in bleeding within \ 6 hours after infusion. Possibly requires \>1 infusion for complete resolution; Fair: Probable or slight relief of pain & slight improvement in bleeding within \ 6 hours after infusion. Requires \>1 infusion for complete resolution; None: No improvement or condition worsens
Time frame: 6 h ± 30 min post-infusion
Population: Bleeding Episodes Analysis Set Consists of all participants who experienced a bleeding episode (BE)
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| On-demand Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 6 Hours | Excellent (n = 16, 5) | 109 bleeding episodes |
| On-demand Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 6 Hours | Good (n = 19, 14) | 355 bleeding episodes |
| On-demand Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 6 Hours | Fair (n = 15, 6) | 142 bleeding episodes |
| On-demand Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 6 Hours | None (n = 6, 2) | 14 bleeding episodes |
| On-demand Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 6 Hours | Rating Not Done (n = 3, 2) | 3 bleeding episodes |
| On-demand Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 6 Hours | Not Available (n = 0, 1) | 0 bleeding episodes |
| Prophylaxis Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 6 Hours | Rating Not Done (n = 3, 2) | 2 bleeding episodes |
| Prophylaxis Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 6 Hours | Excellent (n = 16, 5) | 16 bleeding episodes |
| Prophylaxis Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 6 Hours | None (n = 6, 2) | 5 bleeding episodes |
| Prophylaxis Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 6 Hours | Good (n = 19, 14) | 106 bleeding episodes |
| Prophylaxis Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 6 Hours | Not Available (n = 0, 1) | 2 bleeding episodes |
| Prophylaxis Arm | Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 6 Hours | Fair (n = 15, 6) | 42 bleeding episodes |
Secondary
Assessment of Objective Clinical Symptoms- Visual Analog Scale (VAS): Pain in Adolescents and Adults (≥12 Years Old)
Pain caused by a bleeding episode in adolescents and adults (≥12 years old) was measured at pre-infusion (pre-inf) and at 6 ± 0.5 hours (h) and 24 ± 1 h post-infusion (post-inf) (after the last infusion given to treat a bleeding episode) on the VAS pain scale in millimeters from 0 (no pain) to 100 (worst possible pain). For analysis purposes, if short acting analgesics (duration of activity approximately 6 ± 0.5 h) were used, pain was assigned the highest possible score (100). Pain assessment occurred after each infusion related to single bleeding episodes. In case participants required an additional infusion within 24h, pain was assessed 6 ± 0.5 h and 24 ±1 h following the subsequent infusion. Change in VAS scores at 6 ± 0.5 h and 24 ±1 h post-infusion were also compared relative to pre-infusion VAS scores (ie, (pre-infusion VAS score) - (post-infusion VAS score)).
Time frame: Throughout the study period, 12 months ± 14 days
Population: Additional evaluations for bleeding episodes in the intent-to-treat analysis dataset: consists of all participants with at least 1 bleeding episode treated with investigational product.
| Arm | Measure | Group | Value (MEDIAN) | Dispersion |
|---|---|---|---|---|
| On-demand Arm | Assessment of Objective Clinical Symptoms- Visual Analog Scale (VAS): Pain in Adolescents and Adults (≥12 Years Old) | 6 ± 0.5 hours post-infusion (N= 522, 158) | 10.0 Scores on a scale | Inter-Quartile Range 33 |
| On-demand Arm | Assessment of Objective Clinical Symptoms- Visual Analog Scale (VAS): Pain in Adolescents and Adults (≥12 Years Old) | Change (Pre-Inf to 6h post-inf) (N= 509, 129) | 10.0 Scores on a scale | — |
| On-demand Arm | Assessment of Objective Clinical Symptoms- Visual Analog Scale (VAS): Pain in Adolescents and Adults (≥12 Years Old) | 24 ± 1 hours post-infusion (N= 512,131) | 3.0 Scores on a scale | Inter-Quartile Range 13 |
| On-demand Arm | Assessment of Objective Clinical Symptoms- Visual Analog Scale (VAS): Pain in Adolescents and Adults (≥12 Years Old) | Change (Pre-Inf to 24h post-inf) (N= 489, 102) | 18.0 Scores on a scale | — |
| On-demand Arm | Assessment of Objective Clinical Symptoms- Visual Analog Scale (VAS): Pain in Adolescents and Adults (≥12 Years Old) | Pre-Infusion (N= 513, 130) | 29.0 Scores on a scale | Inter-Quartile Range 43 |
| Prophylaxis Arm | Assessment of Objective Clinical Symptoms- Visual Analog Scale (VAS): Pain in Adolescents and Adults (≥12 Years Old) | Change (Pre-Inf to 24h post-inf) (N= 489, 102) | 37.8 Scores on a scale | — |
| Prophylaxis Arm | Assessment of Objective Clinical Symptoms- Visual Analog Scale (VAS): Pain in Adolescents and Adults (≥12 Years Old) | Pre-Infusion (N= 513, 130) | 49.5 Scores on a scale | Inter-Quartile Range 46.9 |
| Prophylaxis Arm | Assessment of Objective Clinical Symptoms- Visual Analog Scale (VAS): Pain in Adolescents and Adults (≥12 Years Old) | 6 ± 0.5 hours post-infusion (N= 522, 158) | 33.8 Scores on a scale | Inter-Quartile Range 44.6 |
| Prophylaxis Arm | Assessment of Objective Clinical Symptoms- Visual Analog Scale (VAS): Pain in Adolescents and Adults (≥12 Years Old) | 24 ± 1 hours post-infusion (N= 512,131) | 6.2 Scores on a scale | Inter-Quartile Range 19 |
| Prophylaxis Arm | Assessment of Objective Clinical Symptoms- Visual Analog Scale (VAS): Pain in Adolescents and Adults (≥12 Years Old) | Change (Pre-Inf to 6h post-inf) (N= 509, 129) | 19.4 Scores on a scale | — |
Secondary
Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens by Bleeding Etiology, and Bleeding Type
Annualized bleed rates were transformed using the square root of the number of bleeding episodes observed (X bleeds/year), X' = √(X + 0.5). This transformation was performed to stabilize the variance and align the sample distribution with the assumption of normality inherent in using the t-test. The difference in mean transformed ABRs was used to perform statistical tests and generate p-values at a significance level of 5% Participants were Randomized to Receive 1 of the 2 Following Treatment Regimens: 1.On-Demand: FEIBA NF dose & dosing interval as prescribed by treating physician 2.Prophylaxis: 85 ± 15 U/kg of FEIBA NF every other day during 12-month prophylactic period
Time frame: 12 months ± 14 days
Population: Efficacy Intent to Treat Analysis Dataset
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| On-demand Arm | Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens by Bleeding Etiology, and Bleeding Type | Spontaneous Bleeds | 2.2 (bleeds/year)^(1/2) | Standard Deviation 1.8 |
| On-demand Arm | Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens by Bleeding Etiology, and Bleeding Type | Traumatic Bleeds | 1.0 (bleeds/year)^(1/2) | Standard Deviation 1.2 |
| On-demand Arm | Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens by Bleeding Etiology, and Bleeding Type | Joint Bleeds | 2.4 (bleeds/year)^(1/2) | Standard Deviation 1.9 |
| On-demand Arm | Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens by Bleeding Etiology, and Bleeding Type | Non-Joint Bleeds | 0.8 (bleeds/year)^(1/2) | Standard Deviation 0.9 |
| On-demand Arm | Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens by Bleeding Etiology, and Bleeding Type | Spontaneous Joint Bleeds | 2.1 (bleeds/year)^(1/2) | Standard Deviation 1.8 |
| On-demand Arm | Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens by Bleeding Etiology, and Bleeding Type | Spontaneous Non-Joint Bleeds | 0.8 (bleeds/year)^(1/2) | Standard Deviation 0.8 |
| On-demand Arm | Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens by Bleeding Etiology, and Bleeding Type | Traumatic Joint Bleeds | 0.9 (bleeds/year)^(1/2) | Standard Deviation 1.2 |
| On-demand Arm | Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens by Bleeding Etiology, and Bleeding Type | Traumatic Non-Joint Bleeds | -0.0 (bleeds/year)^(1/2) | Standard Deviation 0.9 |
Comparison: Spontaneous Bleedsp-value: 0.0008Two-sample, two-sided t-test
Comparison: Traumatic Bleedsp-value: 0.0199Two-sample, two-sided t-test
Comparison: Joint Bleedsp-value: 0.0006Two-sample, two-sided t-test
Comparison: Non-Joint Bleedsp-value: 0.0227Two-sample, two-sided t-test
Comparison: Spontaneous Joint Bleedsp-value: 0.0013Two-sample, two-sided t-test
Comparison: Spontaneous Non-Joint Bleedsp-value: 0.003Two-sample, two-sided t-test
Comparison: Traumatic Joint Bleedsp-value: 0.0254Two-sample, two-sided t-test
Comparison: Traumatic Non-Joint Bleedsp-value: 0.9322Two-sample, two-sided t-test
Secondary
Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens: New Target Joints
Annualized bleed rates (ABRs) were transformed using the square root of the number of bleeding episodes observed (X bleeds/year), X' = √(X + 0.5). This transformation was performed to stabilize the variance and align the sample distribution with the assumption of normality inherent in using a two-sample, two-sided t-test. The difference in mean transformed ABRs was used to perform statistical tests and generate p-values at a significance level of 5%
Time frame: 12 months ± 14 days
Population: Efficacy Intent to Treat Analysis Dataset: Dataset consists of data from all randomized participants. Dataset includes those who discontinued FEIBA NF but did not withdraw informed consent and were willing to continue providing data. For these participants, only the data collected before FEIBA NF discontinuation is included.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| On-demand Arm | Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens: New Target Joints | 1.6 (bleeds/year)^(1/2) | Standard Deviation 2.2 |
p-value: 0.0271Two-sample, two-sided t-test
Secondary
Health-Related Quality of Life (HRQoL): EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Index Scores
EQ-5D is a participant answered questionnaire scoring 5 dimensions - mobility, self-care, usual activities, pain/discomfort and anxiety/depression. The EQ-5D total score ranges from 0 (worst health state) to 1 (perfect health state) and 1 reflects the best outcome. EQ-5D Index scores based on EQ-5D questionnaire were calculated for participants ≥14 years of age, at screening, 6 months, and at termination visit. Changes in scores at 6 months and termination were also calculated. A relatively higher score represents better quality of life.
Time frame: 12 months ± 14 days
Population: HRQoL Intent-to-Treat Analysis Dataset - comprised of all participants ≥14 years of age who were randomized, had any available assessments at any available study visits (baseline, 6- month, and 12-month) as defined in the protocol
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| On-demand Arm | Health-Related Quality of Life (HRQoL): EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Index Scores | 6 Months (N= 15, 11) | 0.621 Scores on a scale | Standard Deviation 0.188 |
| On-demand Arm | Health-Related Quality of Life (HRQoL): EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Index Scores | Change (Screening - Month 6) (N= 15, 11) | -0.006 Scores on a scale | Standard Deviation 0.2088 |
| On-demand Arm | Health-Related Quality of Life (HRQoL): EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Index Scores | Screening (N= 16, 12) | 0.627 Scores on a scale | Standard Deviation 0.2067 |
| On-demand Arm | Health-Related Quality of Life (HRQoL): EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Index Scores | Change (Screening - Termination) (N= 15, 10) | 0.010 Scores on a scale | Standard Deviation 0.247 |
| On-demand Arm | Health-Related Quality of Life (HRQoL): EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Index Scores | Termination (N= 15, 10) | 0.605 Scores on a scale | Standard Deviation 0.2146 |
| On-demand Arm | Health-Related Quality of Life (HRQoL): EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Index Scores | Change (Month 6 - Termination) (N= 15, 10) | 0.016 Scores on a scale | Standard Deviation 0.1765 |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL): EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Index Scores | Termination (N= 15, 10) | 0.700 Scores on a scale | Standard Deviation 0.1233 |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL): EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Index Scores | Screening (N= 16, 12) | 0.620 Scores on a scale | Standard Deviation 0.1841 |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL): EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Index Scores | 6 Months (N= 15, 11) | 0.729 Scores on a scale | Standard Deviation 0.1392 |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL): EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Index Scores | Change (Month 6 - Termination) (N= 15, 10) | 0.001 Scores on a scale | Standard Deviation 0.1493 |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL): EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Index Scores | Change (Screening - Month 6) (N= 15, 11) | -0.096 Scores on a scale | Standard Deviation 0.2403 |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL): EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Index Scores | Change (Screening - Termination) (N= 15, 10) | -0.075 Scores on a scale | Standard Deviation 0.2594 |
Secondary
Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Adults and Adolescents ≥12 Years Old
General pain was assessed using a VAS pain scale at screening, 6 months, and at termination. Unlike the VAS pain assessment for pain of bleeding episodes (Outcome above), this general pain assessment did not take use of analgesics into account. For the pain scale, a higher number indicates worse pain. The visual analog scale ranges from 0 to 100 where the endpoints are labeled 'Worst imaginable health state' (=0) and 'Best imaginable health state' (=100). A positive change from baseline indicates improvement. Change in VAS scores at 6 months and study termination were also compared relative to Baseline/Screening scores (ie, (Baseline/Screening VAS score) - (VAS score at 6 months and study termination).
Time frame: Baseline, 6 months and 12 months ± 14 days
Population: Health-Related Quality of Life (HRQoL) Intent-to-Treat Analysis Dataset - comprised of all participants ≥12 years old who were randomized, had any available assessments at any available study visits (baseline, 6-month, and 12-month) as defined in the protocol
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| On-demand Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Adults and Adolescents ≥12 Years Old | Screening (N= 17, 15) | 35.2 Scores on a scale | Standard Deviation 30.15 |
| On-demand Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Adults and Adolescents ≥12 Years Old | Month 6 (N= 16, 14) | 36.6 Scores on a scale | Standard Deviation 25.81 |
| On-demand Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Adults and Adolescents ≥12 Years Old | Termination (N= 16, 14) | 32.4 Scores on a scale | Standard Deviation 21.97 |
| On-demand Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Adults and Adolescents ≥12 Years Old | Change (Screening - Month 6) (N= 16, 14) | 0.8 Scores on a scale | Standard Deviation 31.77 |
| On-demand Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Adults and Adolescents ≥12 Years Old | Change (Screening - Termination) (N= 16, 14) | 5.0 Scores on a scale | Standard Deviation 28.7 |
| On-demand Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Adults and Adolescents ≥12 Years Old | Change (Month 6 - Termination) (N= 16, 14) | 4.2 Scores on a scale | Standard Deviation 21.82 |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Adults and Adolescents ≥12 Years Old | Change (Screening - Termination) (N= 16, 14) | 23.2 Scores on a scale | Standard Deviation 46.61 |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Adults and Adolescents ≥12 Years Old | Screening (N= 17, 15) | 55.5 Scores on a scale | Standard Deviation 23.68 |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Adults and Adolescents ≥12 Years Old | Change (Screening - Month 6) (N= 16, 14) | 20.3 Scores on a scale | Standard Deviation 38.91 |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Adults and Adolescents ≥12 Years Old | Month 6 (N= 16, 14) | 32.7 Scores on a scale | Standard Deviation 26.24 |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Adults and Adolescents ≥12 Years Old | Change (Month 6 - Termination) (N= 16, 14) | 2.9 Scores on a scale | Standard Deviation 19.62 |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Adults and Adolescents ≥12 Years Old | Termination (N= 16, 14) | 29.8 Scores on a scale | Standard Deviation 30.48 |
Secondary
Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old
General pain was assessed using the children's VAS pain scale (a facial expression scale with one end marked as no pain and the opposite end marked as the worst possible pain). Assessments were done at the screening, 6 months, and termination visits. Scores on the children's VAS scale are presented as: -No Pain -Mild Pain -Moderate pain -Severe pain -Very severe pain Unlike the VAS pain assessment for pain of bleeding episodes (Outcome above), this general pain assessment did not take use of analgesics into account. Change in VAS scores at 6 months and study termination were also compared relative to Baseline/Screening scores (ie, (Baseline/Screening VAS score) - (VAS score at 6 months and study termination).
Time frame: Baseline, 6 months and 12 months ± 14 days
Population: Health-Related Quality of Life (HRQoL) Intent-to-Treat Analysis Dataset - comprised of all participants \<12 years old who were randomized, had any available assessments at any available study visits (baseline, 6-month, and 12-month) as defined in the protocol
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| On-demand Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Screening - Very Severe Pain | 0 participants |
| On-demand Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Month 6 - Severe Pain | 0 participants |
| On-demand Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Screening - Mild Pain | 0 participants |
| On-demand Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Month 6 - Very Severe Pain | 0 participants |
| On-demand Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Month 6 - No Pain | 0 participants |
| On-demand Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Termination visit - No Pain | 0 participants |
| On-demand Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Screening - Severe Pain | 0 participants |
| On-demand Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Termination visit - Mild Pain | 0 participants |
| On-demand Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Month 6 - Mild Pain | 1 participants |
| On-demand Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Termination visit - Moderate Pain | 1 participants |
| On-demand Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Screening - Moderate Pain | 1 participants |
| On-demand Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Termination visit - Severe Pain | 0 participants |
| On-demand Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Month 6 - Moderate Pain | 1 participants |
| On-demand Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Termination visit - Very Severe Pain | 1 participants |
| On-demand Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Screening - No Pain | 1 participants |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Termination visit - Very Severe Pain | 0 participants |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Screening - No Pain | 0 participants |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Screening - Mild Pain | 1 participants |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Screening - Moderate Pain | 0 participants |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Screening - Severe Pain | 1 participants |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Screening - Very Severe Pain | 0 participants |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Month 6 - No Pain | 0 participants |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Month 6 - Mild Pain | 2 participants |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Month 6 - Moderate Pain | 0 participants |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Month 6 - Severe Pain | 0 participants |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Month 6 - Very Severe Pain | 0 participants |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Termination visit - No Pain | 1 participants |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Termination visit - Mild Pain | 0 participants |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Termination visit - Moderate Pain | 1 participants |
| Prophylaxis Arm | Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old | Termination visit - Severe Pain | 0 participants |
Secondary
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
The Haem-A-QoL instrument has been developed and used in Hemophilia A patients. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. The areas covered by this instrument are: Physical Health (PH), Sports & Leisure (S&L), School & Work (W&S), Dealing with Hemophilia (Dealing), Family Planning (FP), Feeling, Relationships (R'ships), Treatment, View, and Outlook for the Future (Future). A Haem-A-QoL Total Score (Total) was also calculated. For the Haem-A-QoL, higher scores indicate a worse quality of life. Scores on a scale range between 0 and 100. Haem-A-QoL scores at screening, 6 months, and at termination visit were collected. Changes in scores at 6 months and termination were also calculated.
Time frame: 12 months ± 14 days
Population: Health-Related Quality of Life (HRQoL) Intent-to-Treat Analysis Dataset for all participants ≥ 16 years old who were randomized, had any available assessments at any available study visits (baseline, 6-month, and 12-month) as defined in the protocol
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | FP- Change (Screening - Month 6) (N=9, 7) | -3.7 Scores on a scale | Standard Deviation 26.94 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | PH- Termination (N=14, 11) | 56.1 Scores on a scale | Standard Deviation 24.98 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Dealing- Month 6 (N=14, 11) | 25.0 Scores on a scale | Standard Deviation 16.67 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | PH- Change (Screening - Month 6) (N=14, 11) | 14.6 Scores on a scale | Standard Deviation 21.7 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | FP- Change(Screening - Termination)(N=9, 7) | -2.5 Scores on a scale | Standard Deviation 25.57 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | PH- Change(Screening - Termination)(N=14, 11) | 9.3 Scores on a scale | Standard Deviation 23.93 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | View- Termination (N=14, 11) | 47.9 Scores on a scale | Standard Deviation 22.59 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | PH- Change (Month 6 - Termination)(N=14, 11) | -5.3 Scores on a scale | Standard Deviation 10.65 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | FP- Change (Month 6 - Termination)(N=11, 6) | -0.8 Scores on a scale | Standard Deviation 32.68 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | R'ships- Screening (N=15, 11) | 28.3 Scores on a scale | Standard Deviation 36.57 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Dealing- Termination (N=14, 11) | 25.6 Scores on a scale | Standard Deviation 25.21 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | R'ships- Month 6 (N=14, 11) | 19.0 Scores on a scale | Standard Deviation 28.2 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Feeling- Screening (N=15, 12) | 39.6 Scores on a scale | Standard Deviation 28.22 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | R'ships- Termination (N=14, 10) | 17.9 Scores on a scale | Standard Deviation 22.13 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | W&S-Screening (N=13, 10) | 43.8 Scores on a scale | Standard Deviation 25.9 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | R'ships- Change (Screening - Month 6) (N=14, 10) | 7.7 Scores on a scale | Standard Deviation 34.04 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Feeling- Month 6 (N=14, 11) | 33.9 Scores on a scale | Standard Deviation 28.77 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | R'ships- Change(Screening - Termination)(N=14, 10) | 8.9 Scores on a scale | Standard Deviation 30.57 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Dealing- Change (Screening - Month 6) (N=14, 11) | 10.7 Scores on a scale | Standard Deviation 18.61 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | R'ships- Change (Month 6 - Termination)(N=14, 10) | 1.2 Scores on a scale | Standard Deviation 21.4 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Feeling- Termination (N=14, 11) | 30.8 Scores on a scale | Standard Deviation 28.53 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | S&L- Screening (N=12, 8) | 69.3 Scores on a scale | Standard Deviation 17.11 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | View- Change (Screening -Termination)(N=14, 11) | 3.9 Scores on a scale | Standard Deviation 23.79 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | S&L- Month 6 (N=12, 8) | 63.1 Scores on a scale | Standard Deviation 23.09 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Feeling- Change (Screening - Month 6) (N=14, 11) | 4.9 Scores on a scale | Standard Deviation 27.75 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | S&L- Termination (N=12, 8) | 72.3 Scores on a scale | Standard Deviation 17.3 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Dealing- Change(Screening - Termination)(N=14, 11) | 10.1 Scores on a scale | Standard Deviation 27.19 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | S&L- Change (Screening - Month 6) (N=9, 5) | 9.9 Scores on a scale | Standard Deviation 19.75 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Feeling- Change(Screening - Termination)(N=14, 11) | 8.0 Scores on a scale | Standard Deviation 31.91 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | S&L- Change(Screening - Termination)(N=9, 5) | 1.2 Scores on a scale | Standard Deviation 15.91 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | W&S- Month 6 (N=13, 9) | 31.7 Scores on a scale | Standard Deviation 26.82 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | S&L- Change (Month 6 - Termination)(N=11, 7) | -10.2 Scores on a scale | Standard Deviation 13.44 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Feeling- Change (Month 6 - Termination)(N=14, 11) | 3.1 Scores on a scale | Standard Deviation 16.4 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Treatment- Screening (N=15, 12) | 36.9 Scores on a scale | Standard Deviation 13.32 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Dealing- Change (Month 6 - Termination)(N=14, 11) | -0.6 Scores on a scale | Standard Deviation 16.81 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Treatment- Month 6 (N=14, 11) | 30.4 Scores on a scale | Standard Deviation 19.82 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Future- Screening (N=14, 12) | 46.8 Scores on a scale | Standard Deviation 28.46 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Treatment- Termination (N=14, 11) | 34.8 Scores on a scale | Standard Deviation 14.87 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | View- Change (Screening - Month 6) (N=14, 11) | 7.5 Scores on a scale | Standard Deviation 23.68 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Treatment- Change (Screening - Month 6) (N=14, 11) | 6.5 Scores on a scale | Standard Deviation 23.72 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Future- Month 6 (N=14, 11) | 40.2 Scores on a scale | Standard Deviation 26.14 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Treatment-Change(Screening -Termination)(N=14, 11) | 2.1 Scores on a scale | Standard Deviation 17.81 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | FP- Screening (N=9, 8) | 16.7 Scores on a scale | Standard Deviation 20.49 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Treatment- Change (Month 6- Termination)(N=14, 11) | -4.4 Scores on a scale | Standard Deviation 15.07 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Future- Termination (N=14, 11) | 47.9 Scores on a scale | Standard Deviation 25.47 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | View- Screening (N=15, 12) | 52.3 Scores on a scale | Standard Deviation 24.49 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | W&S- Termination (N=12, 9) | 28.6 Scores on a scale | Standard Deviation 22.53 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Future- Change (Screening - Month 6) (N=13, 11) | 4.0 Scores on a scale | Standard Deviation 26.84 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | W&S- Change (Screening - Month 6) (N=12, 8) | 10.9 Scores on a scale | Standard Deviation 30.98 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | FP- Month 6 (N=12, 7) | 20.5 Scores on a scale | Standard Deviation 26.71 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | W&S- Change (Screening -Termination)(N=12, 8) | 16.1 Scores on a scale | Standard Deviation 33.86 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Future- Change(Screening - Termination)(N=13, 11) | -3.8 Scores on a scale | Standard Deviation 20.53 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | W&S- Change (Month 6- Termination)(N=12, 9) | 5.2 Scores on a scale | Standard Deviation 15.95 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Dealing- Screening (N=15, 12) | 35.0 Scores on a scale | Standard Deviation 19.72 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Total- Screening (N=14, 12) | 44.0 Scores on a scale | Standard Deviation 15.5 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Future- Change (Month 6 - Termination)(N=14, 11) | -7.7 Scores on a scale | Standard Deviation 20.25 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Total- Month 6 (N=14, 11) | 37.4 Scores on a scale | Standard Deviation 19.37 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | FP- Termination (N=13, 8) | 19.1 Scores on a scale | Standard Deviation 16.25 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Total- Termination (N=14, 11) | 40.2 Scores on a scale | Standard Deviation 17.55 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | PH- Screening (N=15, 12) | 64.0 Scores on a scale | Standard Deviation 22.46 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Total- Change (Screening - Month 6) (N=13, 11) | 9.2 Scores on a scale | Standard Deviation 18.44 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | View- Change (Month 6- Termination)(N=14, 11) | -3.6 Scores on a scale | Standard Deviation 17.03 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Total- Change (Screening -Termination)(N=13, 11) | 6.1 Scores on a scale | Standard Deviation 15.41 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | PH- Month 6 (N=14, 11) | 50.8 Scores on a scale | Standard Deviation 27.55 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Total- Change (Month 6- Termination)(N=14, 11) | -2.7 Scores on a scale | Standard Deviation 9.21 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | View- Month 6 (N=14, 11) | 44.3 Scores on a scale | Standard Deviation 27.66 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Total- Change (Month 6- Termination)(N=14, 11) | -0.7 Scores on a scale | Standard Deviation 14.4 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | View- Month 6 (N=14, 11) | 40.0 Scores on a scale | Standard Deviation 13.23 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | View- Termination (N=14, 11) | 37.7 Scores on a scale | Standard Deviation 21.26 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | View- Change (Screening - Month 6) (N=14, 11) | 14.3 Scores on a scale | Standard Deviation 17.25 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | View- Change (Screening -Termination)(N=14, 11) | 16.6 Scores on a scale | Standard Deviation 15.01 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | View- Change (Month 6- Termination)(N=14, 11) | 2.3 Scores on a scale | Standard Deviation 20.78 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | W&S-Screening (N=13, 10) | 57.7 Scores on a scale | Standard Deviation 17.87 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | W&S- Month 6 (N=13, 9) | 37.5 Scores on a scale | Standard Deviation 20.73 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Dealing- Screening (N=15, 12) | 26.0 Scores on a scale | Standard Deviation 15.84 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Dealing- Month 6 (N=14, 11) | 18.9 Scores on a scale | Standard Deviation 21.11 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Dealing- Termination (N=14, 11) | 23.7 Scores on a scale | Standard Deviation 22.15 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Dealing- Change (Screening - Month 6) (N=14, 11) | 3.4 Scores on a scale | Standard Deviation 22.62 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Dealing- Change(Screening - Termination)(N=14, 11) | -1.4 Scores on a scale | Standard Deviation 21.37 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Dealing- Change (Month 6 - Termination)(N=14, 11) | -4.8 Scores on a scale | Standard Deviation 21.26 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | FP- Screening (N=9, 8) | 34.9 Scores on a scale | Standard Deviation 31.31 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | FP- Month 6 (N=12, 7) | 14.3 Scores on a scale | Standard Deviation 11.25 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | FP- Termination (N=13, 8) | 21.1 Scores on a scale | Standard Deviation 22.14 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | FP- Change (Screening - Month 6) (N=9, 7) | 14.9 Scores on a scale | Standard Deviation 28.73 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | FP- Change(Screening - Termination)(N=9, 7) | 13.1 Scores on a scale | Standard Deviation 20.26 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | FP- Change (Month 6 - Termination)(N=11, 6) | -8.3 Scores on a scale | Standard Deviation 28.41 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Feeling- Screening (N=15, 12) | 48.2 Scores on a scale | Standard Deviation 29.02 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Feeling- Month 6 (N=14, 11) | 29.0 Scores on a scale | Standard Deviation 17.29 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Feeling- Termination (N=14, 11) | 26.1 Scores on a scale | Standard Deviation 20.69 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Feeling- Change (Screening - Month 6) (N=14, 11) | 17.3 Scores on a scale | Standard Deviation 34.72 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Feeling- Change(Screening - Termination)(N=14, 11) | 20.2 Scores on a scale | Standard Deviation 24.58 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Feeling- Change (Month 6 - Termination)(N=14, 11) | 2.8 Scores on a scale | Standard Deviation 23.94 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Future- Screening (N=14, 12) | 48.6 Scores on a scale | Standard Deviation 23.32 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Future- Month 6 (N=14, 11) | 46.0 Scores on a scale | Standard Deviation 18.03 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Future- Termination (N=14, 11) | 46.8 Scores on a scale | Standard Deviation 18.74 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Future- Change (Screening - Month 6) (N=13, 11) | 1.1 Scores on a scale | Standard Deviation 24.34 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Future- Change(Screening - Termination)(N=13, 11) | 0.3 Scores on a scale | Standard Deviation 16.58 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Future- Change (Month 6 - Termination)(N=14, 11) | -0.8 Scores on a scale | Standard Deviation 16.3 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | PH- Screening (N=15, 12) | 66.4 Scores on a scale | Standard Deviation 24.21 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | PH- Month 6 (N=14, 11) | 43.1 Scores on a scale | Standard Deviation 25.24 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | PH- Termination (N=14, 11) | 42.3 Scores on a scale | Standard Deviation 18.86 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | PH- Change (Screening - Month 6) (N=14, 11) | 21.1 Scores on a scale | Standard Deviation 28.77 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | PH- Change(Screening - Termination)(N=14, 11) | 21.9 Scores on a scale | Standard Deviation 24.79 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | PH- Change (Month 6 - Termination)(N=14, 11) | 0.8 Scores on a scale | Standard Deviation 20.2 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | R'ships- Screening (N=15, 11) | 26.9 Scores on a scale | Standard Deviation 28.83 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | R'ships- Month 6 (N=14, 11) | 15.2 Scores on a scale | Standard Deviation 13.85 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | R'ships- Termination (N=14, 10) | 15.0 Scores on a scale | Standard Deviation 17.48 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | R'ships- Change (Screening - Month 6) (N=14, 10) | 10.4 Scores on a scale | Standard Deviation 27.45 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | R'ships- Change(Screening - Termination)(N=14, 10) | 12.1 Scores on a scale | Standard Deviation 25.49 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | R'ships- Change (Month 6 - Termination)(N=14, 10) | 1.7 Scores on a scale | Standard Deviation 16.57 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | S&L- Screening (N=12, 8) | 83.1 Scores on a scale | Standard Deviation 14.13 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | S&L- Month 6 (N=12, 8) | 69.7 Scores on a scale | Standard Deviation 19.75 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | S&L- Termination (N=12, 8) | 78.1 Scores on a scale | Standard Deviation 12.52 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | S&L- Change (Screening - Month 6) (N=9, 5) | 20.5 Scores on a scale | Standard Deviation 27.97 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | S&L- Change(Screening - Termination)(N=9, 5) | 7.0 Scores on a scale | Standard Deviation 21.68 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | S&L- Change (Month 6 - Termination)(N=11, 7) | -2.5 Scores on a scale | Standard Deviation 15.61 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Treatment- Screening (N=15, 12) | 39.6 Scores on a scale | Standard Deviation 20.7 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Treatment- Month 6 (N=14, 11) | 40.9 Scores on a scale | Standard Deviation 16.96 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Treatment- Termination (N=14, 11) | 42.6 Scores on a scale | Standard Deviation 17.8 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Treatment- Change (Screening - Month 6) (N=14, 11) | 0.0 Scores on a scale | Standard Deviation 16.74 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Treatment-Change(Screening -Termination)(N=14, 11) | -1.7 Scores on a scale | Standard Deviation 23.86 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Treatment- Change (Month 6- Termination)(N=14, 11) | -1.7 Scores on a scale | Standard Deviation 18.7 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | View- Screening (N=15, 12) | 55.6 Scores on a scale | Standard Deviation 11.97 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | W&S- Termination (N=12, 9) | 36.8 Scores on a scale | Standard Deviation 19.63 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | W&S- Change (Screening - Month 6) (N=12, 8) | 25.3 Scores on a scale | Standard Deviation 25.6 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | W&S- Change (Screening -Termination)(N=12, 8) | 25.3 Scores on a scale | Standard Deviation 19.01 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | W&S- Change (Month 6- Termination)(N=12, 9) | 0.7 Scores on a scale | Standard Deviation 21.75 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Total- Screening (N=14, 12) | 49.2 Scores on a scale | Standard Deviation 15.43 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Total- Month 6 (N=14, 11) | 38.1 Scores on a scale | Standard Deviation 11.34 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Total- Termination (N=14, 11) | 38.7 Scores on a scale | Standard Deviation 14.21 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Total- Change (Screening - Month 6) (N=13, 11) | 10.2 Scores on a scale | Standard Deviation 17.84 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old | Total- Change (Screening -Termination)(N=13, 11) | 9.5 Scores on a scale | Standard Deviation 12.77 |
Secondary
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
The Haemo-QoL is a quality of life (QoL) assessment instrument for children and adolescents with haemophilia. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. The areas covered by this instrument are: Physical Health (PH), Sports & School (S&S), Dealing with Hemophilia (Dealing), Family, Feeling, Relationships (R'ships), Treatment, View, Outlook for the Future (Future), Friends, Others, and Support. A Haemo-QoL Total Score (Total) was also calculated. For the Haemo-QoL, higher scores indicate a worse quality of life. Scores on a scale range between 0 and 100. Haemo-QoL scores at screening, 6 months, and at termination visit were collected. Changes in scores at 6 months and termination were also calculated.
Time frame: 12 months ± 14 days
Population: Health-Related Quality of Life (HRQoL) Intent-to-Treat Analysis Dataset - comprised of all participants \<16 years old, had any available assessments at any available study visits (baseline, 6-month, and 12-month) as defined in the protocol
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Treatment- Screening (N=4, 5) | 27.3 Scores on a scale | Standard Deviation 15.38 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | PH- Change (Month 6 - Termination)(N=4, 5) | 8.0 Scores on a scale | Standard Deviation 12.16 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Treatment- Month 6 (N=4, 5) | 30.6 Scores on a scale | Standard Deviation 16.5 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Future- Screening (N=2, 2) | 40.6 Scores on a scale | Standard Deviation 13.26 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Treatment- Termination (N=4, 5) | 35.9 Scores on a scale | Standard Deviation 26.1 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | R'ships- Screening (N=2, 2) | 6.3 Scores on a scale | Standard Deviation 8.84 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Treatment- Change (Screening - Month 6) (N=4, 5) | -3.2 Scores on a scale | Standard Deviation 4.43 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Others- Termination (N=4, 5) | 18.8 Scores on a scale | Standard Deviation 21.11 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Treatment- Change(Screening -Termination)(N=4, 5) | -8.6 Scores on a scale | Standard Deviation 13.86 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | R'ships- Month 6 (N=2, 2) | 18.8 Scores on a scale | Standard Deviation 26.52 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Treatment- Change (Month 6- Termination)(N=4, 5) | -5.3 Scores on a scale | Standard Deviation 11.25 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Future- Change (Screening - Month 6) (N=2, 2) | -3.1 Scores on a scale | Standard Deviation 4.42 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | View-Screening (N=4, 5) | 46.0 Scores on a scale | Standard Deviation 19.33 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | R'ships- Termination (N=2, 2) | 6.3 Scores on a scale | Standard Deviation 8.84 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | View-Month 6 (N=4, 5) | 39.3 Scores on a scale | Standard Deviation 33.02 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Others- Change (Screening - Month 6) (N=4, 5) | 9.4 Scores on a scale | Standard Deviation 8.59 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | View- Termination (N=4, 5) | 32.5 Scores on a scale | Standard Deviation 32.75 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | R'ships- Change (Screening - Month 6) (N=2, 2) | -12.5 Scores on a scale | Standard Deviation 17.68 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | View- Change (Screening - Month 6) (N=4, 5) | 6.7 Scores on a scale | Standard Deviation 14.96 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Friends- Change (Month 6 - Termination)(N=4, 5) | -4.7 Scores on a scale | Standard Deviation 17.95 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | View- Change (Screening -Termination)(N=4, 5) | 13.5 Scores on a scale | Standard Deviation 16.56 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | R'ships-Change(Screening -Termination)(N=2, 2) | 0.0 Scores on a scale | Standard Deviation 0 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | View- Change (Month 6- Termination)(N=4, 5) | 6.8 Scores on a scale | Standard Deviation 8.67 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Others- Change(Screening - Termination)(N=4, 5) | 24.0 Scores on a scale | Standard Deviation 14.18 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Total-Screening (N=4, 5) | 44.9 Scores on a scale | Standard Deviation 12.28 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | R'ships- Change (Month 6- Termination)(N=2, 2) | 12.5 Scores on a scale | Standard Deviation 17.68 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Total-Month 6 (N=4, 5) | 37.4 Scores on a scale | Standard Deviation 17.91 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Future- Change(Screening - Termination)(N=2, 2) | -6.3 Scores on a scale | Standard Deviation 8.84 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Total- Termination (N=4, 5) | 32.2 Scores on a scale | Standard Deviation 17.96 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | S&S- Screening (N=4, 5) | 53.3 Scores on a scale | Standard Deviation 14.74 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Total- Change (Screening - Month 6) (N=4, 5) | 7.5 Scores on a scale | Standard Deviation 7.21 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Others- Change (Month 6 - Termination)(N=4, 5) | 14.6 Scores on a scale | Standard Deviation 19.39 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Total- Change (Screening -Termination)(N=4, 5) | 12.7 Scores on a scale | Standard Deviation 8.69 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | S&S- Month 6 (N=4, 5) | 46.8 Scores on a scale | Standard Deviation 13.42 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Total- Change (Month 6- Termination)(N=4, 5) | 5.2 Scores on a scale | Standard Deviation 3.55 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Future- Month 6 (N=2, 2) | 43.8 Scores on a scale | Standard Deviation 17.68 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Dealing- Screening (N=4, 4) | 33.0 Scores on a scale | Standard Deviation 17.1 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | S&S- Termination (N=4, 5) | 33.2 Scores on a scale | Standard Deviation 5.64 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Dealing- Month 6 (N=4, 4) | 21.4 Scores on a scale | Standard Deviation 2.92 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | PH- Screening (N=4, 5) | 67.0 Scores on a scale | Standard Deviation 24.98 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Dealing- Termination (N=4, 5) | 25.0 Scores on a scale | Standard Deviation 9.22 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | S&S- Change (Screening - Month 6) (N=4, 5) | 6.5 Scores on a scale | Standard Deviation 11.42 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Dealing- Change (Screening - Month 6) (N=4, 4) | 11.6 Scores on a scale | Standard Deviation 15.26 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Future- Change (Month 6 - Termination)(N=2, 2) | -3.1 Scores on a scale | Standard Deviation 4.42 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Dealing- Change(Screening - Termination)(N=4, 4) | 8.0 Scores on a scale | Standard Deviation 15.26 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | S&S- Change (Screening -Termination)(N=4, 5) | 20.1 Scores on a scale | Standard Deviation 14.74 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Dealing- Change (Month 6 - Termination)(N=4, 4) | -3.6 Scores on a scale | Standard Deviation 6.52 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | PH- Month 6 (N=4, 5) | 49.1 Scores on a scale | Standard Deviation 14.69 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Family- Screening (N=4, 5) | 50.5 Scores on a scale | Standard Deviation 23.94 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | S&S- Change (Month 6- Termination)(N=4, 5) | 13.5 Scores on a scale | Standard Deviation 9.1 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Family- Month 6 (N=4, 5) | 43.4 Scores on a scale | Standard Deviation 28.78 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Friends- Change(Screening - Termination)(N=4, 5) | 1.6 Scores on a scale | Standard Deviation 28.58 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Family- Termination (N=4, 5) | 38.3 Scores on a scale | Standard Deviation 21.25 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Support-Screening (N=4, 4) | 32.8 Scores on a scale | Standard Deviation 3.13 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Family- Change (Screening - Month 6) (N=4, 5) | 7.0 Scores on a scale | Standard Deviation 20.31 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | PH- Termination (N=4, 5) | 41.1 Scores on a scale | Standard Deviation 23.05 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Family- Change(Screening - Termination)(N=4, 5) | 12.2 Scores on a scale | Standard Deviation 25.72 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Support-Month 6 (N=4, 4) | 28.1 Scores on a scale | Standard Deviation 10.83 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Family- Change (Month 6 - Termination)(N=4, 5) | 5.2 Scores on a scale | Standard Deviation 13.66 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Others- Screening (N=4, 5) | 42.7 Scores on a scale | Standard Deviation 33.74 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Feeling- Screening (N=4, 5) | 54.8 Scores on a scale | Standard Deviation 31.91 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Support- Termination (N=4, 5) | 17.2 Scores on a scale | Standard Deviation 17.21 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Feeling- Month 6 (N=4, 5) | 41.4 Scores on a scale | Standard Deviation 43.03 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | PH- Change (Screening - Month 6) (N=4, 5) | 17.9 Scores on a scale | Standard Deviation 19.56 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Feeling- Termination (N=4, 5) | 34.4 Scores on a scale | Standard Deviation 39.48 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Support- Change (Screening - Month 6) (N=4, 4) | 4.7 Scores on a scale | Standard Deviation 12.88 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Feeling- Change (Screening - Month 6) (N=4, 5) | 13.4 Scores on a scale | Standard Deviation 16.91 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Future- Termination (N=2, 2) | 46.9 Scores on a scale | Standard Deviation 22.1 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Feeling- Change(Screening - Termination)(N=4, 5) | 20.4 Scores on a scale | Standard Deviation 11.77 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Support- Change (Screening -Termination)(N=4, 4 | 15.6 Scores on a scale | Standard Deviation 18.75 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Feeling- Change (Month 6 - Termination)(N=4, 5) | 7.0 Scores on a scale | Standard Deviation 7.79 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | PH- Change(Screening - Termination)(N=4, 5) | 25.9 Scores on a scale | Standard Deviation 14.4 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Friends- Screening (N=4, 5) | 34.4 Scores on a scale | Standard Deviation 25.26 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Support- Change (Month 6- Termination)(N=4, 4) | 10.9 Scores on a scale | Standard Deviation 10.67 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Friends- Month 6 (N=4, 5) | 28.1 Scores on a scale | Standard Deviation 15.73 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Others- Month 6 (N=4, 5) | 33.3 Scores on a scale | Standard Deviation 40.11 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Friends- Termination (N=4, 5) | 32.8 Scores on a scale | Standard Deviation 12.88 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Friends- Change (Screening - Month 6) (N=4, 5) | 6.3 Scores on a scale | Standard Deviation 25.52 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Friends- Termination (N=4, 5) | 56.3 Scores on a scale | Standard Deviation 27.24 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Friends- Change (Screening - Month 6) (N=4, 5) | 16.3 Scores on a scale | Standard Deviation 24.84 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Friends- Change(Screening - Termination)(N=4, 5) | 2.5 Scores on a scale | Standard Deviation 22.79 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Friends- Change (Month 6 - Termination)(N=4, 5) | -13.8 Scores on a scale | Standard Deviation 20.44 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Future- Screening (N=2, 2) | 43.8 Scores on a scale | Standard Deviation 17.68 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Future- Month 6 (N=2, 2) | 43.8 Scores on a scale | Standard Deviation 17.68 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Future- Termination (N=2, 2) | 28.1 Scores on a scale | Standard Deviation 22.1 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Future- Change (Screening - Month 6) (N=2, 2) | 0.0 Scores on a scale | Standard Deviation 0 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Future- Change(Screening - Termination)(N=2, 2) | 15.6 Scores on a scale | Standard Deviation 4.42 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Future- Change (Month 6 - Termination)(N=2, 2) | 15.6 Scores on a scale | Standard Deviation 4.42 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Others- Screening (N=4, 5) | 26.7 Scores on a scale | Standard Deviation 20.11 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Others- Month 6 (N=4, 5) | 16.7 Scores on a scale | Standard Deviation 15.02 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Others- Termination (N=4, 5) | 13.3 Scores on a scale | Standard Deviation 12.64 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Others- Change (Screening - Month 6) (N=4, 5) | 10.0 Scores on a scale | Standard Deviation 25.79 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Others- Change(Screening - Termination)(N=4, 5) | 13.3 Scores on a scale | Standard Deviation 23.09 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Others- Change (Month 6 - Termination)(N=4, 5) | 3.3 Scores on a scale | Standard Deviation 16.24 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | PH- Screening (N=4, 5) | 42.1 Scores on a scale | Standard Deviation 32.28 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | PH- Month 6 (N=4, 5) | 33.9 Scores on a scale | Standard Deviation 16.66 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | PH- Termination (N=4, 5) | 25.7 Scores on a scale | Standard Deviation 10.83 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | PH- Change (Screening - Month 6) (N=4, 5) | 8.2 Scores on a scale | Standard Deviation 34.43 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | PH- Change(Screening - Termination)(N=4, 5) | 16.4 Scores on a scale | Standard Deviation 29.73 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | PH- Change (Month 6 - Termination)(N=4, 5) | 8.2 Scores on a scale | Standard Deviation 20.66 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | R'ships- Screening (N=2, 2) | 43.8 Scores on a scale | Standard Deviation 8.84 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | R'ships- Month 6 (N=2, 2) | 43.8 Scores on a scale | Standard Deviation 8.84 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | R'ships- Termination (N=2, 2) | 18.8 Scores on a scale | Standard Deviation 26.52 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | R'ships- Change (Screening - Month 6) (N=2, 2) | 0.0 Scores on a scale | Standard Deviation 0 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | R'ships-Change(Screening -Termination)(N=2, 2) | 25.0 Scores on a scale | Standard Deviation 17.68 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | R'ships- Change (Month 6- Termination)(N=2, 2) | 25.0 Scores on a scale | Standard Deviation 17.68 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | S&S- Screening (N=4, 5) | 51.3 Scores on a scale | Standard Deviation 19.82 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | S&S- Month 6 (N=4, 5) | 54.2 Scores on a scale | Standard Deviation 5.53 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | S&S- Termination (N=4, 5) | 49.8 Scores on a scale | Standard Deviation 18.49 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | S&S- Change (Screening - Month 6) (N=4, 5) | -2.8 Scores on a scale | Standard Deviation 18.53 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | S&S- Change (Screening -Termination)(N=4, 5) | 1.5 Scores on a scale | Standard Deviation 18.74 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | S&S- Change (Month 6- Termination)(N=4, 5) | 4.4 Scores on a scale | Standard Deviation 15.19 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Support-Screening (N=4, 4) | 62.5 Scores on a scale | Standard Deviation 31.04 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Support-Month 6 (N=4, 4) | 56.3 Scores on a scale | Standard Deviation 31.87 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Support- Termination (N=4, 5) | 65.0 Scores on a scale | Standard Deviation 15.69 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Support- Change (Screening - Month 6) (N=4, 4) | 6.3 Scores on a scale | Standard Deviation 31.46 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Support- Change (Screening -Termination)(N=4, 4 | -3.1 Scores on a scale | Standard Deviation 18.75 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Support- Change (Month 6- Termination)(N=4, 4) | -9.4 Scores on a scale | Standard Deviation 25.77 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Treatment- Screening (N=4, 5) | 69.6 Scores on a scale | Standard Deviation 25.63 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Treatment- Month 6 (N=4, 5) | 56.8 Scores on a scale | Standard Deviation 15.89 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Treatment- Termination (N=4, 5) | 44.2 Scores on a scale | Standard Deviation 27.1 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Treatment- Change (Screening - Month 6) (N=4, 5) | 12.9 Scores on a scale | Standard Deviation 21.22 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Treatment- Change(Screening -Termination)(N=4, 5) | 25.4 Scores on a scale | Standard Deviation 28 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Treatment- Change (Month 6- Termination)(N=4, 5) | 12.6 Scores on a scale | Standard Deviation 15.53 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | View-Screening (N=4, 5) | 31.9 Scores on a scale | Standard Deviation 14.36 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | View-Month 6 (N=4, 5) | 32.4 Scores on a scale | Standard Deviation 19.41 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | View- Termination (N=4, 5) | 32.2 Scores on a scale | Standard Deviation 8.6 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | View- Change (Screening - Month 6) (N=4, 5) | -0.5 Scores on a scale | Standard Deviation 18.94 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | View- Change (Screening -Termination)(N=4, 5) | -0.3 Scores on a scale | Standard Deviation 10.77 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | View- Change (Month 6- Termination)(N=4, 5) | 0.2 Scores on a scale | Standard Deviation 13.48 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Total-Screening (N=4, 5) | 45.1 Scores on a scale | Standard Deviation 9.16 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Total-Month 6 (N=4, 5) | 37.7 Scores on a scale | Standard Deviation 9.87 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Total- Termination (N=4, 5) | 35.3 Scores on a scale | Standard Deviation 9.06 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Total- Change (Screening - Month 6) (N=4, 5) | 7.4 Scores on a scale | Standard Deviation 16.57 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Total- Change (Screening -Termination)(N=4, 5) | 9.8 Scores on a scale | Standard Deviation 12.35 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Total- Change (Month 6- Termination)(N=4, 5) | 2.5 Scores on a scale | Standard Deviation 9.46 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Dealing- Screening (N=4, 4) | 44.6 Scores on a scale | Standard Deviation 20.72 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Dealing- Month 6 (N=4, 4) | 37.5 Scores on a scale | Standard Deviation 13.2 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Dealing- Termination (N=4, 5) | 32.9 Scores on a scale | Standard Deviation 12.73 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Dealing- Change (Screening - Month 6) (N=4, 4) | 7.1 Scores on a scale | Standard Deviation 8.75 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Dealing- Change(Screening - Termination)(N=4, 4) | 14.3 Scores on a scale | Standard Deviation 12.37 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Dealing- Change (Month 6 - Termination)(N=4, 4) | 7.1 Scores on a scale | Standard Deviation 6.52 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Family- Screening (N=4, 5) | 35.6 Scores on a scale | Standard Deviation 22.05 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Family- Month 6 (N=4, 5) | 36.4 Scores on a scale | Standard Deviation 23.06 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Family- Termination (N=4, 5) | 32.5 Scores on a scale | Standard Deviation 21.09 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Family- Change (Screening - Month 6) (N=4, 5) | -0.7 Scores on a scale | Standard Deviation 16.38 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Family- Change(Screening - Termination)(N=4, 5) | 3.1 Scores on a scale | Standard Deviation 15.51 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Family- Change (Month 6 - Termination)(N=4, 5) | 3.9 Scores on a scale | Standard Deviation 12.2 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Feeling- Screening (N=4, 5) | 34.9 Scores on a scale | Standard Deviation 12.89 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Feeling- Month 6 (N=4, 5) | 22.0 Scores on a scale | Standard Deviation 18.68 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Feeling- Termination (N=4, 5) | 19.2 Scores on a scale | Standard Deviation 15.39 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Feeling- Change (Screening - Month 6) (N=4, 5) | 12.9 Scores on a scale | Standard Deviation 27.77 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Feeling- Change(Screening - Termination)(N=4, 5) | 15.7 Scores on a scale | Standard Deviation 23.2 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Feeling- Change (Month 6 - Termination)(N=4, 5) | 2.8 Scores on a scale | Standard Deviation 15.26 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Friends- Screening (N=4, 5) | 58.8 Scores on a scale | Standard Deviation 20.54 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation | Friends- Month 6 (N=4, 5) | 42.5 Scores on a scale | Standard Deviation 29.78 |
Secondary
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
The Haemo-QoL is a quality of life (QoL) assessment instrument for children and adolescents with haemophilia. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. The areas covered by this instrument are: Physical Health (PH), Sports & School (S&S), Dealing with Hemophilia (Dealing), Family, Feeling, Relationships (R'ships), Treatment, View, Outlook for the Future (Future), Friends, Others, and Support. A Haemo-QoL Total Score (Total) was also calculated. For the Haemo-QoL, higher scores indicate a worse quality of life. Scores on a scale range between 0 and 100. Haemo-QoL scores at screening, 6 months, and at termination visit were collected. Changes in scores at 6 months and termination were also calculated.
Time frame: 12 months ± 14 days
Population: Health-Related Quality of Life (HRQoL) Intent-to-Treat Analysis Dataset - for all participants \<16 years old who were randomized, had any available assessments at any available study visits (baseline, 6-month, and 12-month) as defined in the protocol
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Friends- Month 6 (N=4, 5) | 26.6 Scores on a scale | Standard Deviation 17.95 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | PH- Change (Screening - Month 6) (N=4, 5) | 4.5 Scores on a scale | Standard Deviation 12.16 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Dealing- Termination (N=4, 5) | 21.4 Scores on a scale | Standard Deviation 16.24 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | PH- Change(Screening - Termination)(N=4, 5) | 17.0 Scores on a scale | Standard Deviation 17.1 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Friends- Termination (N=4, 5) | 34.4 Scores on a scale | Standard Deviation 14.88 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | PH- Change (Month 6 - Termination)(N=4, 5) | 12.5 Scores on a scale | Standard Deviation 22.87 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Family- Change(Screening - Termination)(N=4, 5) | 9.5 Scores on a scale | Standard Deviation 15.52 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | R'ships- Screening (N=2, 2) | 12.5 Scores on a scale | Standard Deviation 17.68 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Friends- Change (Screening - Month 6) (N=4, 5) | 9.4 Scores on a scale | Standard Deviation 27.72 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | R'ships- Month 6 (N=2, 2) | 25.0 Scores on a scale | Standard Deviation 35.36 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Dealing- Change (Month 6 - Termination)(N=4, 4) | 4.2 Scores on a scale | Standard Deviation 11.85 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | R'ships- Termination (N=2, 2) | 0.0 Scores on a scale | Standard Deviation 0 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Friends- Change(Screening - Termination)(N=4, 5) | 1.6 Scores on a scale | Standard Deviation 17.95 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | R'ships- Change (Screening - Month 6) (N=2, 2) | -12.5 Scores on a scale | Standard Deviation 17.68 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Family- Change (Month 6 - Termination)(N=4, 5) | 4.5 Scores on a scale | Standard Deviation 12.16 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | R'ships-Change(Screening -Termination)(N=2, 2) | 12.5 Scores on a scale | Standard Deviation 17.68 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Friends- Change (Month 6 - Termination)(N=4, 5) | -7.8 Scores on a scale | Standard Deviation 22.46 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | R'ships- Change (Month 6- Termination)(N=2, 2) | 25.0 Scores on a scale | Standard Deviation 35.36 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Dealing- Month 6 (N=4, 4) | 25.6 Scores on a scale | Standard Deviation 19.31 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | S&S- Screening (N=4, 5) | 67.1 Scores on a scale | Standard Deviation 10.68 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | S&S- Month 6 (N=4, 5) | 62.5 Scores on a scale | Standard Deviation 7.74 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Future- Screening (N=2, 2) | 53.1 Scores on a scale | Standard Deviation 13.26 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | S&S- Termination (N=4, 5) | 55.6 Scores on a scale | Standard Deviation 17.16 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Feeling- Screening (N=4, 5) | 44.1 Scores on a scale | Standard Deviation 14.25 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | S&S- Change (Screening - Month 6) (N=4, 5) | 4.6 Scores on a scale | Standard Deviation 6.89 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Future- Month 6 (N=2, 2) | 40.6 Scores on a scale | Standard Deviation 13.26 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | S&S- Change (Screening -Termination)(N=4, 5) | 11.5 Scores on a scale | Standard Deviation 12.02 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Family- Screening (N=4, 5) | 55.3 Scores on a scale | Standard Deviation 19.05 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | S&S- Change (Month 6- Termination)(N=4, 5) | 6.9 Scores on a scale | Standard Deviation 18.07 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Future- Termination (N=2, 2) | 31.3 Scores on a scale | Standard Deviation 0 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Support-Screening (N=4, 4) | 28.1 Scores on a scale | Standard Deviation 25.77 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Feeling- Month 6 (N=4, 5) | 50.3 Scores on a scale | Standard Deviation 27.02 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Support-Month 6 (N=4, 4) | 18.8 Scores on a scale | Standard Deviation 29.32 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Future- Change (Screening - Month 6) (N=2, 2) | 12.5 Scores on a scale | Standard Deviation 0 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Support- Termination (N=4, 5) | 32.8 Scores on a scale | Standard Deviation 32.02 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Dealing- Change (Screening - Month 6) (N=4, 4) | 0.3 Scores on a scale | Standard Deviation 8.61 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Support- Change (Screening - Month 6) (N=4, 4) | 9.4 Scores on a scale | Standard Deviation 24.21 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Future- Change(Screening - Termination)(N=2, 2) | 21.9 Scores on a scale | Standard Deviation 13.26 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Support- Change (Screening -Termination)(N=4, 4 | -4.7 Scores on a scale | Standard Deviation 13.86 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Feeling- Termination (N=4, 5) | 33.6 Scores on a scale | Standard Deviation 36.07 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Support- Change (Month 6- Termination)(N=4, 4) | -14.1 Scores on a scale | Standard Deviation 21.27 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Future- Change (Month 6 - Termination)(N=2, 2) | 9.4 Scores on a scale | Standard Deviation 13.26 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Treatment- Screening (N=4, 5) | 29.0 Scores on a scale | Standard Deviation 13.23 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Family- Month 6 (N=4, 5) | 50.3 Scores on a scale | Standard Deviation 27.65 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Treatment- Month 6 (N=4, 5) | 21.9 Scores on a scale | Standard Deviation 6.9 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Others- Screening (N=4, 5) | 27.1 Scores on a scale | Standard Deviation 34.44 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Treatment- Termination (N=4, 5) | 17.1 Scores on a scale | Standard Deviation 22.12 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Feeling- Change (Screening - Month 6) (N=4, 5) | -6.3 Scores on a scale | Standard Deviation 24.27 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Treatment- Change (Screening - Month 6) (N=4, 5) | 7.1 Scores on a scale | Standard Deviation 10.1 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Others- Month 6 (N=4, 5) | 42.7 Scores on a scale | Standard Deviation 39.14 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Treatment- Change(Screening -Termination)(N=4, 5) | 11.9 Scores on a scale | Standard Deviation 17.16 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Dealing- Screening (N=4, 4) | 25.9 Scores on a scale | Standard Deviation 11.8 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Treatment- Change (Month 6- Termination)(N=4, 5) | 4.8 Scores on a scale | Standard Deviation 22.47 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Others- Termination (N=4, 5) | 28.1 Scores on a scale | Standard Deviation 32.87 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | View-Screening (N=4, 5) | 47.4 Scores on a scale | Standard Deviation 18.25 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Feeling- Change(Screening - Termination)(N=4, 5) | 10.5 Scores on a scale | Standard Deviation 27.92 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | View-Month 6 (N=4, 5) | 46.3 Scores on a scale | Standard Deviation 24.45 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Others- Change (Screening - Month 6) (N=4, 5) | -15.6 Scores on a scale | Standard Deviation 16.8 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | View- Termination (N=4, 5) | 32.8 Scores on a scale | Standard Deviation 35.68 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Family- Termination (N=4, 5) | 45.8 Scores on a scale | Standard Deviation 31.03 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | View- Change (Screening - Month 6) (N=4, 5) | 1.1 Scores on a scale | Standard Deviation 6.38 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Others- Change(Screening - Termination)(N=4, 5) | -1.0 Scores on a scale | Standard Deviation 15.36 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | View- Change (Screening -Termination)(N=4, 5) | 14.7 Scores on a scale | Standard Deviation 18.62 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Feeling- Change (Month 6 - Termination)(N=4, 5) | 16.7 Scores on a scale | Standard Deviation 13.85 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | View- Change (Month 6- Termination)(N=4, 5) | 13.5 Scores on a scale | Standard Deviation 13.02 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Others- Change (Month 6 - Termination)(N=4, 5) | 14.6 Scores on a scale | Standard Deviation 19.98 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Total-Screening (N=4, 5) | 43.7 Scores on a scale | Standard Deviation 13.67 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Dealing- Change(Screening - Termination)(N=4, 4) | 4.5 Scores on a scale | Standard Deviation 7.92 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Total-Month 6 (N=4, 5) | 42.0 Scores on a scale | Standard Deviation 13.84 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | PH- Screening (N=4, 5) | 58.0 Scores on a scale | Standard Deviation 11.06 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Total- Termination (N=4, 5) | 34.5 Scores on a scale | Standard Deviation 21.83 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Friends- Screening (N=4, 5) | 35.9 Scores on a scale | Standard Deviation 20.65 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Total- Change (Screening - Month 6) (N=4, 5) | 1.7 Scores on a scale | Standard Deviation 1.19 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | PH- Month 6 (N=4, 5) | 53.6 Scores on a scale | Standard Deviation 13.98 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Total- Change (Screening -Termination)(N=4, 5) | 9.1 Scores on a scale | Standard Deviation 8.57 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Family- Change (Screening - Month 6) (N=4, 5) | 5.0 Scores on a scale | Standard Deviation 9.26 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Total- Change (Month 6- Termination)(N=4, 5) | 7.4 Scores on a scale | Standard Deviation 8.91 |
| On-demand Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | PH- Termination (N=4, 5) | 41.1 Scores on a scale | Standard Deviation 11.85 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Total- Change (Month 6- Termination)(N=4, 5) | 2.8 Scores on a scale | Standard Deviation 7.06 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Dealing- Screening (N=4, 4) | 39.3 Scores on a scale | Standard Deviation 17 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Dealing- Month 6 (N=4, 4) | 36.6 Scores on a scale | Standard Deviation 16.07 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Dealing- Termination (N=4, 5) | 28.6 Scores on a scale | Standard Deviation 13.36 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Dealing- Change (Screening - Month 6) (N=4, 4) | 2.7 Scores on a scale | Standard Deviation 7.92 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Dealing- Change(Screening - Termination)(N=4, 4) | 13.4 Scores on a scale | Standard Deviation 5.36 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Dealing- Change (Month 6 - Termination)(N=4, 4) | 10.7 Scores on a scale | Standard Deviation 7.72 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Family- Screening (N=4, 5) | 50.9 Scores on a scale | Standard Deviation 8.72 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Family- Month 6 (N=4, 5) | 49.1 Scores on a scale | Standard Deviation 10.27 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Family- Termination (N=4, 5) | 39.8 Scores on a scale | Standard Deviation 13.77 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Family- Change (Screening - Month 6) (N=4, 5) | 1.7 Scores on a scale | Standard Deviation 15.65 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Family- Change(Screening - Termination)(N=4, 5) | 11.1 Scores on a scale | Standard Deviation 19.22 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Family- Change (Month 6 - Termination)(N=4, 5) | 9.3 Scores on a scale | Standard Deviation 5.24 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Feeling- Screening (N=4, 5) | 38.2 Scores on a scale | Standard Deviation 2.77 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Feeling- Month 6 (N=4, 5) | 37.5 Scores on a scale | Standard Deviation 22.21 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Feeling- Termination (N=4, 5) | 27.8 Scores on a scale | Standard Deviation 16.97 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Feeling- Change (Screening - Month 6) (N=4, 5) | 0.7 Scores on a scale | Standard Deviation 24.02 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Feeling- Change(Screening - Termination)(N=4, 5) | 10.5 Scores on a scale | Standard Deviation 19.45 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Feeling- Change (Month 6 - Termination)(N=4, 5) | 9.7 Scores on a scale | Standard Deviation 20.64 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Friends- Screening (N=4, 5) | 52.1 Scores on a scale | Standard Deviation 17.18 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Friends- Month 6 (N=4, 5) | 36.3 Scores on a scale | Standard Deviation 29.78 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Friends- Termination (N=4, 5) | 66.3 Scores on a scale | Standard Deviation 20.06 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Friends- Change (Screening - Month 6) (N=4, 5) | 15.8 Scores on a scale | Standard Deviation 21.88 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Friends- Change(Screening - Termination)(N=4, 5) | -14.2 Scores on a scale | Standard Deviation 28.31 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Friends- Change (Month 6 - Termination)(N=4, 5) | -30.0 Scores on a scale | Standard Deviation 31.68 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Future- Screening (N=2, 2) | 43.8 Scores on a scale | Standard Deviation 17.68 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Future- Month 6 (N=2, 2) | 37.5 Scores on a scale | Standard Deviation 8.84 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Future- Termination (N=2, 2) | 43.8 Scores on a scale | Standard Deviation 8.84 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Future- Change (Screening - Month 6) (N=2, 2) | 6.3 Scores on a scale | Standard Deviation 8.84 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Future- Change(Screening - Termination)(N=2, 2) | 0.0 Scores on a scale | Standard Deviation 8.84 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Future- Change (Month 6 - Termination)(N=2, 2) | -6.3 Scores on a scale | Standard Deviation 0 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Others- Screening (N=4, 5) | 29.2 Scores on a scale | Standard Deviation 16.67 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Others- Month 6 (N=4, 5) | 24.2 Scores on a scale | Standard Deviation 15.7 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Others- Termination (N=4, 5) | 25.8 Scores on a scale | Standard Deviation 13.63 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Others- Change (Screening - Month 6) (N=4, 5) | 5.0 Scores on a scale | Standard Deviation 20.28 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Others- Change(Screening - Termination)(N=4, 5) | 3.3 Scores on a scale | Standard Deviation 15.98 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Others- Change (Month 6 - Termination)(N=4, 5) | -1.7 Scores on a scale | Standard Deviation 10.87 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | PH- Screening (N=4, 5) | 52.1 Scores on a scale | Standard Deviation 23.23 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | PH- Month 6 (N=4, 5) | 48.0 Scores on a scale | Standard Deviation 17.23 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | PH- Termination (N=4, 5) | 40.4 Scores on a scale | Standard Deviation 17.02 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | PH- Change (Screening - Month 6) (N=4, 5) | 4.1 Scores on a scale | Standard Deviation 32.71 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | PH- Change(Screening - Termination)(N=4, 5) | 11.8 Scores on a scale | Standard Deviation 29.6 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | PH- Change (Month 6 - Termination)(N=4, 5) | 7.7 Scores on a scale | Standard Deviation 22.25 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | R'ships- Screening (N=2, 2) | 43.8 Scores on a scale | Standard Deviation 8.84 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | R'ships- Month 6 (N=2, 2) | 43.8 Scores on a scale | Standard Deviation 8.84 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | R'ships- Termination (N=2, 2) | 37.5 Scores on a scale | Standard Deviation 17.68 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | R'ships- Change (Screening - Month 6) (N=2, 2) | 0.0 Scores on a scale | Standard Deviation 0 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | R'ships-Change(Screening -Termination)(N=2, 2) | 6.3 Scores on a scale | Standard Deviation 8.84 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | R'ships- Change (Month 6- Termination)(N=2, 2) | 6.3 Scores on a scale | Standard Deviation 8.84 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | S&S- Month 6 (N=4, 5) | 56.5 Scores on a scale | Standard Deviation 9.07 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | S&S- Termination (N=4, 5) | 57.2 Scores on a scale | Standard Deviation 17.29 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | S&S- Change (Screening - Month 6) (N=4, 5) | 2.6 Scores on a scale | Standard Deviation 16.32 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | S&S- Change (Screening -Termination)(N=4, 5) | 1.9 Scores on a scale | Standard Deviation 10.22 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | S&S- Change (Month 6- Termination)(N=4, 5) | -0.8 Scores on a scale | Standard Deviation 8.94 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Support-Screening (N=4, 4) | 51.6 Scores on a scale | Standard Deviation 9.38 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Support-Month 6 (N=4, 4) | 42.2 Scores on a scale | Standard Deviation 17.95 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Support- Termination (N=4, 5) | 48.8 Scores on a scale | Standard Deviation 20.44 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Support- Change (Screening - Month 6) (N=4, 4) | 9.4 Scores on a scale | Standard Deviation 10.83 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Support- Change (Screening -Termination)(N=4, 4 | 3.1 Scores on a scale | Standard Deviation 15.73 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Support- Change (Month 6- Termination)(N=4, 4) | -6.3 Scores on a scale | Standard Deviation 8.84 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Treatment- Screening (N=4, 5) | 64.2 Scores on a scale | Standard Deviation 22.6 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Treatment- Month 6 (N=4, 5) | 54.8 Scores on a scale | Standard Deviation 14.53 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Treatment- Termination (N=4, 5) | 41.0 Scores on a scale | Standard Deviation 13.34 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Treatment- Change (Screening - Month 6) (N=4, 5) | 9.4 Scores on a scale | Standard Deviation 13.06 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Treatment- Change(Screening -Termination)(N=4, 5) | 23.2 Scores on a scale | Standard Deviation 16.94 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Treatment- Change (Month 6- Termination)(N=4, 5) | 13.8 Scores on a scale | Standard Deviation 11.14 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | View-Screening (N=4, 5) | 36.7 Scores on a scale | Standard Deviation 16.94 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | View-Month 6 (N=4, 5) | 36.4 Scores on a scale | Standard Deviation 22.04 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | View- Termination (N=4, 5) | 30.8 Scores on a scale | Standard Deviation 12.85 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | View- Change (Screening - Month 6) (N=4, 5) | 0.3 Scores on a scale | Standard Deviation 7.63 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | View- Change (Screening -Termination)(N=4, 5) | 5.9 Scores on a scale | Standard Deviation 10.08 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | View- Change (Month 6- Termination)(N=4, 5) | 5.6 Scores on a scale | Standard Deviation 16.7 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Total-Screening (N=4, 5) | 48.1 Scores on a scale | Standard Deviation 8.84 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Total-Month 6 (N=4, 5) | 42.3 Scores on a scale | Standard Deviation 11.08 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Total- Termination (N=4, 5) | 39.5 Scores on a scale | Standard Deviation 8.52 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Total- Change (Screening - Month 6) (N=4, 5) | 5.7 Scores on a scale | Standard Deviation 14.97 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | Total- Change (Screening -Termination)(N=4, 5) | 8.6 Scores on a scale | Standard Deviation 8.72 |
| Prophylaxis Arm | Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation | S&S- Screening (N=4, 5) | 59.1 Scores on a scale | Standard Deviation 23.73 |
Secondary
Number of New Target Joints
Target Joints are defined as ≥4 bleeds/6 months in any one of the following joints: ankles, knees, elbows and hips
Time frame: 12 months ± 14 days
Population: Efficacy Intent to Treat Analysis Dataset: Dataset consists of data from all randomized participants. Dataset includes those who discontinued FEIBA NF but did not withdraw informed consent and were willing to continue providing data. For these participants, only the data collected before FEIBA NF discontinuation is included.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| On-demand Arm | Number of New Target Joints | 23 new target joints |
| Prophylaxis Arm | Number of New Target Joints | 7 new target joints |
Secondary
Number of Related Thromboembolic Adverse Events (AEs)
Time frame: 12 months ± 14 days
Population: Safety Analysis Set
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| On-demand Arm | Number of Related Thromboembolic Adverse Events (AEs) | 0 Related thromboembolic AEs |
| Prophylaxis Arm | Number of Related Thromboembolic Adverse Events (AEs) | 0 Related thromboembolic AEs |
Secondary
Pharmacoeconomics: Annual Days Lost Due to Bleeding (Work or School)
Time frame: 12 months ± 14 days
Population: Intent to Treat Analysis Set
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| On-demand Arm | Pharmacoeconomics: Annual Days Lost Due to Bleeding (Work or School) | 16.4 days | Standard Deviation 25.76 |
| Prophylaxis Arm | Pharmacoeconomics: Annual Days Lost Due to Bleeding (Work or School) | 8.8 days | Standard Deviation 14.42 |
Secondary
Pharmacoeconomics: Annual Number of Emergency Room Visits
Time frame: 12 months ± 14 days
Population: Pharmacoeconomic Analysis Set
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| On-demand Arm | Pharmacoeconomics: Annual Number of Emergency Room Visits | 0.4 Emergency room visits | Standard Deviation 0.98 |
| Prophylaxis Arm | Pharmacoeconomics: Annual Number of Emergency Room Visits | 0.4 Emergency room visits | Standard Deviation 1.09 |
Secondary
Pharmacoeconomics: Annual Number of Hospitalizations for Bleeding
Time frame: 12 months ± 14 days
Population: Pharmacoeconomic Analysis Set
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| On-demand Arm | Pharmacoeconomics: Annual Number of Hospitalizations for Bleeding | 0.6 hospitalizations | Standard Deviation 1.15 |
| Prophylaxis Arm | Pharmacoeconomics: Annual Number of Hospitalizations for Bleeding | 0.6 hospitalizations | Standard Deviation 2.03 |
Secondary
Pharmacoeconomics: Annual Number of Hospitalizations for Indwelling Line
Time frame: 12 months ± 14 days
Population: Pharmacoeconomic Analysis Set
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| On-demand Arm | Pharmacoeconomics: Annual Number of Hospitalizations for Indwelling Line | 0.1 hospitalizations | Standard Deviation 0.32 |
| Prophylaxis Arm | Pharmacoeconomics: Annual Number of Hospitalizations for Indwelling Line | 0.1 hospitalizations | Standard Deviation 0.25 |
Secondary
Pharmacoeconomics: Annual Number of Physician's Office Visits
Time frame: 12 months ± 14 days
Population: Pharmacoeconomic Analysis Set
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| On-demand Arm | Pharmacoeconomics: Annual Number of Physician's Office Visits | 2.2 Physician's office visits | Standard Deviation 4.15 |
| Prophylaxis Arm | Pharmacoeconomics: Annual Number of Physician's Office Visits | 2.6 Physician's office visits | Standard Deviation 4.6 |
Secondary
Pharmacoeconomics: Annual Total Length of Hospitalization for Bleeding
Time frame: 12 months ± 14 days
Population: Pharmacoeconomic Analysis Set
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| On-demand Arm | Pharmacoeconomics: Annual Total Length of Hospitalization for Bleeding | 5.0 Days | Standard Deviation 9.48 |
| Prophylaxis Arm | Pharmacoeconomics: Annual Total Length of Hospitalization for Bleeding | 5.3 Days | Standard Deviation 17 |
Secondary
Pharmacoeconomics: Annual Total Length of Hospitalization for Indwelling Line
Time frame: 12 months ± 14 days
Population: Pharmacoeconomic Analysis Set
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| On-demand Arm | Pharmacoeconomics: Annual Total Length of Hospitalization for Indwelling Line | 0.7 Days | Standard Deviation 2.61 |
| Prophylaxis Arm | Pharmacoeconomics: Annual Total Length of Hospitalization for Indwelling Line | 0.7 Days | Standard Deviation 2.75 |
Secondary
Pharmacoeconomics: Annual Total Number of Days Lost (Work or School)
Time frame: 12 months ± 14 days
Population: Pharmacoeconomic Analysis Set
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| On-demand Arm | Pharmacoeconomics: Annual Total Number of Days Lost (Work or School) | 17.4 Days | Standard Deviation 25.42 |
| Prophylaxis Arm | Pharmacoeconomics: Annual Total Number of Days Lost (Work or School) | 15.4 Days | Standard Deviation 24.28 |
Secondary
Rate of Related Adverse Events (AEs) During or Within 1 Hour of Infusion Per Year
Time frame: 12 months ± 14 days
Population: Safety Analysis Set
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| On-demand Arm | Rate of Related Adverse Events (AEs) During or Within 1 Hour of Infusion Per Year | 0.000 Related AEs within/during 1hr per year |
| Prophylaxis Arm | Rate of Related Adverse Events (AEs) During or Within 1 Hour of Infusion Per Year | 0.000 Related AEs within/during 1hr per year |
Secondary
Rate of Related Adverse Events (AEs) Per Year
Time frame: 12 months ± 14 days
Population: Safety Analysis Set
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| On-demand Arm | Rate of Related Adverse Events (AEs) Per Year | 0.000 Related AEs per year |
| Prophylaxis Arm | Rate of Related Adverse Events (AEs) Per Year | 0.000 Related AEs per year |
Secondary
The Number of Bleeding Episode (BE) Which Required 1, 2, 3, or ≥4 Infusions to Control Bleeding
Time frame: 12 months ± 14 days
Population: Additional Evaluations for Bleeding Episodes Analysis Set - Consists of all participants with at least 1 bleeding episode treated with investigational product, FEIBA NF.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| On-demand Arm | The Number of Bleeding Episode (BE) Which Required 1, 2, 3, or ≥4 Infusions to Control Bleeding | 1 infusion | 352 Bleeding Episodes (BEs) |
| On-demand Arm | The Number of Bleeding Episode (BE) Which Required 1, 2, 3, or ≥4 Infusions to Control Bleeding | 2 infusions | 134 Bleeding Episodes (BEs) |
| On-demand Arm | The Number of Bleeding Episode (BE) Which Required 1, 2, 3, or ≥4 Infusions to Control Bleeding | 3 infusions | 62 Bleeding Episodes (BEs) |
| On-demand Arm | The Number of Bleeding Episode (BE) Which Required 1, 2, 3, or ≥4 Infusions to Control Bleeding | ≥4 infusions | 75 Bleeding Episodes (BEs) |
| Prophylaxis Arm | The Number of Bleeding Episode (BE) Which Required 1, 2, 3, or ≥4 Infusions to Control Bleeding | ≥4 infusions | 21 Bleeding Episodes (BEs) |
| Prophylaxis Arm | The Number of Bleeding Episode (BE) Which Required 1, 2, 3, or ≥4 Infusions to Control Bleeding | 1 infusion | 98 Bleeding Episodes (BEs) |
| Prophylaxis Arm | The Number of Bleeding Episode (BE) Which Required 1, 2, 3, or ≥4 Infusions to Control Bleeding | 3 infusions | 13 Bleeding Episodes (BEs) |
| Prophylaxis Arm | The Number of Bleeding Episode (BE) Which Required 1, 2, 3, or ≥4 Infusions to Control Bleeding | 2 infusions | 41 Bleeding Episodes (BEs) |
Secondary
Total Weight Adjusted Dose to Control a Bleeding Episode
Time frame: 12 months ± 14 days
Population: Additional Evaluations for Bleeding Episodes Analysis Set - Consists of all participants with at least 1 bleeding episode treated with investigational product, FEIBA NF.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| On-demand Arm | Total Weight Adjusted Dose to Control a Bleeding Episode | 4049.7 Units/kg |
| Prophylaxis Arm | Total Weight Adjusted Dose to Control a Bleeding Episode | 1524.9 Units/kg |
p-value: 0.0067Mann-Whitney tests (Wilcoxon-Rank Sum)
Secondary
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
-Hepatitis A Virus Antibody (HAV Ab) -Hepatitis B Virus Core Antibody (HBcAb) -Hepatitis B Virus Surface Antibody (HBsAb) -Hepatitis B Virus Surface Antigen (HBsAg) -Hepatitis C Virus (HCV)
Time frame: 12 months ± 14 days
Population: Safety Analysis Set
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HAV Ab: Screening Negative; Termination Positive | 1 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HAV Ab: Screening Negative; Termination Negative | 8 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HAV Ab: Screening Positive; Termination Positive | 8 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HAV Ab: Screening Positive; Termination Negative | 1 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HAV Ab: Serology not available | 1 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBcAb: Screening Negative; Termination Positive | 0 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBcAb: Screening Negative; Termination Negative | 8 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBcAb: Screening Positive; Termination Positive | 9 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBcAb: Screening Positive; Termination Negative | 1 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBcAb: Serology not available | 1 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBsAb: Screening Negative; Termination Positive | 2 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBsAb: Screening Negative; Termination Negative | 2 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBsAb: Screening Positive; Termination Positive | 13 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBsAb: Screening Positive; Termination Negative | 1 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBsAb: Serology not available | 1 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBsAg: Screening Negative; Termination Positive | 0 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBsAg: Screening Negative; Termination Negative | 18 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBsAg: Serology not available | 1 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HCV: Screening Negative; Termination Positive | 0 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HCV: Screening Negative; Termination Negative | 6 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HCV: Screening Positive; Termination Positive | 12 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HCV: Screening Positive; Termination Negative | 0 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HCV: Serology not available | 1 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBsAb: Screening Negative; Termination Negative | 2 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HAV Ab: Screening Negative; Termination Positive | 0 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBsAg: Serology not available | 1 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HAV Ab: Screening Negative; Termination Negative | 11 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBsAb: Screening Positive; Termination Positive | 9 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HAV Ab: Screening Positive; Termination Positive | 4 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HCV: Screening Positive; Termination Positive | 6 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HAV Ab: Screening Positive; Termination Negative | 1 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBsAb: Screening Positive; Termination Negative | 0 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HAV Ab: Serology not available | 1 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HCV: Screening Negative; Termination Positive | 0 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBcAb: Screening Negative; Termination Positive | 1 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBsAb: Serology not available | 1 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBcAb: Screening Negative; Termination Negative | 11 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HCV: Serology not available | 1 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBcAb: Screening Positive; Termination Positive | 4 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBsAg: Screening Negative; Termination Positive | 0 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBcAb: Screening Positive; Termination Negative | 0 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HCV: Screening Negative; Termination Negative | 10 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBcAb: Serology not available | 1 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBsAg: Screening Negative; Termination Negative | 16 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HBsAb: Screening Negative; Termination Positive | 5 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C | HCV: Screening Positive; Termination Negative | 0 participants |
Secondary
Viral Serology From Screening Visit and Study Termination Visit: HIV-1/2 Antibody (Ab)
Time frame: 12 months ± 14 days
Population: Safety Analysis Set
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: HIV-1/2 Antibody (Ab) | HIV 1/2 Ab: Screen Negative; Termination Positive | 0 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: HIV-1/2 Antibody (Ab) | HIV 1/2 Ab: Screen Negative; Termination Negative | 18 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: HIV-1/2 Antibody (Ab) | HIV 1/2 Ab: : Serology not available | 1 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: HIV-1/2 Antibody (Ab) | HIV 1/2 Ab: Screen Negative; Termination Positive | 0 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: HIV-1/2 Antibody (Ab) | HIV 1/2 Ab: Screen Negative; Termination Negative | 16 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: HIV-1/2 Antibody (Ab) | HIV 1/2 Ab: : Serology not available | 1 participants |
Secondary
Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgG Antibody [IV]
Normal range (0 - 0.89 IV); High (\> 0.89 IV) - Parvovirus B19 IgG Antibody \[IV\] (Parvo IgG Ab)
Time frame: 12 months ± 14 days
Population: Safety Analysis Set
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgG Antibody [IV] | Parvo IgG Ab: Screening High ; Termination High | 14 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgG Antibody [IV] | Parvo IgG Ab: Screening Normal ; Termination High | 1 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgG Antibody [IV] | Parvo IgG Ab: Screening Normal; Termination Normal | 0 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgG Antibody [IV] | Parvo IgG Ab: Serology not available | 1 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgG Antibody [IV] | Parvo IgG Ab: Screening High ; Termination Normal | 3 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgG Antibody [IV] | Parvo IgG Ab: Serology not available | 1 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgG Antibody [IV] | Parvo IgG Ab: Screening High ; Termination Normal | 1 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgG Antibody [IV] | Parvo IgG Ab: Screening High ; Termination High | 13 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgG Antibody [IV] | Parvo IgG Ab: Screening Normal; Termination Normal | 2 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgG Antibody [IV] | Parvo IgG Ab: Screening Normal ; Termination High | 0 participants |
Secondary
Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgM Antibody [IV]
Normal range (0 - 0.89 IV); High (\> 0.89 IV) - Parvovirus B19 IgM Antibody \[IV\] (Parvo IgM Ab)
Time frame: 12 months ± 14 days
Population: Safety Analysis Set
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgM Antibody [IV] | Parvo IgM Ab: Screening High ; Termination High | 1 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgM Antibody [IV] | Parvo IgM Ab: Screening Normal ; Termination High | 0 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgM Antibody [IV] | Parvo IgM Ab: Screening Normal; Termination Normal | 17 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgM Antibody [IV] | Parvo IgM Ab: Serology not available | 1 participants |
| On-demand Arm | Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgM Antibody [IV] | Parvo IgM Ab: Screening High ; Termination Normal | 0 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgM Antibody [IV] | Parvo IgM Ab: Serology not available | 1 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgM Antibody [IV] | Parvo IgM Ab: Screening High ; Termination Normal | 0 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgM Antibody [IV] | Parvo IgM Ab: Screening High ; Termination High | 0 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgM Antibody [IV] | Parvo IgM Ab: Screening Normal; Termination Normal | 16 participants |
| Prophylaxis Arm | Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgM Antibody [IV] | Parvo IgM Ab: Screening Normal ; Termination High | 0 participants |