Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
Conditions
Brief summary
This study is conducted in Europe and North and South America. The primary aim of this observational study is to evaluate the frequency and pattern of bleeding episodes in haemophilia patients receiving preventative treatment with activated recombinant human factor VII. The secondary aim is to evaluate which patients are selected for this treatment, the dose and dose intervals used, and the safety of activated recombinant human factor VII when used as prevention. The study also aims to increase understanding of the unmet medical need and clinical relevance of preventative treatment in haemophilia patients.
Interventions
DRUGeptacog alfa (activated)
Retrospective data collection of the use of activated recombinant human factor VII as prophylaxis in haemophilia patients with inhibitors
Sponsors
Novo Nordisk A/S
Study design
Observational model
CASE_ONLY
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
MALE
Healthy volunteers
No
Inclusion criteria
* Haemophilia A or B with inhibitors * Prescribed use of activated recombinant human factor VII for any type of prophylaxis with a duration of at least 30 days
Exclusion criteria
* Prophylaxis prescribed post-surgery * One or more coagulation disorders in addition to haemophilia
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percent Change in Total Bleed Episodes Per Month - Bleeding Population | Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Percent change of bleeds per month in the pre-prophylaxis period and bleeds per month in the prophylaxis period |
| Percent Change in Total Bleed Episodes Per Month - Frequent Bleeding Population | Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Percent change of bleeds per month in the pre-prophylaxis period and bleeds per month in the prophylaxis period |
| Percent Change in Total Bleed Episodes Per Month Per Age Categories - Bleeding Population | Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Percent change of bleeds per month between the pre-prophylaxis period and the prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years |
| Percent Change in Total Bleed Episodes Per Month Per Age Categories - Frequent Bleeding Population | Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Percent change of bleeds per month between the pre-prophylaxis period and the prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years |
| Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Infrequent Dosing | Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Infrequent dosing was defined as less than two doses per week. All participants = paediatrics, adolescents and adults. |
| Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Dosing Three Times Per Week | Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Three times per week dosing was defined as dosing two to four times per week. All participants = paediatrics, adolescents and adults. |
| Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Daily Dosing | Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Daily dosing was defined as 5 to 7 doses per week. All participants = paediatrics, adolescents and adults. |
| Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Frequent Dosing | Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Percent change of bleeds per month between the pre-prophylaxis period and the prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Frequent dosing was defined as 7 or more doses per week. All participants = paediatrics, adolescents and adults. |
| Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Infrequent Dosing | Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Infrequent dosing was defined as less than two doses per week. All participants = paediatrics, adolescents and adults. |
| Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Dosing Three Times Per Week | Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Three times per week dosing was defined as dosing two to four times per week. All participants = paediatrics, adolescents and adults. |
| Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Daily Dosing | Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Daily dosing was defined as 5 to 7 doses per week. All participants = paediatrics, adolescents and adults. |
| Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Frequent Dosing | Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Frequent dosing was defined as 7 or more doses per week. All participants = paediatrics, adolescents and adults. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - Bleeding Population | Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Healthcare resource consumption evaluated the absolute change in number of total hospital length of stay and school/work absences during the pre-prophylaxis period to the prophylaxis period. |
| Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - Frequent Bleeding Population | Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Healthcare resource consumption evaluated the absolute change in number of total hospital length of stay and school/work absences during the pre-prophylaxis period to the prophylaxis period. |
| Individual Dose by Dose Regimen and Age Group - Bleeding Population, Paediatric | Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Individual activated recombinant human factor VII dose for paediatric patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week). |
| Number of Physician Reported Outcome Assessment in Prophylaxis in Percentage of Patients | Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Physician's assessment of prophylaxis outcome as successful, partially successful, unsuccessful, or unable to determine |
| Physician Reported Outcome Assessment in Prophylaxis in Number of Patients | Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Physician's assessment of prophylaxis outcome as successful, partially successful, unsuccessful, or unable to determine |
| Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adolescent | Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Individual activated recombinant human factor VII dose for adolescent patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week). |
| Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adult | Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Individual activated recombinant human factor VII dose for adult patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week). |
| Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Paediatric | Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Individual activated recombinant human factor VII dose for paediatric patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week). |
| Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adolescent | Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Individual activated recombinant human factor VII dose for adolescent patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week). |
| Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adult | Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Individual activated recombinant human factor VII dose for adult patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week). |
| Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Bleeding Population | Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Percent change in bleed episodes per month between pre-prophylaxis period and prophylaxis period by location of joint, target joint (defined as 3 or more documented bleeds in the same joint over the course of 6 months) or non-joint. All joints = target joints and non-target joints. |
| Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Frequent Bleeding Population | Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Percent change in bleed episodes per month between pre-prophylaxis period and prophylaxis period by location of joint, target joint (= 3 or more documented bleeds in the same joint over the course of 6 months) or non-joint. All joints = target joints and non-target joints. |
| Healthcare Resource Consumption of Visits, Consultations, and Hospital Admissions Per Month - All Patients | Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Healthcare resource consumption evaluated the absolute change in number of outpatient clinical visits, physician consultations and hospital admissions during the pre-prophylaxis to the prophylaxis period. |
| Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Bleeding Population | Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Healthcare resource consumption evaluated the absolute change in number of outpatient clinical visits, physician consultations and hospital admissions during the pre-prophylaxis period to the prophylaxis period. |
| Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Frequent Bleeding Population | Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Healthcare resource consumption evaluated the absolute change in number of outpatient clinical visits, physician consultations and hospital admissions during the pre-prophylaxis period to the prophylaxis period. |
| Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - All Patients | Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days. | Healthcare resource consumption evaluated the absolute change in number of total hospital length of stay and school/work absences during the pre-prophylaxis period to the prophylaxis period. |
Countries
Argentina, Canada, Croatia, Czechia, France, Germany, Ireland, Italy, Slovakia, Spain, Sweden, Switzerland, United Kingdom, United States
Participant flow
Recruitment details
Participating sites: Argentina (1), Canada (1), Croatia (1), Czech Republic (1), Germany (1), France (1), Ireland (1), Italy (4), Slovakia (1), Spain (3), Sweden (2), Switzerland (2), United Kingdom (1), and the United States of America (11)
Participants by arm
| Arm | Count |
|---|---|
| Activated Recombinant Human Factor VII Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits. | 86 |
| Total | 86 |
Baseline characteristics
| Characteristic | Activated Recombinant Human Factor VII |
|---|---|
| Age, Continuous All, n=86 | 11.6 years STANDARD_DEVIATION 13.03 |
| Age, Continuous Bleeding population, n=74 | 10.9 years STANDARD_DEVIATION 12.45 |
| Age, Continuous Frequent bleeding population, n=36 | 12.1 years STANDARD_DEVIATION 12.67 |
| Age, Customized 18 years or above | 18 participants |
| Age, Customized Below 12 years | 61 participants |
| Age, Customized Between 12 and 17 years | 7 participants |
| Dose frequency All, n=82 | 5.2 doses per week |
| Dose frequency Bleeding population, n=73 | 5.8 doses per week |
| Dose frequency Frequent bleeding population, n=36 | 5.2 doses per week |
| Dose frequency Missing, n=4 | NA doses per week |
| Duration of prophylaxis All, n=86 | 288 days |
| Duration of prophylaxis Bleeding population, n=74 | 293 days |
| Duration of prophylaxis Frequent bleeding population, n=36 | 293 days |
| Ethnicity (NIH/OMB) Hispanic or Latino | 12 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 33 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 41 Participants |
| Gender Female | 0 Participants |
| Gender Male | 86 Participants |
| Peak historical inhibitor titre recorded All, n=81 | 517.7 Bethesda Units (BU)/mL STANDARD_DEVIATION 1164.41 |
| Peak historical inhibitor titre recorded Bleeding population, n=72 | 571 Bethesda Units (BU)/mL STANDARD_DEVIATION 1224.88 |
| Peak historical inhibitor titre recorded Frequent bleeding population, n=35 | 737.9 Bethesda Units (BU)/mL STANDARD_DEVIATION 1457 |
| Peak historical inhibitor titre recorded Missing, n=5 | NA Bethesda Units (BU)/mL |
| Race (NIH/OMB) American Indian or Alaska Native | 1 Participants |
| Race (NIH/OMB) Asian | 1 Participants |
| Race (NIH/OMB) Black or African American | 7 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 1 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 33 Participants |
| Race (NIH/OMB) White | 43 Participants |
| Reason for prophylaxis In preparation for surgery | 0 participants |
| Reason for prophylaxis Missing | 3 participants |
| Reason for prophylaxis Other | 2 participants |
| Reason for prophylaxis Prep. for rehab./planned phys. activity | 2 participants |
| Reason for prophylaxis Prevention of recurrent bleeding | 79 participants |
| Region of Enrollment Argentina | 4 participants |
| Region of Enrollment Canada | 1 participants |
| Region of Enrollment Croatia | 2 participants |
| Region of Enrollment Czech Republic | 1 participants |
| Region of Enrollment France | 17 participants |
| Region of Enrollment Germany | 3 participants |
| Region of Enrollment Ireland | 5 participants |
| Region of Enrollment Italy | 7 participants |
| Region of Enrollment Slovakia | 1 participants |
| Region of Enrollment Spain | 10 participants |
| Region of Enrollment Sweden | 4 participants |
| Region of Enrollment Switzerland | 2 participants |
| Region of Enrollment United Kingdom | 1 participants |
| Region of Enrollment United States | 28 participants |
| Relationship to timing of Immune Tolerance Induction (ITI) During ITI | 8 participants |
| Relationship to timing of Immune Tolerance Induction (ITI) ITI dates unknown | 0 participants |
| Relationship to timing of Immune Tolerance Induction (ITI) Not candidate for ITI | 27 participants |
| Relationship to timing of Immune Tolerance Induction (ITI) Not reported | 7 participants |
| Relationship to timing of Immune Tolerance Induction (ITI) Post-ITI | 25 participants |
| Relationship to timing of Immune Tolerance Induction (ITI) Pre-ITI | 19 participants |
| Weekly dose All, n=82 | 710 mcg/kg |
| Weekly dose Bleeding population, n=73 | 742.5 mcg/kg |
| Weekly dose Frequent bleeding population, n=36 | 656.2 mcg/kg |
| Weekly dose Missing, n=4 | NA mcg/kg |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 0 / 86 |
| serious Total, serious adverse events | 1 / 86 |
Outcome results
Primary
Percent Change in Total Bleed Episodes Per Month - Bleeding Population
Percent change of bleeds per month in the pre-prophylaxis period and bleeds per month in the prophylaxis period
Time frame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month - Bleeding Population | -45 percent change (%) in bleeds per month |
Primary
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Daily Dosing
Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Daily dosing was defined as 5 to 7 doses per week. All participants = paediatrics, adolescents and adults.
Time frame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Daily Dosing | All, n=29 | -48 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Daily Dosing | Paediatric, n=26 | -48 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Daily Dosing | Adolescent, n=2 | -43 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Daily Dosing | Adult, n=1 | -51 percent change (%) in bleeds per month |
Primary
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Dosing Three Times Per Week
Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Three times per week dosing was defined as dosing two to four times per week. All participants = paediatrics, adolescents and adults.
Time frame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Dosing Three Times Per Week | All, n=32 | -52 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Dosing Three Times Per Week | Paediatric, n=18 | -50 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Dosing Three Times Per Week | Adolescent, n=3 | 12 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Dosing Three Times Per Week | Adult, n=11 | -72 percent change (%) in bleeds per month |
Primary
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Frequent Dosing
Percent change of bleeds per month between the pre-prophylaxis period and the prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Frequent dosing was defined as 7 or more doses per week. All participants = paediatrics, adolescents and adults.
Time frame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Frequent Dosing | All, n=11 | -18 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Frequent Dosing | Paediatric, n=8 | -47 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Frequent Dosing | Adolescent, n=2 | 40 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Frequent Dosing | Adult, n=1 | 35 percent change (%) in bleeds per month |
Primary
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Infrequent Dosing
Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Infrequent dosing was defined as less than two doses per week. All participants = paediatrics, adolescents and adults.
Time frame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Infrequent Dosing | All, n=1 | -6 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Infrequent Dosing | Paediatric, n=1 | -6 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Infrequent Dosing | Adolescent, n=0 | 0 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Infrequent Dosing | Adult, n=0 | 0 percent change (%) in bleeds per month |
Primary
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Daily Dosing
Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Daily dosing was defined as 5 to 7 doses per week. All participants = paediatrics, adolescents and adults.
Time frame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Daily Dosing | All, n=15 | -59 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Daily Dosing | Paediatric, n=13 | -62 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Daily Dosing | Adolescent, n=1 | 47 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Daily Dosing | Adult, n=1 | -51 percent change (%) in bleeds per month |
Primary
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Dosing Three Times Per Week
Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Three times per week dosing was defined as dosing two to four times per week. All participants = paediatrics, adolescents and adults.
Time frame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Dosing Three Times Per Week | All, n=17 | -56 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Dosing Three Times Per Week | Padiatric, n=11 | -57 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Dosing Three Times Per Week | Adolescent, n=2 | -10 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Dosing Three Times Per Week | Adult, n=4 | -76 percent change (%) in bleeds per month |
Primary
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Frequent Dosing
Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Frequent dosing was defined as 7 or more doses per week. All participants = paediatrics, adolescents and adults.
Time frame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Frequent Dosing | All, n=3 | 33 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Frequent Dosing | Paediatric, n=2 | -13 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Frequent Dosing | Adolescent, n=0 | 0 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Frequent Dosing | Adult, n=1 | 35 percent change (%) in bleeds per month |
Primary
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Infrequent Dosing
Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Infrequent dosing was defined as less than two doses per week. All participants = paediatrics, adolescents and adults.
Time frame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Infrequent Dosing | All, n=1 | -6 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Infrequent Dosing | Paediatric, n=1 | -6 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Infrequent Dosing | Adolescent, n=0 | 0 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Infrequent Dosing | Adult, n=0 | 0 percent change (%) in bleeds per month |
Primary
Percent Change in Total Bleed Episodes Per Month - Frequent Bleeding Population
Percent change of bleeds per month in the pre-prophylaxis period and bleeds per month in the prophylaxis period
Time frame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month - Frequent Bleeding Population | -51 percent change (%) in bleeds per month |
Primary
Percent Change in Total Bleed Episodes Per Month Per Age Categories - Bleeding Population
Percent change of bleeds per month between the pre-prophylaxis period and the prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years
Time frame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month Per Age Categories - Bleeding Population | Paediatric, n=54 | -48 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month Per Age Categories - Bleeding Population | Adolescent, n=7 | 0 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month Per Age Categories - Bleeding Population | Adult, n=13 | -56 percent change (%) in bleeds per month |
Primary
Percent Change in Total Bleed Episodes Per Month Per Age Categories - Frequent Bleeding Population
Percent change of bleeds per month between the pre-prophylaxis period and the prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years
Time frame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month Per Age Categories - Frequent Bleeding Population | Paediatric, n=27 | -56 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month Per Age Categories - Frequent Bleeding Population | Adolescent, n=3 | 5 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Percent Change in Total Bleed Episodes Per Month Per Age Categories - Frequent Bleeding Population | Adult, n=6 | -49 percent change (%) in bleeds per month |
Secondary
Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - All Patients
Healthcare resource consumption evaluated the absolute change in number of total hospital length of stay and school/work absences during the pre-prophylaxis period to the prophylaxis period.
Time frame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Whole population of all male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (RFVIIa) for at least 30 days.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Activated Recombinant Human Factor VII | Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - All Patients | Total hospital length of stay | -0.654 change in days per month | Standard Deviation 3.43 |
| Activated Recombinant Human Factor VII | Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - All Patients | School/work absences | -1.365 change in days per month | Standard Deviation 1.99 |
Secondary
Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - Bleeding Population
Healthcare resource consumption evaluated the absolute change in number of total hospital length of stay and school/work absences during the pre-prophylaxis period to the prophylaxis period.
Time frame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Bleeding population with at least one bleed in the pre-prophylaxis of approximately 6 months.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Activated Recombinant Human Factor VII | Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - Bleeding Population | Total hospital length of stay | -0.794 change in days per month | Standard Deviation 3.55 |
| Activated Recombinant Human Factor VII | Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - Bleeding Population | School/work absences | -1.365 change in days per month | Standard Deviation 1.99 |
Secondary
Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - Frequent Bleeding Population
Healthcare resource consumption evaluated the absolute change in number of total hospital length of stay and school/work absences during the pre-prophylaxis period to the prophylaxis period.
Time frame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Activated Recombinant Human Factor VII | Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - Frequent Bleeding Population | Total hospital length of stay | -1.064 change in days per month | Standard Deviation 2.38 |
| Activated Recombinant Human Factor VII | Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - Frequent Bleeding Population | School/work absences | -1.927 change in days per month | Standard Deviation 2.29 |
Secondary
Healthcare Resource Consumption of Visits, Consultations, and Hospital Admissions Per Month - All Patients
Healthcare resource consumption evaluated the absolute change in number of outpatient clinical visits, physician consultations and hospital admissions during the pre-prophylaxis to the prophylaxis period.
Time frame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Whole population of all male patients diagnosed with haemophilia A or B with inhibitor, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Activated Recombinant Human Factor VII | Healthcare Resource Consumption of Visits, Consultations, and Hospital Admissions Per Month - All Patients | Out patient clinical visits | -0.011 change in events per month | Standard Deviation 1.07 |
| Activated Recombinant Human Factor VII | Healthcare Resource Consumption of Visits, Consultations, and Hospital Admissions Per Month - All Patients | Physician consultations | 0.043 change in events per month | Standard Deviation 1.66 |
| Activated Recombinant Human Factor VII | Healthcare Resource Consumption of Visits, Consultations, and Hospital Admissions Per Month - All Patients | Hospital admissions | -0.097 change in events per month | Standard Deviation 0.27 |
Secondary
Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Bleeding Population
Healthcare resource consumption evaluated the absolute change in number of outpatient clinical visits, physician consultations and hospital admissions during the pre-prophylaxis period to the prophylaxis period.
Time frame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Activated Recombinant Human Factor VII | Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Bleeding Population | Out patient clinical visits | 0.0090 change in events per month | Standard Deviation 1.117 |
| Activated Recombinant Human Factor VII | Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Bleeding Population | Physician consultations | 0.053 change in events per month | Standard Deviation 1.76 |
| Activated Recombinant Human Factor VII | Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Bleeding Population | Hospital admissions | -0.11 change in events per month | Standard Deviation 0.28 |
Secondary
Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Frequent Bleeding Population
Healthcare resource consumption evaluated the absolute change in number of outpatient clinical visits, physician consultations and hospital admissions during the pre-prophylaxis period to the prophylaxis period.
Time frame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Activated Recombinant Human Factor VII | Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Frequent Bleeding Population | Out patient clinical visits | 0.0576 change in events per month | Standard Deviation 1.39 |
| Activated Recombinant Human Factor VII | Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Frequent Bleeding Population | Physician consultations | 0.213 change in events per month | Standard Deviation 2.49 |
| Activated Recombinant Human Factor VII | Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Frequent Bleeding Population | Hospital admissions | -0.11 change in events per month | Standard Deviation 0.33 |
Secondary
Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adolescent
Individual activated recombinant human factor VII dose for adolescent patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).
Time frame: Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adolescent | Infrequent dosing, n=0 | 0 mcg/kg |
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adolescent | Dosing three times per week, n=3 | 198 mcg/kg |
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adolescent | Daily dosing, n=2 | 138.9 mcg/kg |
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adolescent | Frequent dosing, n=2 | 138 mcg/kg |
Secondary
Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adult
Individual activated recombinant human factor VII dose for adult patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).
Time frame: Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adult | Infrequent dosing, n=0 | 0 mcg/kg |
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adult | Dosing three times per week, n=11 | 159.7 mcg/kg |
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adult | Daily dosing, n=1 | 117.6 mcg/kg |
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adult | Frequent dosing, n=1 | 103.3 mcg/kg |
Secondary
Individual Dose by Dose Regimen and Age Group - Bleeding Population, Paediatric
Individual activated recombinant human factor VII dose for paediatric patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).
Time frame: Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Bleeding Population, Paediatric | Infrequent dosing, n=1 | 133.3 mcg/kg |
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Bleeding Population, Paediatric | Dosing three times per week, n=18 | 185.4 mcg/kg |
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Bleeding Population, Paediatric | Daily dosing, n=26 | 141.9 mcg/kg |
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Bleeding Population, Paediatric | Frequent dosing, n=8 | 135 mcg/kg |
Secondary
Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adolescent
Individual activated recombinant human factor VII dose for adolescent patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).
Time frame: Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adolescent | Infrequent dosing, n=0 | 0 mcg/kg |
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adolescent | Dosing three times per week, n=2 | 138 mcg/kg |
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adolescent | Daily dosing, n=1 | 280 mcg/kg |
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adolescent | Frequent dosing, n=0 | 0 mcg/kg |
Secondary
Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adult
Individual activated recombinant human factor VII dose for adult patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).
Time frame: Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adult | Infrequent dosing, n=0 | 0 mcg/kg |
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adult | Dosing three times per week, n=4 | 162 mcg/kg |
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adult | Daily dosing, n=1 | 117.6 mcg/kg |
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adult | Frequent dosing, n=1 | 103.3 mcg/kg |
Secondary
Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Paediatric
Individual activated recombinant human factor VII dose for paediatric patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).
Time frame: Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Paediatric | Infrequent dosing, n=1 | 133.3 mcg/kg |
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Paediatric | Dosing three times per week, n=11 | 197.2 mcg/kg |
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Paediatric | Daily dosing, n=13 | 135.9 mcg/kg |
| Activated Recombinant Human Factor VII | Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Paediatric | Frequent dosing, n=2 | 131 mcg/kg |
Secondary
Number of Physician Reported Outcome Assessment in Prophylaxis in Percentage of Patients
Physician's assessment of prophylaxis outcome as successful, partially successful, unsuccessful, or unable to determine
Time frame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Whole population of all male patients diagnosed with haemophilia A or B with inhibitor, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Activated Recombinant Human Factor VII | Number of Physician Reported Outcome Assessment in Prophylaxis in Percentage of Patients | Successful | 40 percentage of patients |
| Activated Recombinant Human Factor VII | Number of Physician Reported Outcome Assessment in Prophylaxis in Percentage of Patients | Partially successful | 23 percentage of patients |
| Activated Recombinant Human Factor VII | Number of Physician Reported Outcome Assessment in Prophylaxis in Percentage of Patients | Unsuccessful | 8 percentage of patients |
| Activated Recombinant Human Factor VII | Number of Physician Reported Outcome Assessment in Prophylaxis in Percentage of Patients | Unable to determine | 10 percentage of patients |
| Activated Recombinant Human Factor VII | Number of Physician Reported Outcome Assessment in Prophylaxis in Percentage of Patients | Data missing or not reported | 17 percentage of patients |
Secondary
Physician Reported Outcome Assessment in Prophylaxis in Number of Patients
Physician's assessment of prophylaxis outcome as successful, partially successful, unsuccessful, or unable to determine
Time frame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Whole population of all male patients diagnosed with haemophilia A or B with inhibitor, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Activated Recombinant Human Factor VII | Physician Reported Outcome Assessment in Prophylaxis in Number of Patients | Successful | 35 patients |
| Activated Recombinant Human Factor VII | Physician Reported Outcome Assessment in Prophylaxis in Number of Patients | Partially successful | 20 patients |
| Activated Recombinant Human Factor VII | Physician Reported Outcome Assessment in Prophylaxis in Number of Patients | Unsuccessful | 7 patients |
| Activated Recombinant Human Factor VII | Physician Reported Outcome Assessment in Prophylaxis in Number of Patients | Unable to determine | 9 patients |
| Activated Recombinant Human Factor VII | Physician Reported Outcome Assessment in Prophylaxis in Number of Patients | Data missing or not reported | 15 patients |
Secondary
Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Bleeding Population
Percent change in bleed episodes per month between pre-prophylaxis period and prophylaxis period by location of joint, target joint (defined as 3 or more documented bleeds in the same joint over the course of 6 months) or non-joint. All joints = target joints and non-target joints.
Time frame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Bleeding population with at least one bleed in the pre-prophylaxis period of approx. 6 months. Patients from the French sites were not included because bleed location was not collected. Bleed episodes with no recorded locations were not included in the analysis
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Activated Recombinant Human Factor VII | Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Bleeding Population | Non-joints | -33 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Bleeding Population | All joints | -39 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Bleeding Population | Target joints | -50 percent change (%) in bleeds per month |
Secondary
Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Frequent Bleeding Population
Percent change in bleed episodes per month between pre-prophylaxis period and prophylaxis period by location of joint, target joint (= 3 or more documented bleeds in the same joint over the course of 6 months) or non-joint. All joints = target joints and non-target joints.
Time frame: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approx. 6 months. Patients from the French sites were not included because bleed location was not collected in these patients. Bleed episodes with no recorded locations were not included in the analysis
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Activated Recombinant Human Factor VII | Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Frequent Bleeding Population | All joints | -46 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Frequent Bleeding Population | Target joints | -56 percent change (%) in bleeds per month |
| Activated Recombinant Human Factor VII | Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Frequent Bleeding Population | Non-joints | -42 percent change (%) in bleeds per month |