Hemophilia A
Conditions
Keywords
FVIII inhibitor
Brief summary
An open-label, single-arm, post- authorization pragmatic clinical trial on the safety and efficacy of Xyntha (Moroctocog-alfa (AF-CC), Recombinant FVIII) in subjects with hemophilia A in usual care settings in China for approximately 6 months or or approximately 50 exposure days whichever occurs first
Detailed description
The purpose of this post-approval study is to provide supplementary information relating to the use of Xyntha (Moroctocog-alfa (AF-CC), Recombinant FVIII) in Chinese subjects with hemophilia A, especially on the safety and efficacy in different populations of Chinese hemophilia A patients, in particular in pediatric patients \<6 years of age, pediatric patients ≥6 to ≤12 years of age, Previously Untreated Patients (PUPs) , subjects receiving prophylaxis treatment after enrollment in the study, and severe patients (FVIII:C \<1%).
Interventions
Enrolled subjects will be treated with intravenous infusions of Xyntha for: • On-Demand treatment, • Surgical Prophylaxis at a dose and frequency prescribed by the subject's treating physician in accordance with the Xyntha label and will be adjusted solely according to medical and therapeutic necessity.
Sponsors
Pfizer
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Male and/or Female subjects with Hemophilia A. * Subjects/parents/legal representatives must be able to comply with registry procedures (informed consent/assent process, clinical visits, reporting of infusion and bleed data, reporting of adverse events, etc). * Evidence of a personally signed and dated informed consent document indicating that the subject (or a legally acceptable representative, parent(s)/legal guardian) has been informed of all pertinent aspects of the study.
Exclusion criteria
* Presence of any other bleeding disorder in addition to hemophilia A. * Treatment with immunomodulatory therapy (e.g., intravenous immunoglobulin, routine systemic corticosteroids, cyclosporins, anti-TNF agents) within 30 days prior to study entry or planned use for the duration of their study participation. * Subjects with a past history of, or current factor VIII inhibitor. For laboratory-based assessments, any Bethesda inhibitor titer greater than the laboratory's normal range or ≥0.6 BU/mL. * Subjects with known hypersensitivity to the active substance or to any of the excipients of Xyntha. * Subjects with a known hypersensitivity to Chinese Hamster Ovary cell proteins. * Unwilling or unable to follow the terms of the protocol. * Any condition which may compromise the subject's ability to comply with and/or perform study-related activities or that poses a clinical contraindication to study participation (these conditions include, but are not limited to, inadequate medical history to assure study eligibility; expectation of poor compliance in provision of observations for study-related documentation), in the opinion of the Investigator. * Participation in other studies involving investigational drug(s) (Phases 1-4) within 30 days before the current study begins and/or during study participation (exception for studies on Xyntha). * Subjects who are investigational site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the Investigator, or subjects who are Pfizer employees directly involved in the conduct of the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With Factor VIII (FVIII) Inhibitors | From Day 1 up to 28 calendar days after End of Treatment (participants had received treatment for 6 months or when participants had achieved 50 exposure days [EDs] whichever occurred first). | Percentage of participants with the product medically important event (MIE) (FVIII inhibitor development during the study). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Response Assessment of On-Demand Treatment of Bleeds | From Day 1 up to participants had received treatment for 6 months or when participants had achieved 50 EDs whichever occurred first. | The proportion of infusions (initial and subsequent for a bleed) in each response category (excellent, good, moderate, no response) was reported. Excellent: Definite pain relief and/or improvement in signs of bleeding starting within 8 hours after an infusion, with no additional infusion administered. Good: Definite pain relief and/or improvement in signs of bleeding starting within 8 hours after an infusion, with at least 1 additional infusion administered for complete resolution of the bleeding episode or definite pain relief and/or improvement in signs of bleeding starting after 8 hours following the infusion, with no additional infusion administered. Moderate: Probable or slight improvement starting after 8 hours following the infusion, with at least 1 additional infusion administered for complete resolution of the bleeding episode. No Response: No improvement at all between infusions or during the 24 hour interval following an infusion, or condition worsens. |
| Number of Infusions Needed to Treat Each New Bleed for On-Demand Treatment | From Day 1 up to participants had received treatment for 6 months or when participants had achieved 50 EDs whichever occurred first. | The number of Xyntha infusions administered to treat a bleed was determined. This was calculated by adding the on-demand initial treatment and any on-demand follow-up infusions for the same bleed (same bleed start date/time). |
| Frequency of Xyntha Infusions to Treat Each New Bleed for On-Demand Group | From Day 1 up to participants had received treatment for 6 months or when participants had achieved 50 EDs whichever occurred first. | The number of bleeds resolved with 1, 2, 3, 4, or \>4 infusions was reported for each of the categories (1, 2, 3, 4, or \>4 infusions needed to treat the bleed), in which the numerator was the number of bleeds falling into each category, and the denominator was the total number of new bleeds across all participants. |
| Hemostatic Efficacy for Surgical Prophylaxis Treatment | From day of surgery to postoperative period (at least 1-3 days post operation or until adequate wound healing for minor surgery or 4-6 days post operation or until threat resolved or adequate wound healing for major surgery) | Assessment of hemostatic efficacy was determined by the investigator and/or surgeon using the 4 point Surgical Hemostasis Efficacy Rating Scale. Excellent: Achieved hemostasis comparable to that expected after similar surgery in a non hemophilic participant. Good: Prolonged time to hemostasis, with somewhat increased bleeding compared to that expected after similar surgery in a non hemophilic participant. Moderate: Obviously delayed hemostasis, but manageable with additional infusions. No Response: No hemostatic response. The percentage of observations in each hemostatic efficacy response category (excellent, good, moderate, none) was reported. |
| Number of Participants With All Causality Treatment-Emergent Adverse Events (AEs) or Serious Adverse Events (SAEs) | From Day 1 up to 28 calendar days after End of Treatment (participants had received treatment for 6 months or when participants had achieved 50 EDs whichever occurred first). | An AE was any untoward medical occurrence without regard to causality in a participant who received study drug. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. An AE was considered treatment emergent if it started for the first time in a participant on or after the first day of active treatment, or the event started before the first day of active treatment but increased in severity during active treatment. AEs included both SAEs and non-serious AEs. |
| Number of Participants With Transfusion Requirement for Surgical Prophylaxis Treatment | From day of surgery to postoperative period (at least 1-3 days post operation or until adequate wound healing for minor surgery or 4-6 days post operation or until threat resolved or adequate wound healing for major surgery) | Number of participants with transfusion requirement for surgical prophylaxis treatment. Transfusion requirements during the intraoperative and the postoperative period were assessed by investigator or surgeon. The number of units and types of blood products transfused were recorded if applicable. |
| Average Infusion Dose and Total Factor VIII Consumption for On-Demand Treatment and Surgical Prophylaxis Treatment | On-Demand Group: Day 1 up to 6 months or 50 EDs whichever occurred first. Surgical Prophylaxis Group: Day of surgery to postoperative period. The duration of postoperative period is specified in previous endpoints. | The total amount (IU) infused for each Xyntha infusion recorded in the study drug infusion log case report form (CRF) was summed to calculate the total factor VIII consumption for each participant. The average infusion dose for each participant was calculated as his total factor VIII consumption (in IU) divided by the number of infusions administered. The total factor VIII consumption, divided by number of infusions, was summarized similarly to average infusion dose (IU). |
| Percentage of Less Than Expected Therapeutic Effect (LETE) in the On-Demand Setting | From Day 1 up to participants had received treatment for 6 months or participants had achieved 50 EDs whichever occurred first. | LETE occurred in the on-demand setting if 2 successive No Response ratings were recorded after 2 successive Xyntha drug infusions, respectively.The infusions must have been administered within 24 hours (less than or equal to 24 hours) of each other for treatment of the same bleeding event in the absence of confounding factors (prespecified). Therefore, LETE in the on-demand setting was based on the response to treatment of a bleeding episode (including those occurring during the surgical prophylaxis period). Note that on-demand treatments administered during the surgical prophylaxis period were also to be included. |
| Number of Confirmed LETE in the Low Recovery Setting | From Day 1 up to participants had received treatment for 6 months or when participants had achieved 50 EDs whichever occurred first. | LETE could also be lower than expected recovery of FVIII in the opinion of the investigator following infusion of Xyntha in the absence of confounding factors. The only confounding factors for low recovery were: known presence or subsequent identification of a FVIII inhibitor; known compromised Xyntha; faulty administration of Xyntha, including inadequate dosing. |
| Actual Estimated Blood Loss for Surgical Prophylaxis Treatment | From day of surgery to postoperative period (at least 1-3 days post operation or until adequate wound healing for minor surgery or 4-6 days post operation or until threat resolved or adequate wound healing for major surgery) | Number of participants with blood loss in each category (Abnormal, Normal, and Absence). Blood loss during the intraoperative and the postoperative period were assessed by investigator or surgeon, which were rated as Abnormal, Normal, and Absence. Abnormal blood loss meant the blood loss was higher over the expectation for the non hemophilic participant. |
Countries
China
Participant flow
Pre-assignment details
As there were 2 participants who rolled over from the surgical prophylaxis group to the on-demand group should be counted once in total column, the total number of baseline participants was 85.
Participants by arm
| Arm | Count |
|---|---|
| On-Demand Group Participants were treated with intravenous infusions of Xyntha 500 International Unit (IU)/vial at a dose and frequency prescribed by the participant's treating physician in accordance with the China Xyntha Package Insert for approximately 6 months or approximately 50 exposure days (EDs). | 73 |
| Surgical Prophylaxis Group Participants were treated with intravenous infusions of Xyntha 500 IU/vial. The treatment duration for surgical prophylaxis was decided by the investigator depending on surgery nature and participant conditions. | 14 |
| Total | 87 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 1 | 0 |
| Overall Study | Protocol Violation | 1 | 0 |
| Overall Study | Withdrawal by Subject | 1 | 0 |
Baseline characteristics
| Characteristic | On-Demand Group | Surgical Prophylaxis Group | Total |
|---|---|---|---|
| Age, Continuous | 7.2 years STANDARD_DEVIATION 4.8 | 21.0 years STANDARD_DEVIATION 15 | 9.5 years STANDARD_DEVIATION 9 |
| Sex: Female, Male Female | 1 Participants | 0 Participants | 1 Participants |
| Sex: Female, Male Male | 72 Participants | 14 Participants | 86 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — | — / — |
| other Total, other adverse events | 62 / 73 | 9 / 14 | 70 / 85 |
| serious Total, serious adverse events | 7 / 73 | 1 / 14 | 8 / 85 |
Outcome results
Primary
Percentage of Participants With Factor VIII (FVIII) Inhibitors
Percentage of participants with the product medically important event (MIE) (FVIII inhibitor development during the study).
Time frame: From Day 1 up to 28 calendar days after End of Treatment (participants had received treatment for 6 months or when participants had achieved 50 exposure days [EDs] whichever occurred first).
Population: The safety analysis set was defined as all participants who received at least one dose of Xyntha during the study.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| On-Demand Group | Percentage of Participants With Factor VIII (FVIII) Inhibitors | 8.22 percentage of participants |
| Surgical Prophylaxis Group | Percentage of Participants With Factor VIII (FVIII) Inhibitors | 7.14 percentage of participants |
Secondary
Actual Estimated Blood Loss for Surgical Prophylaxis Treatment
Number of participants with blood loss in each category (Abnormal, Normal, and Absence). Blood loss during the intraoperative and the postoperative period were assessed by investigator or surgeon, which were rated as Abnormal, Normal, and Absence. Abnormal blood loss meant the blood loss was higher over the expectation for the non hemophilic participant.
Time frame: From day of surgery to postoperative period (at least 1-3 days post operation or until adequate wound healing for minor surgery or 4-6 days post operation or until threat resolved or adequate wound healing for major surgery)
Population: Surgical prophylaxis participants during their surgical prophylaxis period. The data for this outcome was not planned to be analyzed for the on-demand group. Number of participants analyzed signifies participants evaluable for this outcome measure.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| On-Demand Group | Actual Estimated Blood Loss for Surgical Prophylaxis Treatment | Abnormal | 0 partcipants |
| On-Demand Group | Actual Estimated Blood Loss for Surgical Prophylaxis Treatment | Normal | 13 partcipants |
| On-Demand Group | Actual Estimated Blood Loss for Surgical Prophylaxis Treatment | Absence | 0 partcipants |
Secondary
Average Infusion Dose and Total Factor VIII Consumption for On-Demand Treatment and Surgical Prophylaxis Treatment
The total amount (IU) infused for each Xyntha infusion recorded in the study drug infusion log case report form (CRF) was summed to calculate the total factor VIII consumption for each participant. The average infusion dose for each participant was calculated as his total factor VIII consumption (in IU) divided by the number of infusions administered. The total factor VIII consumption, divided by number of infusions, was summarized similarly to average infusion dose (IU).
Time frame: On-Demand Group: Day 1 up to 6 months or 50 EDs whichever occurred first. Surgical Prophylaxis Group: Day of surgery to postoperative period. The duration of postoperative period is specified in previous endpoints.
Population: All participants who received at least one dose of Xyntha during the study
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| On-Demand Group | Average Infusion Dose and Total Factor VIII Consumption for On-Demand Treatment and Surgical Prophylaxis Treatment | Average Infusion Dose | 674.68 International Unit (IU) | Standard Deviation 357.613 |
| On-Demand Group | Average Infusion Dose and Total Factor VIII Consumption for On-Demand Treatment and Surgical Prophylaxis Treatment | Total FVIII Consumption per Participant | 25211.0 International Unit (IU) | Standard Deviation 22035.35 |
| Surgical Prophylaxis Group | Average Infusion Dose and Total Factor VIII Consumption for On-Demand Treatment and Surgical Prophylaxis Treatment | Average Infusion Dose | 1245.07 International Unit (IU) | Standard Deviation 494.268 |
| Surgical Prophylaxis Group | Average Infusion Dose and Total Factor VIII Consumption for On-Demand Treatment and Surgical Prophylaxis Treatment | Total FVIII Consumption per Participant | 39860.7 International Unit (IU) | Standard Deviation 27546.15 |
Secondary
Frequency of Xyntha Infusions to Treat Each New Bleed for On-Demand Group
The number of bleeds resolved with 1, 2, 3, 4, or \>4 infusions was reported for each of the categories (1, 2, 3, 4, or \>4 infusions needed to treat the bleed), in which the numerator was the number of bleeds falling into each category, and the denominator was the total number of new bleeds across all participants.
Time frame: From Day 1 up to participants had received treatment for 6 months or when participants had achieved 50 EDs whichever occurred first.
Population: Participants with a bleed during the study for which on-demand treatment with Xyntha was administered. The data for this outcome was not planned to be analyzed for the surgical prophylaxis group.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| On-Demand Group | Frequency of Xyntha Infusions to Treat Each New Bleed for On-Demand Group | 1 Infusion | 63.0 percentage of bleeds |
| On-Demand Group | Frequency of Xyntha Infusions to Treat Each New Bleed for On-Demand Group | 2 Infusions | 21.9 percentage of bleeds |
| On-Demand Group | Frequency of Xyntha Infusions to Treat Each New Bleed for On-Demand Group | 3 Infusions | 8.6 percentage of bleeds |
| On-Demand Group | Frequency of Xyntha Infusions to Treat Each New Bleed for On-Demand Group | 4 Infusions | 6.0 percentage of bleeds |
| On-Demand Group | Frequency of Xyntha Infusions to Treat Each New Bleed for On-Demand Group | >4 Infusions | 0.5 percentage of bleeds |
Secondary
Hemostatic Efficacy for Surgical Prophylaxis Treatment
Assessment of hemostatic efficacy was determined by the investigator and/or surgeon using the 4 point Surgical Hemostasis Efficacy Rating Scale. Excellent: Achieved hemostasis comparable to that expected after similar surgery in a non hemophilic participant. Good: Prolonged time to hemostasis, with somewhat increased bleeding compared to that expected after similar surgery in a non hemophilic participant. Moderate: Obviously delayed hemostasis, but manageable with additional infusions. No Response: No hemostatic response. The percentage of observations in each hemostatic efficacy response category (excellent, good, moderate, none) was reported.
Time frame: From day of surgery to postoperative period (at least 1-3 days post operation or until adequate wound healing for minor surgery or 4-6 days post operation or until threat resolved or adequate wound healing for major surgery)
Population: Surgical prophylaxis participants during their surgical prophylaxis period. The data for this outcome was not planned to be analyzed for the on-demand group.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| On-Demand Group | Hemostatic Efficacy for Surgical Prophylaxis Treatment | Day of Surgery: Excellent | 71.4 percentage of observations |
| On-Demand Group | Hemostatic Efficacy for Surgical Prophylaxis Treatment | Day of Surgery: Good | 28.6 percentage of observations |
| On-Demand Group | Hemostatic Efficacy for Surgical Prophylaxis Treatment | Day of Surgery: Moderate | 0 percentage of observations |
| On-Demand Group | Hemostatic Efficacy for Surgical Prophylaxis Treatment | Day of Surgery: None | 0 percentage of observations |
| On-Demand Group | Hemostatic Efficacy for Surgical Prophylaxis Treatment | Post-Operative: Excellent | 75.5 percentage of observations |
| On-Demand Group | Hemostatic Efficacy for Surgical Prophylaxis Treatment | Post-Operative: Good | 24.5 percentage of observations |
| On-Demand Group | Hemostatic Efficacy for Surgical Prophylaxis Treatment | Post-Operative: Moderate | 0 percentage of observations |
| On-Demand Group | Hemostatic Efficacy for Surgical Prophylaxis Treatment | Post-Operative: None | 0 percentage of observations |
Secondary
Number of Confirmed LETE in the Low Recovery Setting
LETE could also be lower than expected recovery of FVIII in the opinion of the investigator following infusion of Xyntha in the absence of confounding factors. The only confounding factors for low recovery were: known presence or subsequent identification of a FVIII inhibitor; known compromised Xyntha; faulty administration of Xyntha, including inadequate dosing.
Time frame: From Day 1 up to participants had received treatment for 6 months or when participants had achieved 50 EDs whichever occurred first.
Population: The safety analysis set was defined as all participants who received at least one dose of Xyntha during the study.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| On-Demand Group | Number of Confirmed LETE in the Low Recovery Setting | 0 LETE bleeds |
| Surgical Prophylaxis Group | Number of Confirmed LETE in the Low Recovery Setting | 0 LETE bleeds |
Secondary
Number of Infusions Needed to Treat Each New Bleed for On-Demand Treatment
The number of Xyntha infusions administered to treat a bleed was determined. This was calculated by adding the on-demand initial treatment and any on-demand follow-up infusions for the same bleed (same bleed start date/time).
Time frame: From Day 1 up to participants had received treatment for 6 months or when participants had achieved 50 EDs whichever occurred first.
Population: Participants with a bleed during the study for which on-demand treatment with Xyntha was administered. The data for this outcome was not planned to be analyzed for the surgical prophylaxis group.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| On-Demand Group | Number of Infusions Needed to Treat Each New Bleed for On-Demand Treatment | 1.6 infusions | Standard Deviation 0.94 |
Secondary
Number of Participants With All Causality Treatment-Emergent Adverse Events (AEs) or Serious Adverse Events (SAEs)
An AE was any untoward medical occurrence without regard to causality in a participant who received study drug. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. An AE was considered treatment emergent if it started for the first time in a participant on or after the first day of active treatment, or the event started before the first day of active treatment but increased in severity during active treatment. AEs included both SAEs and non-serious AEs.
Time frame: From Day 1 up to 28 calendar days after End of Treatment (participants had received treatment for 6 months or when participants had achieved 50 EDs whichever occurred first).
Population: The safety analysis set was defined as all participants who received at least one dose of Xyntha during the study.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| On-Demand Group | Number of Participants With All Causality Treatment-Emergent Adverse Events (AEs) or Serious Adverse Events (SAEs) | Participants with AEs | 65 participants |
| On-Demand Group | Number of Participants With All Causality Treatment-Emergent Adverse Events (AEs) or Serious Adverse Events (SAEs) | Participants with SAEs | 7 participants |
| Surgical Prophylaxis Group | Number of Participants With All Causality Treatment-Emergent Adverse Events (AEs) or Serious Adverse Events (SAEs) | Participants with AEs | 10 participants |
| Surgical Prophylaxis Group | Number of Participants With All Causality Treatment-Emergent Adverse Events (AEs) or Serious Adverse Events (SAEs) | Participants with SAEs | 1 participants |
Secondary
Number of Participants With Transfusion Requirement for Surgical Prophylaxis Treatment
Number of participants with transfusion requirement for surgical prophylaxis treatment. Transfusion requirements during the intraoperative and the postoperative period were assessed by investigator or surgeon. The number of units and types of blood products transfused were recorded if applicable.
Time frame: From day of surgery to postoperative period (at least 1-3 days post operation or until adequate wound healing for minor surgery or 4-6 days post operation or until threat resolved or adequate wound healing for major surgery)
Population: Surgical prophylaxis participants during their surgical prophylaxis period. The data for this outcome was not planned to be analyzed for the on-demand group.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| On-Demand Group | Number of Participants With Transfusion Requirement for Surgical Prophylaxis Treatment | 2 participants |
Secondary
Percentage of Less Than Expected Therapeutic Effect (LETE) in the On-Demand Setting
LETE occurred in the on-demand setting if 2 successive No Response ratings were recorded after 2 successive Xyntha drug infusions, respectively.The infusions must have been administered within 24 hours (less than or equal to 24 hours) of each other for treatment of the same bleeding event in the absence of confounding factors (prespecified). Therefore, LETE in the on-demand setting was based on the response to treatment of a bleeding episode (including those occurring during the surgical prophylaxis period). Note that on-demand treatments administered during the surgical prophylaxis period were also to be included.
Time frame: From Day 1 up to participants had received treatment for 6 months or participants had achieved 50 EDs whichever occurred first.
Population: The safety analysis set was defined as all participants who received at least one dose of Xyntha during the study.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| On-Demand Group | Percentage of Less Than Expected Therapeutic Effect (LETE) in the On-Demand Setting | 0.06 percentage of bleeding episodes |
Secondary
Response Assessment of On-Demand Treatment of Bleeds
The proportion of infusions (initial and subsequent for a bleed) in each response category (excellent, good, moderate, no response) was reported. Excellent: Definite pain relief and/or improvement in signs of bleeding starting within 8 hours after an infusion, with no additional infusion administered. Good: Definite pain relief and/or improvement in signs of bleeding starting within 8 hours after an infusion, with at least 1 additional infusion administered for complete resolution of the bleeding episode or definite pain relief and/or improvement in signs of bleeding starting after 8 hours following the infusion, with no additional infusion administered. Moderate: Probable or slight improvement starting after 8 hours following the infusion, with at least 1 additional infusion administered for complete resolution of the bleeding episode. No Response: No improvement at all between infusions or during the 24 hour interval following an infusion, or condition worsens.
Time frame: From Day 1 up to participants had received treatment for 6 months or when participants had achieved 50 EDs whichever occurred first.
Population: Participants with a bleed during the study for which on-demand treatment with Xyntha was administered. The data for this outcome was not planned to be analyzed for the surgical prophylaxis group.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| On-Demand Group | Response Assessment of On-Demand Treatment of Bleeds | No Response | 0.3 percentage of infusions |
| On-Demand Group | Response Assessment of On-Demand Treatment of Bleeds | Data Not Recorded | 0.1 percentage of infusions |
| On-Demand Group | Response Assessment of On-Demand Treatment of Bleeds | Excellent | 46.9 percentage of infusions |
| On-Demand Group | Response Assessment of On-Demand Treatment of Bleeds | Good | 40.0 percentage of infusions |
| On-Demand Group | Response Assessment of On-Demand Treatment of Bleeds | Moderate | 12.7 percentage of infusions |