Hemophilia A
Conditions
Brief summary
The purpose of this survey is to understand the following items in the actual clinical use of ADYNOVATE in patients: 1. Unexpected adverse drug reactions 2. Occurrence of adverse drug reactions in the actual clinical use 3. Factors that may affect safety and efficacy 4. Occurrence of Factor VIII inhibitor development in patients with coagulation factor VIII deficiency (hereinafter hemophilia A) 5. Safety and efficacy for hemophilia A patients who received routine prophylactic therapy and on-demand therapy
Interventions
BIOLOGICALADYNOVATE
Antihemophilic Factor (Recombinant), PEGylated
Sponsors
Takeda
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Hemophilia A patients who receive ADYNOVATE, including previously treated patients with Factor VIII deficiency (PTPs), and previously untreated patients with Factor VIII deficiency (PUPs) who are treated with ADYNOVATE.
Exclusion criteria
* Patients not administered ADYNOVATE.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Hemostatic Effectiveness of Study Drug on an On-Demand Regimen | Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs | Percentage of each category of hemostatic effectiveness for an on-demand regimen assessed by the investigator was reported. Hemostatic effectiveness was assessed by the investigator with following 4-point ordinal scale: Excellent, Good, Fair, Poor. |
| Number of Doses Per a Bleeding Episode of Study Drug an On-Demand Regimen | Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs | Number of doses per a bleeding episode of study drug on an on-demand regimen was reported. |
| Hemostatic Effectiveness of Study Drug on Treatment of Breakthrough Bleeding Episodes With a Prophylaxis Regimen | Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs | Percentage of each category of hemostatic effectiveness for treatment of breakthrough bleeding episodes in a prophylaxis regimen assessed by the investigator was reported. Hemostatic effectiveness was assessed by the investigator with following 4-point ordinal scale: Excellent, Good, Fair, Poor. |
| Number of Participants Who Discontinued the Use of Study Drug | Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs | Number of previously treated patients (PTPs) and previously untreated patients (PUPs) who discontinued the use of ADYNOVATE were reported. |
| Annual Bleed Rate (ABR) of Spontaneous Bleeding Episodes on a Prophylaxis Regimen | Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs | Annual bleed rate (ABR) of spontaneous bleeding episodes in PTPs and PUPs on a prophylaxis regimen were reported. Annual bleed rate is calculated by the number of bleeding episodes observed during administration period divided by the duration of administration period, after that multiplied with 365.2425. |
| Annual Bleed Rate (ABR) of Breakthrough Bleeding Episodes on a Prophylaxis Regimen | Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs | Annual bleed rate (ABR) of breakthrough bleeding episodes in PTPs and PUPs on a prophylaxis regimen were reported. Annual bleed rate is calculated by the number of bleeding episodes observed during administration period divided by the duration of administration period, after that multiplied with 365.2425. |
| Duration of Treatment of Study Drug on a Prophylaxis Regimen | Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs. | Duration of treatment of study drug on a prophylaxis regimen was reported. |
| Duration of Treatment of Study Drug an On-Demand Regimen | Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs | Duration of treatment of study drug on an on-demand regimen was reported. |
| Dose Per Administration of Study Drug on a Prophylaxis Regimen | Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs | Dose per administration of study drug on a prophylaxis regimen was reported. |
| Dose Per Administration of Study Drug an On-Demand Regimen | Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs | Dose per administration of study drug on an on-demand regimen was reported. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Who Experience Factor VIII Inhibition, Dermatitis Atopic or Eczema as an Adverse Event (AE) | Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs | Number of PTPs and PUPs who experienced factor VIII inhibition, dermatitis atopic or eczema as an AE related to development of inhibitors, shock or anaphylaxis was reported. |
| Number of Doses Per a Week of Study Drug on a Prophylaxis Regimen | Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs | Number of doses per a week of study drug on a prophylaxis regimen was reported. |
Countries
Japan
Participant flow
Recruitment details
Participants took part in the survey at 66 investigative sites in Japan, from 1 February 2017 to 15 September 2023.
Pre-assignment details
Participants with a historical diagnosis of hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) were enrolled. This included previously treated patients with Factor VIII deficiency (PTPs) and previously untreated patients with Factor VIII deficiency (PUPs) who were treated with ADYNOVATE. Participants received ADYNOVATE intravenous Infusion as part of a routine medical care.
Participants by arm
| Arm | Count |
|---|---|
| Previously Treated Patients (PTPs) Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously treated patients with Factor VIII deficiency (PTPs) who had 4 or more exposure days to other Factor VIII (FVIII) products. | 123 |
| Previously Untreated Patients (PUPs) Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously untreated patients with Factor VIII deficiency (PUPs) who had 3 or less previous exposure days to other Factor VIII (FVIII) products. | 12 |
| Total | 135 |
Baseline characteristics
| Characteristic | Total | Previously Untreated Patients (PUPs) | Previously Treated Patients (PTPs) |
|---|---|---|---|
| Age, Customized < 12 years | 35 Participants | 6 Participants | 29 Participants |
| Age, Customized >= 12 years, < 18 years | 5 Participants | 1 Participants | 4 Participants |
| Age, Customized >= 18 years, < 65 years | 80 Participants | 3 Participants | 77 Participants |
| Age, Customized 65 years or more | 9 Participants | 2 Participants | 7 Participants |
| Age, Customized Unknown | 6 Participants | 0 Participants | 6 Participants |
| Age of Diagnosis of Hemophilia A >= 1, < 12 years old | 31 Participants | 3 Participants | 28 Participants |
| Age of Diagnosis of Hemophilia A >= 18, < 65 years old | 7 Participants | 3 Participants | 4 Participants |
| Age of Diagnosis of Hemophilia A <=18 years | 4 Participants | 1 Participants | 3 Participants |
| Age of Diagnosis of Hemophilia A < 1 year old | 41 Participants | 3 Participants | 38 Participants |
| Age of Diagnosis of Hemophilia A >= 65 years old | 2 Participants | 1 Participants | 1 Participants |
| Age of Diagnosis of Hemophilia A Unknown | 50 Participants | 1 Participants | 49 Participants |
| Allergy Had Allergy | 8 Participants | 1 Participants | 7 Participants |
| Allergy Had No Allergy | 122 Participants | 11 Participants | 111 Participants |
| Allergy Unknown | 5 Participants | 0 Participants | 5 Participants |
| Congenital Hemophilia A | 135 Participants | 12 Participants | 123 Participants |
| Dosing Regimen before the Start of Administration of ADYNOVATE Had Not Treated | 7 Participants | 7 Participants | 0 Participants |
| Dosing Regimen before the Start of Administration of ADYNOVATE On-demand Replacement Therapy | 25 Participants | 4 Participants | 21 Participants |
| Dosing Regimen before the Start of Administration of ADYNOVATE Prophylaxis and On-demand Replacement Therapy | 22 Participants | 0 Participants | 22 Participants |
| Dosing Regimen before the Start of Administration of ADYNOVATE Prophylaxis Therapy | 78 Participants | 0 Participants | 78 Participants |
| Dosing Regimen before the Start of Administration of ADYNOVATE The Other Therapy | 3 Participants | 1 Participants | 2 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 135 Participants | 12 Participants | 123 Participants |
| Experience of Serious Bleeding Had Experience of Serious Bleeding | 19 Participants | 0 Participants | 19 Participants |
| Experience of Serious Bleeding Had No Experience of Serious Bleeding | 110 Participants | 12 Participants | 98 Participants |
| Experience of Serious Bleeding Unknown | 6 Participants | 0 Participants | 6 Participants |
| Experience of Surgery Had Experience of Surgery | 29 Participants | 2 Participants | 27 Participants |
| Experience of Surgery Had No Experience of Surgery | 100 Participants | 9 Participants | 91 Participants |
| Experience of Surgery Unknown | 6 Participants | 1 Participants | 5 Participants |
| Factor VIII Inhibitor Development Had Factor VIII Inhibitor Development | 7 Participants | 0 Participants | 7 Participants |
| Factor VIII Inhibitor Development Had No Factor VIII Inhibitor Development | 104 Participants | 7 Participants | 97 Participants |
| Factor VIII Inhibitor Development Unknown | 24 Participants | 5 Participants | 19 Participants |
| Family History of Hemophilia Had Family History of Hemophilia | 51 Participants | 5 Participants | 46 Participants |
| Family History of Hemophilia Had No Family History of Hemophilia | 56 Participants | 4 Participants | 52 Participants |
| Family History of Hemophilia Unknown | 28 Participants | 3 Participants | 25 Participants |
| Family History of Inhibitor Development Had Family History of Inhibitor Development | 6 Participants | 0 Participants | 6 Participants |
| Family History of Inhibitor Development Had No Family History of Inhibitor Development | 90 Participants | 5 Participants | 85 Participants |
| Family History of Inhibitor Development Unknown | 39 Participants | 7 Participants | 32 Participants |
| Healthcare Category Inpatient | 9 Participants | 5 Participants | 4 Participants |
| Healthcare Category Outpatient | 126 Participants | 7 Participants | 119 Participants |
| Hemophilic Arthropathy Had Hemophilic Arthropathy | 63 Participants | 3 Participants | 60 Participants |
| Hemophilic Arthropathy Had No Hemophilic Arthropathy | 69 Participants | 8 Participants | 61 Participants |
| Hemophilic Arthropathy Unknown | 3 Participants | 1 Participants | 2 Participants |
| Hepatic Impairment Had Hepatic Impairment | 8 Participants | 0 Participants | 8 Participants |
| Hepatic Impairment Had No Hepatic Impairment | 126 Participants | 12 Participants | 114 Participants |
| Hepatic Impairment Unknown | 1 Participants | 0 Participants | 1 Participants |
| Medical Complications Had Medical Complications | 35 Participants | 3 Participants | 32 Participants |
| Medical Complications Had No Medical Complications | 98 Participants | 8 Participants | 90 Participants |
| Medical Complications Unknown | 2 Participants | 1 Participants | 1 Participants |
| Medical History Had Medical History | 35 Participants | 2 Participants | 33 Participants |
| Medical History Had No Medical History | 96 Participants | 10 Participants | 86 Participants |
| Medical History Unknown | 4 Participants | 0 Participants | 4 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 135 Participants | 12 Participants | 123 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 0 Participants | 0 Participants | 0 Participants |
| Region of Enrollment Japan | 135 Participants | 12 Participants | 123 Participants |
| Renal Impairment Had No Renal Impairment | 132 Participants | 12 Participants | 120 Participants |
| Renal Impairment Had Renal Impairment | 2 Participants | 0 Participants | 2 Participants |
| Renal Impairment Unknown | 1 Participants | 0 Participants | 1 Participants |
| Severity of Hemophilia A Mild | 9 Participants | 2 Participants | 7 Participants |
| Severity of Hemophilia A Moderate | 18 Participants | 2 Participants | 16 Participants |
| Severity of Hemophilia A Severe | 101 Participants | 8 Participants | 93 Participants |
| Severity of Hemophilia A Unknown | 7 Participants | 0 Participants | 7 Participants |
| Sex: Female, Male Female | 1 Participants | 1 Participants | 0 Participants |
| Sex: Female, Male Male | 134 Participants | 11 Participants | 123 Participants |
| Status of Concomitant Drugs | 41 Participants | 7 Participants | 34 Participants |
| Status of Concomitant Therapies | 1 Participants | 0 Participants | 1 Participants |
| Target Joint of Hemophilic Arthropathy Had No Target Joint of Hemophilic Arthropathy | 110 Participants | 10 Participants | 100 Participants |
| Target Joint of Hemophilic Arthropathy Had Target Joint of Hemophilic Arthropathy | 17 Participants | 0 Participants | 17 Participants |
| Target Joint of Hemophilic Arthropathy Unknown | 8 Participants | 2 Participants | 6 Participants |
| Times of Bleeding in One Year >= 10 times per a year | 14 Participants | 0 Participants | 14 Participants |
| Times of Bleeding in One Year >= 1, < 10 times per a year | 44 Participants | 0 Participants | 44 Participants |
| Times of Bleeding in One Year < 1 time per a year | 33 Participants | 4 Participants | 29 Participants |
| Times of Bleeding in One Year Unknown | 44 Participants | 8 Participants | 36 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 123 | 0 / 12 |
| other Total, other adverse events | 1 / 123 | 5 / 12 |
| serious Total, serious adverse events | 2 / 123 | 1 / 12 |
Outcome results
Primary
Annual Bleed Rate (ABR) of Breakthrough Bleeding Episodes on a Prophylaxis Regimen
Annual bleed rate (ABR) of breakthrough bleeding episodes in PTPs and PUPs on a prophylaxis regimen were reported. Annual bleed rate is calculated by the number of bleeding episodes observed during administration period divided by the duration of administration period, after that multiplied with 365.2425.
Time frame: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs
Population: Efficacy analysis set: all participants who received at least one dose of protocol treatment without major protocol deviation. The number analyzed was the number of participants with data available for analysis who treated with a prophylaxis regimen and experienced breakthrough bleeding episodes during the administration period.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Previously Treated Patients (PTPs) | Annual Bleed Rate (ABR) of Breakthrough Bleeding Episodes on a Prophylaxis Regimen | 3.16 Bleeds per year |
| Previously Untreated Patients (PUPs) | Annual Bleed Rate (ABR) of Breakthrough Bleeding Episodes on a Prophylaxis Regimen | 2.91 Bleeds per year |
Primary
Annual Bleed Rate (ABR) of Spontaneous Bleeding Episodes on a Prophylaxis Regimen
Annual bleed rate (ABR) of spontaneous bleeding episodes in PTPs and PUPs on a prophylaxis regimen were reported. Annual bleed rate is calculated by the number of bleeding episodes observed during administration period divided by the duration of administration period, after that multiplied with 365.2425.
Time frame: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs
Population: Efficacy analysis set: all participants who received at least one dose of protocol treatment without major protocol deviation. The number analyzed was the number of participants with data available for analysis who treated with a prophylaxis regimen and experienced spontaneous bleeding episodes during the administration period.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Previously Treated Patients (PTPs) | Annual Bleed Rate (ABR) of Spontaneous Bleeding Episodes on a Prophylaxis Regimen | 1.99 Bleeds per year |
Primary
Dose Per Administration of Study Drug an On-Demand Regimen
Dose per administration of study drug on an on-demand regimen was reported.
Time frame: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs
Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. The number analyzed was the number of participants with data available for analysis who treated with an on-demand regimen during the administration period.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Previously Treated Patients (PTPs) | Dose Per Administration of Study Drug an On-Demand Regimen | 36.8 IU per kilograms (kg) | Standard Deviation 13.88 |
| Previously Untreated Patients (PUPs) | Dose Per Administration of Study Drug an On-Demand Regimen | 29.7 IU per kilograms (kg) | Standard Deviation 12.42 |
Primary
Dose Per Administration of Study Drug on a Prophylaxis Regimen
Dose per administration of study drug on a prophylaxis regimen was reported.
Time frame: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs
Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. The number analyzed was the number of participants with data available for analysis who treated with a prophylaxis regimen during the administration period.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Previously Treated Patients (PTPs) | Dose Per Administration of Study Drug on a Prophylaxis Regimen | 39.4 IU per kilograms (kg) | Standard Deviation 12.69 |
| Previously Untreated Patients (PUPs) | Dose Per Administration of Study Drug on a Prophylaxis Regimen | 45.8 IU per kilograms (kg) | Standard Deviation 7.7 |
Primary
Duration of Treatment of Study Drug an On-Demand Regimen
Duration of treatment of study drug on an on-demand regimen was reported.
Time frame: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs
Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. The number analyzed was the number of participants with data available for analysis who treated with an on-demand regimen during the administration period.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Previously Treated Patients (PTPs) | Duration of Treatment of Study Drug an On-Demand Regimen | 13.3 days | Standard Deviation 22.42 |
| Previously Untreated Patients (PUPs) | Duration of Treatment of Study Drug an On-Demand Regimen | 5.0 days | Standard Deviation 2.65 |
Primary
Duration of Treatment of Study Drug on a Prophylaxis Regimen
Duration of treatment of study drug on a prophylaxis regimen was reported.
Time frame: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs.
Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. The number analyzed was the number of participants with data available for analysis who treated with a prophylaxis regimen during the administration period.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Previously Treated Patients (PTPs) | Duration of Treatment of Study Drug on a Prophylaxis Regimen | 343.0 days | Standard Deviation 69.33 |
| Previously Untreated Patients (PUPs) | Duration of Treatment of Study Drug on a Prophylaxis Regimen | 510.7 days | Standard Deviation 256.04 |
Primary
Hemostatic Effectiveness of Study Drug on an On-Demand Regimen
Percentage of each category of hemostatic effectiveness for an on-demand regimen assessed by the investigator was reported. Hemostatic effectiveness was assessed by the investigator with following 4-point ordinal scale: Excellent, Good, Fair, Poor.
Time frame: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs
Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. The number analyzed was the number of participants with data available for analysis who treated with on-demand regimen during the administration period.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Previously Treated Patients (PTPs) | Hemostatic Effectiveness of Study Drug on an On-Demand Regimen | Excellent | 32.76 Percentage of bleeding episodes |
| Previously Treated Patients (PTPs) | Hemostatic Effectiveness of Study Drug on an On-Demand Regimen | Good | 60.34 Percentage of bleeding episodes |
| Previously Treated Patients (PTPs) | Hemostatic Effectiveness of Study Drug on an On-Demand Regimen | Fair | 6.90 Percentage of bleeding episodes |
| Previously Treated Patients (PTPs) | Hemostatic Effectiveness of Study Drug on an On-Demand Regimen | Poor | 0.00 Percentage of bleeding episodes |
| Previously Untreated Patients (PUPs) | Hemostatic Effectiveness of Study Drug on an On-Demand Regimen | Poor | 33.33 Percentage of bleeding episodes |
| Previously Untreated Patients (PUPs) | Hemostatic Effectiveness of Study Drug on an On-Demand Regimen | Excellent | 33.33 Percentage of bleeding episodes |
| Previously Untreated Patients (PUPs) | Hemostatic Effectiveness of Study Drug on an On-Demand Regimen | Fair | 0.00 Percentage of bleeding episodes |
| Previously Untreated Patients (PUPs) | Hemostatic Effectiveness of Study Drug on an On-Demand Regimen | Good | 33.33 Percentage of bleeding episodes |
Primary
Hemostatic Effectiveness of Study Drug on Treatment of Breakthrough Bleeding Episodes With a Prophylaxis Regimen
Percentage of each category of hemostatic effectiveness for treatment of breakthrough bleeding episodes in a prophylaxis regimen assessed by the investigator was reported. Hemostatic effectiveness was assessed by the investigator with following 4-point ordinal scale: Excellent, Good, Fair, Poor.
Time frame: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs
Population: Efficacy analysis set: all participants who received at least one dose of protocol treatment without major protocol deviation. The number analyzed was the number of participants with data available for analysis who treated with a prophylaxis regimen and experienced breakthrough bleeding episodes during the administration period.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Previously Treated Patients (PTPs) | Hemostatic Effectiveness of Study Drug on Treatment of Breakthrough Bleeding Episodes With a Prophylaxis Regimen | Excellent | 37.21 Percentage of bleeding episodes |
| Previously Treated Patients (PTPs) | Hemostatic Effectiveness of Study Drug on Treatment of Breakthrough Bleeding Episodes With a Prophylaxis Regimen | Good | 62.79 Percentage of bleeding episodes |
| Previously Treated Patients (PTPs) | Hemostatic Effectiveness of Study Drug on Treatment of Breakthrough Bleeding Episodes With a Prophylaxis Regimen | Fair | 0.00 Percentage of bleeding episodes |
| Previously Treated Patients (PTPs) | Hemostatic Effectiveness of Study Drug on Treatment of Breakthrough Bleeding Episodes With a Prophylaxis Regimen | Poor | 0.00 Percentage of bleeding episodes |
| Previously Untreated Patients (PUPs) | Hemostatic Effectiveness of Study Drug on Treatment of Breakthrough Bleeding Episodes With a Prophylaxis Regimen | Poor | 20.00 Percentage of bleeding episodes |
| Previously Untreated Patients (PUPs) | Hemostatic Effectiveness of Study Drug on Treatment of Breakthrough Bleeding Episodes With a Prophylaxis Regimen | Excellent | 20.00 Percentage of bleeding episodes |
| Previously Untreated Patients (PUPs) | Hemostatic Effectiveness of Study Drug on Treatment of Breakthrough Bleeding Episodes With a Prophylaxis Regimen | Fair | 0.00 Percentage of bleeding episodes |
| Previously Untreated Patients (PUPs) | Hemostatic Effectiveness of Study Drug on Treatment of Breakthrough Bleeding Episodes With a Prophylaxis Regimen | Good | 40.00 Percentage of bleeding episodes |
Primary
Number of Doses Per a Bleeding Episode of Study Drug an On-Demand Regimen
Number of doses per a bleeding episode of study drug on an on-demand regimen was reported.
Time frame: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs
Population: Efficacy analysis set: all participants who received at least one dose of protocol treatment without major protocol deviation. The number analyzed was the number of participants with data available for analysis who treated with an on-demand regimen during the administration period.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Previously Treated Patients (PTPs) | Number of Doses Per a Bleeding Episode of Study Drug an On-Demand Regimen | 6.0 Number of doses per a bleeding episode | Standard Deviation 4.45 |
| Previously Untreated Patients (PUPs) | Number of Doses Per a Bleeding Episode of Study Drug an On-Demand Regimen | 5.0 Number of doses per a bleeding episode | Standard Deviation 4.36 |
Primary
Number of Participants Who Discontinued the Use of Study Drug
Number of previously treated patients (PTPs) and previously untreated patients (PUPs) who discontinued the use of ADYNOVATE were reported.
Time frame: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs
Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Previously Treated Patients (PTPs) | Number of Participants Who Discontinued the Use of Study Drug | 10 Participants |
| Previously Untreated Patients (PUPs) | Number of Participants Who Discontinued the Use of Study Drug | 5 Participants |
Secondary
Number of Doses Per a Week of Study Drug on a Prophylaxis Regimen
Number of doses per a week of study drug on a prophylaxis regimen was reported.
Time frame: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs
Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. The number analyzed was the number of participants with data available for analysis who treated with a prophylaxis regimen during the administration period.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Previously Treated Patients (PTPs) | Number of Doses Per a Week of Study Drug on a Prophylaxis Regimen | 2.0 Number of doses per a week | Standard Deviation 0.38 |
| Previously Untreated Patients (PUPs) | Number of Doses Per a Week of Study Drug on a Prophylaxis Regimen | 1.8 Number of doses per a week | Standard Deviation 0.41 |
Secondary
Number of Participants Who Experience Factor VIII Inhibition, Dermatitis Atopic or Eczema as an Adverse Event (AE)
Number of PTPs and PUPs who experienced factor VIII inhibition, dermatitis atopic or eczema as an AE related to development of inhibitors, shock or anaphylaxis was reported.
Time frame: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs
Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Previously Treated Patients (PTPs) | Number of Participants Who Experience Factor VIII Inhibition, Dermatitis Atopic or Eczema as an Adverse Event (AE) | Factor VIII Inhibition | 1 Participants |
| Previously Treated Patients (PTPs) | Number of Participants Who Experience Factor VIII Inhibition, Dermatitis Atopic or Eczema as an Adverse Event (AE) | Dermatitis Atopic | 0 Participants |
| Previously Treated Patients (PTPs) | Number of Participants Who Experience Factor VIII Inhibition, Dermatitis Atopic or Eczema as an Adverse Event (AE) | Eczema | 0 Participants |
| Previously Untreated Patients (PUPs) | Number of Participants Who Experience Factor VIII Inhibition, Dermatitis Atopic or Eczema as an Adverse Event (AE) | Factor VIII Inhibition | 3 Participants |
| Previously Untreated Patients (PUPs) | Number of Participants Who Experience Factor VIII Inhibition, Dermatitis Atopic or Eczema as an Adverse Event (AE) | Dermatitis Atopic | 1 Participants |
| Previously Untreated Patients (PUPs) | Number of Participants Who Experience Factor VIII Inhibition, Dermatitis Atopic or Eczema as an Adverse Event (AE) | Eczema | 1 Participants |