Hemophilia B
Conditions
Keywords
BeneFIX Regulatory Post Marketing Commitment Plan
Brief summary
The survey is intended to investigate the following matters, etc. under the actual use status after marketing in all patients who are administered this drug for a certain period of time after the launch. 1. Occurrence status of adverse events 2. Factors that may influence the safety 3. Efficacy In addition, the following occurrence statuses will be investigated as priority items of the survey: Incidence rate of inhibitor, reduction in drug, efficacy, Allergic reaction, and Thrombosis.
Detailed description
Patients with hemophilia B (congenital blood coagulation factor IX deficiency) who are administered this drug. Patients with hemophilia B are either patients for whom treatment was already started with a blood coagulation factor IX product (Previously treated patients: hereinafter, PTPs) or patients who have no history of treatment with a blood coagulation factor IX product in the past and for whom treatment will be started with this drug for the first time (Previously untreated patients: hereinafter, PUPs). The definition of PUPs in this survey is Patients in whom the total number of days of administration of a blood coagulation factor IX product other than this drug before administration of this drug was 3 exposure days (actual number of days of administration: hereinafter, EDs) or less.
Interventions
DRUGNonacog Alfa (Genetical Recombination)
Patients with hemophilia B (congenital blood coagulation factor IX deficiency) who are administered this drug. Patients with hemophilia B are either patients for whom treatment was already started with a blood coagulation factor IX product (Previously treated patients: hereinafter, PTPs) or patients who have no history of treatment with a blood coagulation factor IX product in the past and for whom treatment will be started with this drug for the first time (Previously untreated patients: hereinafter, PUPs). The definition of PUPs in this survey is Patients in whom the total number of days of administration of a blood coagulation factor IX product other than this drug before administration of this drug was 3 exposure days (actual number of days of administration: hereinafter, EDs) or less.
Sponsors
Pfizer
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* All patients with hemophilia B scheduled to receive treatment with BeneFIX will be eligible for the surveillance. * No patient will be excluded because of prior inhibitor history; however, complete patient history and demographics will be collected.
Exclusion criteria
* Patients not administered BeneFIX.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Bleeding Episodes (Annual Bleeding Event Rate) During Periodic Replacement Therapy | 2 years for PTPs, 1 year for PUPs | Annual bleeding rate (ABR) was calculated as the number of total bleeding events occured during prophylaxis period devided by the period (events/year). ABR for other replacement treatment period was also calculated to evaluate the differences between types of treatment. If the period used for ABR calculation was less or equal than 7 days, the relevant data was regarded as missing. |
| Number of Administrations Required for Hemostasis for Bleeding Events | 2 years for PTPs, 1 year for PUPs | Mean number of administrations for hemostasis in replacement therapy for bleeding events. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Subjective Evaluation of Each Therapeutic Administration for Bleeding Episodes | 2 years for PTPs, 1 year for PUPs | Propotion of subjects whose physicians evaluated the effect of BeneFIX for bleeding episodes as excellent or good was calculated. |
Countries
Japan
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| BeneFIX (Nonacog Alfa) Participants, who received BeneFIX injection intravenously as indicated in the approved local product document, were observed for periods of 1 year for PTPs and 2 years for PUPs. The dosage can be adjusted as per physician's discretion. | 312 |
| Total | 312 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | No Info. After 1st Day, No Drug Admin. | 1 |
| Overall Study | Safety Unassessable, No Drug Admin. | 1 |
Baseline characteristics
| Characteristic | BeneFIX (Nonacog Alfa) |
|---|---|
| Age, Customized <15 years | 124 Participants |
| Age, Customized ≥15 years <65 years | 179 Participants |
| Age, Customized ≥65 years | 8 Participants |
| Age, Customized Unknown | 1 Participants |
| Hemophilic Arthropathy No | 179 Participants |
| Hemophilic Arthropathy Yes | 133 Participants |
| History of Inhibitor No | 306 Participants |
| History of Inhibitor Yes | 6 Participants |
| Number of Bleeding in the Past 1 Year ˂10 Times | 194 Participants |
| Number of Bleeding in the Past 1 Year ≥10 to ˂50 Times | 73 Participants |
| Number of Bleeding in the Past 1 Year ≥50 Times | 8 Participants |
| Number of Bleeding in the Past 1 Year Unknown | 37 Participants |
| Previously Treated History Others | 3 Participants |
| Previously Treated History PTPs | 281 Participants |
| Previously Treated History PUPs | 28 Participants |
| Severity Mild | 43 Participans |
| Severity Moderate | 98 Participans |
| Severity Severe | 156 Participans |
| Severity Unknown | 15 Participans |
| Sex: Female, Male Female | 4 Participants |
| Sex: Female, Male Male | 308 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 35 / 312 |
| serious Total, serious adverse events | 5 / 312 |
Outcome results
Primary
Number of Administrations Required for Hemostasis for Bleeding Events
Mean number of administrations for hemostasis in replacement therapy for bleeding events.
Time frame: 2 years for PTPs, 1 year for PUPs
Population: Efficacy analysis set who recieved at least one administration and had at least one efficacy evaluation was used for this analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| BeneFIX (Nonacog Alfa) | Number of Administrations Required for Hemostasis for Bleeding Events | 1.8 administrations | Full Range 4.18 |
Primary
Number of Bleeding Episodes (Annual Bleeding Event Rate) During Periodic Replacement Therapy
Annual bleeding rate (ABR) was calculated as the number of total bleeding events occured during prophylaxis period devided by the period (events/year). ABR for other replacement treatment period was also calculated to evaluate the differences between types of treatment. If the period used for ABR calculation was less or equal than 7 days, the relevant data was regarded as missing.
Time frame: 2 years for PTPs, 1 year for PUPs
Population: Efficacy analysis set who recieved at least one administration and had at least one efficacy evaluation was used for this analysis.
| Arm | Measure | Value (MEDIAN) | Dispersion |
|---|---|---|---|
| BeneFIX (Nonacog Alfa) | Number of Bleeding Episodes (Annual Bleeding Event Rate) During Periodic Replacement Therapy | 2.00 Bleeding events/year | Inter-Quartile Range 11.29 |
Secondary
Subjective Evaluation of Each Therapeutic Administration for Bleeding Episodes
Propotion of subjects whose physicians evaluated the effect of BeneFIX for bleeding episodes as excellent or good was calculated.
Time frame: 2 years for PTPs, 1 year for PUPs
Population: Efficacy analysis set who recieved at least one administration and had at least one efficacy evaluation was used for this analysis.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| BeneFIX (Nonacog Alfa) | Subjective Evaluation of Each Therapeutic Administration for Bleeding Episodes | 93.7 percentage |