Hemophilia B
Conditions
Brief summary
The purpose of this survey is to understand the following items observed in the actual clinical use of RIXUBIS. 1. Unexpected adverse drug reactions 2. Occurrence of adverse drug reactions in the actual clinical use 3. Factors that may affect safety and effectiveness 4. Occurrence of Factor IX (FIX) inhibitor development in patients with coagulation FIX deficiency 5. Safety and effectiveness for hemophilia B patients who received routine prophylactic therapy, on-demand therapy and perioperative therapy
Interventions
BIOLOGICALRIXUBIS
Sponsors
Takeda
Baxalta now part of Shire
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Patients with hemophilia B scheduled to receive treatment with RIXUBIS
Exclusion criteria
* Patients not administered RIXUBIS
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Hemostatic Effectiveness of Rixubis in Surgery-Perioperative and Postoperative Based on a 4-Point Ordinal Scale (Excellent, Moderate, Good, Poor) for Participants Who Received Perioperative Therapy During the Study | Assessed at the time of discharge from recovery room; and at 24 to 72 hours postoperatively | Number of participants in hemostatic effectiveness of Rixubis with a 4-point ordinal scale (Excellent, Moderate, Good, Poor) for perioperative therapy was reported in this outcome measure. The definition of each scale was following: Excellent; Amount of bleeding is smaller than expected. Good; Amount of bleeding is within the expected range. Fair; Amount of bleeding is greater than expected, with use of additional concomitant medication. Poor; Hemostasis difficulty. |
| Number of Doses to Treat A Bleed of Participants on An On-Demand Regimen | At bleed resolution throughout the study period of approximately 4 ½ years | Number of doses to treat a bleed of participants on an on-demand regimen was reported in this outcome measure. |
| Hemostatic Effectiveness of Rixubis for Participants on An On-Demand Regimen Based on a 4-Point Ordinal Scale (Excellent, Moderate, Good, Poor) | At bleed resolution throughout the study period of approximately 4 ½ years | Number of participants in hemostatic effectiveness of Rixubis with a 4-point ordinal scale (Excellent, Moderate, Good, Poor) for an on-demand regimen was reported in this outcome measure. The definition of each scale was following: Excellent; After a single infusion, complete disappearance of pain and objective decrease of bleeding symptom (swelling, tenderness, and increase in range of motion in musculoskeletal bleeding case) were observed. Good; After a single infusion, there were definitive relief of pain and improvement of bleeding symptom. Fair; After a single infusion, there were a probable or slight relief of pain and a mild improvement of bleeding signs. Poor; Improvement was not observed or symptom was aggravated. |
| Number of Participants Who Discontinued the Use of Nonacog Gamma (Genetical Recombination) | Throughout the study period, approximately 4 ½ years | Number of participants who discontinued the use of Nonacog Gamma (Genetical Recombination) was reported in this outcome measure. |
| Number of Participants Who Developed a Factor IX (FIX) Inhibitor | Throughout the study period, approximately 4 ½ years | Number of participants who developed a Factor IX (FIX) Inhibitor was reported in this outcome measure. |
| Annual Bleed Rate (ABR): Number of Times of Bleeding During the Study | Throughout the study period, approximately 4 ½ years | Annual bleed rate (ABR) was defined as the number of times of bleeding during the study. ABR was reported in this outcome measure. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Who Experienced Adverse Events of Thromboembolism | Throughout the study period, approximately 4 ½ years | Number of participants who experienced adverse events of thromboembolism was reported in this outcome measure. |
| Number of Participants Who Experienced Adverse Events of Shock or Anaphylaxis | Throughout the study period, approximately 4 ½ years | Number of participants who experienced adverse events of shock or anaphylaxis was reported in this outcome measure. |
Countries
Japan
Participant flow
Recruitment details
Participants took part in the survey at 2 investigative sites in Japan, from November 16, 2016 to May 11, 2022.
Pre-assignment details
Participants with a historical diagnosis of hemophilia B were enrolled. Participants received Nonacog Gamma (Genetical Recombination) as part of a routine medical care.
Participants by arm
| Arm | Count |
|---|---|
| Nonacog Gamma (Genetical Recombination) Nonacog Gamma (Genetical Recombination) is reconstituted with the attached 5 mL reconstitution diluent and administered by intravenous injection. Do not infuse any faster than 10 mL per minute. Normally, administer 50 international units per kg body weight per time. Adjust a dose based on a participant's condition. Participants received interventions as part of routine medical care. | 2 |
| Total | 2 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Protocol Violation | 4 |
Baseline characteristics
| Characteristic | Nonacog Gamma (Genetical Recombination) | — |
|---|---|---|
| Age, Categorical <=18 years | 0 Participants | — |
| Age, Categorical >=65 years | 0 Participants | — |
| Age, Categorical Between 18 and 65 years | 2 Participants | — |
| Race and Ethnicity Not Collected | — | — Participants |
| Region of Enrollment Japan | 2 Participants | — |
| Sex: Female, Male Female | 0 Participants | — |
| Sex: Female, Male Male | 2 Participants | — |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 2 |
| other Total, other adverse events | 1 / 2 |
| serious Total, serious adverse events | 0 / 2 |
Outcome results
Primary
Annual Bleed Rate (ABR): Number of Times of Bleeding During the Study
Annual bleed rate (ABR) was defined as the number of times of bleeding during the study. ABR was reported in this outcome measure.
Time frame: Throughout the study period, approximately 4 ½ years
Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. The number analyzed is the number of participants who received routine prophylactic therapy.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Nonacog Gamma (Genetical Recombination) | Annual Bleed Rate (ABR): Number of Times of Bleeding During the Study | 0 bleeds |
Primary
Hemostatic Effectiveness of Rixubis for Participants on An On-Demand Regimen Based on a 4-Point Ordinal Scale (Excellent, Moderate, Good, Poor)
Number of participants in hemostatic effectiveness of Rixubis with a 4-point ordinal scale (Excellent, Moderate, Good, Poor) for an on-demand regimen was reported in this outcome measure. The definition of each scale was following: Excellent; After a single infusion, complete disappearance of pain and objective decrease of bleeding symptom (swelling, tenderness, and increase in range of motion in musculoskeletal bleeding case) were observed. Good; After a single infusion, there were definitive relief of pain and improvement of bleeding symptom. Fair; After a single infusion, there were a probable or slight relief of pain and a mild improvement of bleeding signs. Poor; Improvement was not observed or symptom was aggravated.
Time frame: At bleed resolution throughout the study period of approximately 4 ½ years
Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. The number analyzed is the number of participants who received an on-demand regimen.
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Nonacog Gamma (Genetical Recombination) | Hemostatic Effectiveness of Rixubis for Participants on An On-Demand Regimen Based on a 4-Point Ordinal Scale (Excellent, Moderate, Good, Poor) | None | 0 Participants |
| Nonacog Gamma (Genetical Recombination) | Hemostatic Effectiveness of Rixubis for Participants on An On-Demand Regimen Based on a 4-Point Ordinal Scale (Excellent, Moderate, Good, Poor) | Excellent | 1 Participants |
| Nonacog Gamma (Genetical Recombination) | Hemostatic Effectiveness of Rixubis for Participants on An On-Demand Regimen Based on a 4-Point Ordinal Scale (Excellent, Moderate, Good, Poor) | Good | 0 Participants |
| Nonacog Gamma (Genetical Recombination) | Hemostatic Effectiveness of Rixubis for Participants on An On-Demand Regimen Based on a 4-Point Ordinal Scale (Excellent, Moderate, Good, Poor) | Moderate | 0 Participants |
Primary
Hemostatic Effectiveness of Rixubis in Surgery-Perioperative and Postoperative Based on a 4-Point Ordinal Scale (Excellent, Moderate, Good, Poor) for Participants Who Received Perioperative Therapy During the Study
Number of participants in hemostatic effectiveness of Rixubis with a 4-point ordinal scale (Excellent, Moderate, Good, Poor) for perioperative therapy was reported in this outcome measure. The definition of each scale was following: Excellent; Amount of bleeding is smaller than expected. Good; Amount of bleeding is within the expected range. Fair; Amount of bleeding is greater than expected, with use of additional concomitant medication. Poor; Hemostasis difficulty.
Time frame: Assessed at the time of discharge from recovery room; and at 24 to 72 hours postoperatively
Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. The number analyzed is the number of participants who received perioperative therapy. Number of participants who received perioperative therapy during this study was zero (N=0).
Primary
Number of Doses to Treat A Bleed of Participants on An On-Demand Regimen
Number of doses to treat a bleed of participants on an on-demand regimen was reported in this outcome measure.
Time frame: At bleed resolution throughout the study period of approximately 4 ½ years
Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. The number analyzed is the number of participants who received an on-demand regimen.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Nonacog Gamma (Genetical Recombination) | Number of Doses to Treat A Bleed of Participants on An On-Demand Regimen | 1 doses |
Primary
Number of Participants Who Developed a Factor IX (FIX) Inhibitor
Number of participants who developed a Factor IX (FIX) Inhibitor was reported in this outcome measure.
Time frame: Throughout the study period, approximately 4 ½ years
Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Nonacog Gamma (Genetical Recombination) | Number of Participants Who Developed a Factor IX (FIX) Inhibitor | 0 Participants |
Primary
Number of Participants Who Discontinued the Use of Nonacog Gamma (Genetical Recombination)
Number of participants who discontinued the use of Nonacog Gamma (Genetical Recombination) was reported in this outcome measure.
Time frame: Throughout the study period, approximately 4 ½ years
Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Nonacog Gamma (Genetical Recombination) | Number of Participants Who Discontinued the Use of Nonacog Gamma (Genetical Recombination) | 1 Participants |
Secondary
Number of Participants Who Experienced Adverse Events of Shock or Anaphylaxis
Number of participants who experienced adverse events of shock or anaphylaxis was reported in this outcome measure.
Time frame: Throughout the study period, approximately 4 ½ years
Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Nonacog Gamma (Genetical Recombination) | Number of Participants Who Experienced Adverse Events of Shock or Anaphylaxis | 0 Participants |
Secondary
Number of Participants Who Experienced Adverse Events of Thromboembolism
Number of participants who experienced adverse events of thromboembolism was reported in this outcome measure.
Time frame: Throughout the study period, approximately 4 ½ years
Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Nonacog Gamma (Genetical Recombination) | Number of Participants Who Experienced Adverse Events of Thromboembolism | 0 Participants |