Hemophilia A
Conditions
Brief summary
This study is to describe the safety and efficacy of Xyntha® during the usual care setting.
Detailed description
non probability sample
Interventions
DRUGXyntha : coagulation factor IIIV (recombinant)
Xyntha will be administered according to physician's discretion.
Sponsors
Pfizer
Study design
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Patients or legally authorized representatives of pediatric patients agree to provide written informed consent form (data privacy statement). * Pediatric and adult patients who have been treated with Xyntha for Hemophilia A from first approved date by KFDA, or who are planned to be newly prescribed Xyntha.
Exclusion criteria
* Patients with a known history of hypersensitivity to original or reformulated Xyntha or any component of the product. * Patients with a known history of hypersensitivity to hamster protein. * Patients participating in an interventional trial of any investigational drug or device.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Percentage of Participants by Family History of Factor VIII Inhibitor | 4 years |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Xyntha Infusions Used to Treat Each New Bleed for On-Demand Therapy in Participants Less Than (<)18 Years of Age | 4 years | — |
| Number of Xyntha Infusions Used to Treat Each New Bleed for On-Demand Therapy in Participants Greater Than or Equal to (≥) 18 Years of Age | 4 years | — |
| Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy (All Participants) | 4 years | Response categories were excellent, good, moderate, or no response. |
| Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants <18 Years of Age | 4 years | Response categories were excellent, good, moderate, or no response. |
| Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants ≥18 Years of Age | 4 years | Response categories were excellent, good, moderate, and no response. |
| Number of Participants With Less-Than-Expected Therapeutic Effect (LETE) for On-Demand Therapy | 4 years | Less than expected therapeutic effect was defined as a 'no response' rating after each of two successive infusions less than or equl to (≤) 24 hours of on-demand therapy. |
| Average Infusion Dose Per Bleeding for On-Demand Therapy (All Participants) | 4 years | — |
| Average Infusion Dose Per Bleeding for On-Demand Therapy in Participants <18 Years of Age | 4 years | — |
| Average Infusion Dose Per Bleeding for On-Demand Therapy in Participants ≥18 Years of Age | 4 years | — |
| Number of Xyntha Infusions Used to Treat Each New Bleed for On-Demand Therapy (All Participants) | 4 years | — |
| Annualized Bleeding Rates During Prophylaxis | 4 years | Annualized bleeding rate defined as total number of breakthrough bleeds within 48 hours (for prophylaxis purpose) divided by (/) \[(total period of date of bleeding)/365.25)\] |
| Number of Participants With LETE Bleeds Within 48 Hours of a Preventive/Prophylaxis Dose of Xyntha | 4 years | Less than expected therapeutic effect for prophylaxis therapy defined as breakthrough (spontaneous/non-traumatic) bleed wtihin 48 hours of prophylaxis infusion. |
| Average Infusion Dose During Prophylaxis (All Participants) | 4 years | — |
| Average Infusion Dose During Prophylaxis in Participants <18 Years of Age | 4 years | — |
| Average Infusion Dose During Prophylaxis in Participants ≥18 Years of Age | 4 years | — |
| Total Factor Consumption for On-Demand Therapy and During Prophylaxis (All Participants) | 4 years | — |
| Total Factor Consumption for On-Demand Therapy and During Prophylaxis in Participants <18 Years of Age | 4 years | — |
| Total Factor Consumption for On-Demand Therapy and During Prophylaxis in Participants ≥18 Years of Age | 4 years | — |
| Percentage of Participants Experiencing Hemorrhages During Prophylaxis | 4 years | — |
Countries
South Korea
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Xyntha Non-interventional, observational study of participants who were prescribed and received at least 1 dose of Xyntha 250, 500, 1000, or 2000 IU. | 42 |
| Total | 42 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Other | 34 |
Baseline characteristics
| Characteristic | Xyntha |
|---|---|
| Age, Continuous | 36.4 years STANDARD_DEVIATION 14.5 |
| Sex: Female, Male Female | 2 Participants |
| Sex: Female, Male Male | 40 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 10 / 42 |
| serious Total, serious adverse events | 0 / 42 |
Outcome results
Primary
Percentage of Participants by Family History of Factor VIII Inhibitor
Time frame: 4 years
Population: All enrolled participants.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Xyntha | Percentage of Participants by Family History of Factor VIII Inhibitor | No | 100.0 percentage of participants |
| Xyntha | Percentage of Participants by Family History of Factor VIII Inhibitor | Yes | 0 percentage of participants |
Secondary
Annualized Bleeding Rates During Prophylaxis
Annualized bleeding rate defined as total number of breakthrough bleeds within 48 hours (for prophylaxis purpose) divided by (/) \[(total period of date of bleeding)/365.25)\]
Time frame: 4 years
Population: Data were not analyzed as information on breakthrough bleeds within 48 hours was not collected.
Secondary
Average Infusion Dose During Prophylaxis (All Participants)
Time frame: 4 years
Population: All enrolled participants; only participants with nonmissing data were included in the analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xyntha | Average Infusion Dose During Prophylaxis (All Participants) | 1926.70 IU | Standard Deviation 532.9 |
Secondary
Average Infusion Dose During Prophylaxis in Participants <18 Years of Age
Time frame: 4 years
Population: All enrolled participants; only participants \<18 years were included in the analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xyntha | Average Infusion Dose During Prophylaxis in Participants <18 Years of Age | 1243.75 IU | Standard Deviation 894.51 |
Secondary
Average Infusion Dose During Prophylaxis in Participants ≥18 Years of Age
Time frame: 4 years
Population: All enrolled participants; only participants ≥18 years with nonmissing data were included in the analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xyntha | Average Infusion Dose During Prophylaxis in Participants ≥18 Years of Age | 2000.16 IU | Standard Deviation 437.59 |
Secondary
Average Infusion Dose Per Bleeding for On-Demand Therapy (All Participants)
Time frame: 4 years
Population: All enrolled participants; only participants who received on-demand therapy were included in the analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xyntha | Average Infusion Dose Per Bleeding for On-Demand Therapy (All Participants) | 1997.00 IU | Standard Deviation 892.77 |
Secondary
Average Infusion Dose Per Bleeding for On-Demand Therapy in Participants <18 Years of Age
Time frame: 4 years
Population: All enrolled participants; only participants \<18 years who received on-demand therapy were included in the analysis.
| Arm | Measure | Value (MEDIAN) | Dispersion |
|---|---|---|---|
| Xyntha | Average Infusion Dose Per Bleeding for On-Demand Therapy in Participants <18 Years of Age | 725.00 IU | Full Range 892.77 |
Secondary
Average Infusion Dose Per Bleeding for On-Demand Therapy in Participants ≥18 Years of Age
Time frame: 4 years
Population: All enrolled participants; only participants ≥18 years who received on-demand therapy were included in the analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xyntha | Average Infusion Dose Per Bleeding for On-Demand Therapy in Participants ≥18 Years of Age | 2315.00 IU | Standard Deviation 623 |
Secondary
Number of Participants With Less-Than-Expected Therapeutic Effect (LETE) for On-Demand Therapy
Less than expected therapeutic effect was defined as a 'no response' rating after each of two successive infusions less than or equl to (≤) 24 hours of on-demand therapy.
Time frame: 4 years
Population: Data were not analyzed as no participants experienced rating of 'no response'.
Secondary
Number of Participants With LETE Bleeds Within 48 Hours of a Preventive/Prophylaxis Dose of Xyntha
Less than expected therapeutic effect for prophylaxis therapy defined as breakthrough (spontaneous/non-traumatic) bleed wtihin 48 hours of prophylaxis infusion.
Time frame: 4 years
Population: Data were not analyzed as information on breakthrough bleeds within 48 hours was not collected.
Secondary
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy (All Participants)
Response categories were excellent, good, moderate, or no response.
Time frame: 4 years
Population: All enrolled participants; only participants who received on-demand therapy were included in the analysis.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Xyntha | Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy (All Participants) | Excellent | 5 Number of responses |
| Xyntha | Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy (All Participants) | Good | 2 Number of responses |
| Xyntha | Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy (All Participants) | Moderate | 0 Number of responses |
| Xyntha | Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy (All Participants) | No response | 0 Number of responses |
Secondary
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants <18 Years of Age
Response categories were excellent, good, moderate, or no response.
Time frame: 4 years
Population: All enrolled participants; only participants \<18 years who received on-demand therapy were included in the analysis.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Xyntha | Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants <18 Years of Age | Excellent | 1 number of responses |
| Xyntha | Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants <18 Years of Age | Good | 0 number of responses |
| Xyntha | Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants <18 Years of Age | Moderate | 0 number of responses |
| Xyntha | Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants <18 Years of Age | No response | 0 number of responses |
Secondary
Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants ≥18 Years of Age
Response categories were excellent, good, moderate, and no response.
Time frame: 4 years
Population: All enrolled participants; only participants ≥18 years who receive don-demand therapy were included in the analysis.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Xyntha | Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants ≥18 Years of Age | Excellent | 4 number of responses |
| Xyntha | Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants ≥18 Years of Age | Good | 2 number of responses |
| Xyntha | Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants ≥18 Years of Age | Moderate | 0 number of responses |
| Xyntha | Number of Responses by Type of Response for All Xyntha Infusions for Treatment of a Bleed for On-Demand Therapy in Participants ≥18 Years of Age | No response | 0 number of responses |
Secondary
Number of Xyntha Infusions Used to Treat Each New Bleed for On-Demand Therapy (All Participants)
Time frame: 4 years
Population: All enrolled participants; only participants who received on-demand therapy were included in the analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xyntha | Number of Xyntha Infusions Used to Treat Each New Bleed for On-Demand Therapy (All Participants) | 1.40 infusions | Standard Deviation 0.55 |
Secondary
Number of Xyntha Infusions Used to Treat Each New Bleed for On-Demand Therapy in Participants Greater Than or Equal to (≥) 18 Years of Age
Time frame: 4 years
Population: All enrolled participants; participants aged ≥18 years who received on-demand therapy were included in the analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xyntha | Number of Xyntha Infusions Used to Treat Each New Bleed for On-Demand Therapy in Participants Greater Than or Equal to (≥) 18 Years of Age | 1.50 infusions | Standard Deviation 0.58 |
Secondary
Number of Xyntha Infusions Used to Treat Each New Bleed for On-Demand Therapy in Participants Less Than (<)18 Years of Age
Time frame: 4 years
Population: All enrolled participants; only participants aged \<18 years who received on-demand therapy were included in the analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xyntha | Number of Xyntha Infusions Used to Treat Each New Bleed for On-Demand Therapy in Participants Less Than (<)18 Years of Age | 1.00 infusions | Standard Deviation 0 |
Secondary
Percentage of Participants Experiencing Hemorrhages During Prophylaxis
Time frame: 4 years
Population: All enrolled participants; n (number) equals (=) number of participants with nonmissing values
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Xyntha | Percentage of Participants Experiencing Hemorrhages During Prophylaxis | All participants (n=41) | 0 percentage of participants |
| Xyntha | Percentage of Participants Experiencing Hemorrhages During Prophylaxis | Participants <18 years (n=4) | 0 percentage of participants |
| Xyntha | Percentage of Participants Experiencing Hemorrhages During Prophylaxis | Participants ≥18 years (n=37) | 0 percentage of participants |
Secondary
Total Factor Consumption for On-Demand Therapy and During Prophylaxis (All Participants)
Time frame: 4 years
Population: All enrolled participants; participants were excluded from the analysis if total number of infusions for prophylaxis was unknown.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xyntha | Total Factor Consumption for On-Demand Therapy and During Prophylaxis (All Participants) | 66371.00 IU | Standard Deviation 60985.45 |
Secondary
Total Factor Consumption for On-Demand Therapy and During Prophylaxis in Participants <18 Years of Age
Time frame: 4 years
Population: All enrolled participants; only participants \<18 years were included in the analysis and participants were excluded from the analysis if total number of infusions for prophylaxis was unknown.
| Arm | Measure | Value (MEDIAN) | Dispersion |
|---|---|---|---|
| Xyntha | Total Factor Consumption for On-Demand Therapy and During Prophylaxis in Participants <18 Years of Age | 2900.00 IU | Full Range 894.51 |
Secondary
Total Factor Consumption for On-Demand Therapy and During Prophylaxis in Participants ≥18 Years of Age
Time frame: 4 years
Population: All enrolled participants; only participants ≥18 years were included in the analysis; participants were excluded from the analysis if total number of infusions for prophylaxis was unknown.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xyntha | Total Factor Consumption for On-Demand Therapy and During Prophylaxis in Participants ≥18 Years of Age | 70104.59 IU | Standard Deviation 60704.87 |