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Switching to nonacog beta pegol in hemophilia B: Outcomes from a Canadian real-world, multicenter, retrospective
Davide Matino1, Alfonso Iorio1, Arun Keepanasseril1
1McMaster University Hamilton ON Canada.
Insights
Switching to nonacog beta pegol (N9-GP) for hemophilia B prophylaxis in Canada significantly reduced bleeding rates and factor consumption. This real-world study demonstrates N9-GP
Area of Science:
- Hematology
- Pharmacology
- Real-world evidence
Background:
- The Canadian Bleeding Disorders Registry (CBDR) collects data from 24 hemophilia treatment centers.
- Nonacog beta pegol (N9-GP) is a treatment for hemophilia B approved in Canada in 2018.
Purpose of the Study:
- To evaluate the effectiveness of N9-GP in managing hemophilia B in a real-world clinical setting.
- To assess treatment outcomes after switching to N9-GP from previous factor IX products.
Main Methods:
- Retrospective analysis of Canadian male patients (aged 7-72) with hemophilia B from the CBDR.
- Inclusion criteria required at least 6 months of N9-GP prophylaxis and 6 months of prior treatment data.
- Comparison of bleeding rates and factor consumption before and after switching to N9-GP.
Main Results:
- Forty-two patients were analyzed, with 62% having severe hemophilia B.
- Patients switching from rFIXFc and SHL rFIX showed reduced annualized bleeding rates on N9-GP (0.73 and 2.10, respectively).
- Factor consumption decreased with N9-GP compared to previous treatments (e.g., 2152 IU/kg vs 3018 IU/kg for SHL rFIX).
Conclusions:
- Real-world data suggest N9-GP provides effective bleeding control in hemophilia B patients.
- Switching to N9-GP resulted in lower factor consumption, regardless of the prior treatment.
- N9-GP demonstrates optimal bleeding control and reduced factor use in a real-world setting.
Background:
The Canadian Bleeding Disorders Registry (CBDR) captures data from 24 hemophilia treatment centers and patients directly. Nonacog beta pegol (N9-GP) was approved in Canada in 2018.
Objectives:
To assess treatment outcomes following switching to N9-GP in a real-world setting.
Methods:
CBDR data for Canadian male patients (aged 7-72 years) with hemophilia B receiving prophylactic N9-GP for ≥6 months as of March 31, 2021, were included. To allow comparison with the previously used products, only patients for whom data were available in the CBDR for at least 6 months before the switch to N9-GP were included in this retrospective analysis.
Results:
Forty-two patients were included in the analysis (total observation period: 148.0 patient-years). The distribution of disease severity was 62% severe, 36% moderate, 2% mild, with 62% of patients previously receiving recombinant factor IX-Fc-fusion protein (rFIXFc) and 38% previously receiving standard half-life (SHL) recombinant factor IX (rFIX). During a median follow-up period of 2.3 years on N9-GP prophylaxis, 232 bleeds were reported in 30 patients, 29% of patients reported zero bleeds. The median overall annualized bleeding rate on N9-GP was 0.73 for patients switching from rFIXFc (previously 1.44) and 2.10 for patients switching from SHL rFIX (previously 6.06). Median total annualized factor consumption (IU/kg) was lower with N9-GP than with previous SHL rFIX (2152 vs 3018) and previous rFIXFc (1766 vs 2278).
Conclusions:
Results from this first real-world study of N9-GP in patients with hemophilia B suggest optimal bleeding control with low factor consumption after switching to N9-GP, irrespective of the previous product.
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