Dataset Information

Switching to nonacog beta pegol in hemophilia B: Outcomes from a Canadian real-world, multicenter, retrospective study.

ABSTRACT:

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.

SUBMITTER: Matino D

PROVIDER: S-EPMC8971162 | biostudies-literature | 2022 Mar

REPOSITORIES: biostudies-literature

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Publications

Switching to nonacog beta pegol in hemophilia B: Outcomes from a Canadian real-world, multicenter, retrospective study.

Matino Davide D Iorio Alfonso A Keepanasseril Arun A Germini Federico F Caillaud Alexandre A Carcao Manuel M Hews-Girard Julia J Iserman Emma E James Paula P Lee Adrienne A Phua Chai W CW Sun Haowei Linda HL Teitel Jerome J Poon Man-Chiu MC

Research and practice in thrombosis and haemostasis 20220331 3

<h4>Background</h4>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.<h4>Objectives</h4>To assess treatment outcomes following switching to N9-GP in a real-world setting.<h4>Methods</h4>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 pr ...[more]

PMID: 35386274

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Dataset Information

Switching to nonacog beta pegol in hemophilia B: Outcomes from a Canadian real-world, multicenter, retrospective study.

Background

Objectives

Methods

Results

Conclusions

Publications

Switching to nonacog beta pegol in hemophilia B: Outcomes from a Canadian real-world, multicenter, retrospective study.

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