Matino Davide, Iorio Alfonso, Keepanasseril Arun, Germini Federico, Caillaud Alexandre, Carcao Manuel, Hews-Girard Julia, Iserman Emma, James Paula, Lee Adrienne, Phua Chai W, Sun Haowei Linda, Teitel Jerome, Poon Man-Chiu
McMaster University Hamilton ON Canada.
BioPharm Medical Affairs Novo Nordisk Canada Inc. Mississauga ON Canada.
Res Pract Thromb Haemost. 2022 Mar 31;6(3):e12661. doi: 10.1002/rth2.12661. eCollection 2022 Mar.
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.
To assess treatment outcomes following switching to N9-GP in a real-world setting.
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.
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).
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.
加拿大出血性疾病登记处（CBDR）直接收集来自24个血友病治疗中心和患者的数据。非阿考糖β聚乙二醇（N9-GP）于2018年在加拿大获批。
评估在实际临床环境中转换为N9-GP后的治疗效果。
纳入截至2021年3月31日在加拿大接受预防性N9-GP治疗≥6个月的7至72岁男性B型血友病患者的CBDR数据。为了与先前使用的产品进行比较，本回顾性分析仅纳入了在转换为N9-GP前至少6个月在CBDR中有可用数据的患者。
42例患者纳入分析（总观察期：148.0患者年）。疾病严重程度分布为62%重度、36%中度、2%轻度，62%的患者先前接受重组因子IX-Fc融合蛋白（rFIXFc），38%的患者先前接受标准半衰期（SHL）重组因子IX（rFIX）。在接受N9-GP预防的中位随访期2.3年期间，30例患者报告了232次出血，29%的患者报告零出血。从rFIXFc转换的患者，N9-GP的中位总体年化出血率为0.73（先前为1.44）；从SHL rFIX转换的患者，N9-GP的中位总体年化出血率为2.10（先前为6.06）。N9-GP的中位年化总因子消耗量（IU/kg）低于先前的SHL rFIX（2152对3018）和先前的rFIXFc（1766对2278）。
这项关于N9-GP在B型血友病患者中的首次真实世界研究结果表明，转换为N9-GP后，无论先前使用何种产品，均可实现最佳出血控制且因子消耗量低。
