法国B-SURE研究中重组FIX Fc在B型血友病中的真实世界使用情况及疗效

Real-world usage and effectiveness of recombinant factor IX Fc in haemophilia B from the B-SURE study in France.

作者信息

Chambost Hervé, Repessé Yohann, Genre-Volot Fabienne, Desprez Dominique, Castet Sabine Marie, Vanderbecken Stéphane, Zidi Meriem, Gandossi Corinne, Nüesch Eveline, Palmborg Helena, Santagostino Elena

机构信息

AP-HM, Department of Paediatric Haematology, Immunology and Oncology, La Timone Children's Hospital and Aix Marseille University, C2VN, Marseille, France.

Haemophilia Treatment Centre, University Hospital of Caen, Caen, France.

出版信息

Ther Adv Hematol. 2025 Jan 26;16:20406207241311535. doi: 10.1177/20406207241311535. eCollection 2025.

DOI:10.1177/20406207241311535

PMID:39872008

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11770721/

Abstract

BACKGROUND

More real-world data are needed to complement existing phase III studies on the efficacy and safety of recombinant factor IX Fc fusion protein (rFIXFc) in people with haemophilia B.

OBJECTIVES

We report final data from the B-SURE study, evaluating the real-world usage and effectiveness of rFIXFc in France.

METHODS

Previously treated patients (all ages/severities) received on-demand or prophylactic rFIXFc during B-SURE. Annualised bleeding rate (ABR), injection frequency (IF) and factor consumption (FC) were prospectively evaluated for patients on rFIXFc prophylaxis (primary endpoints). Six months of retrospective factor IX (FIX) data were collected for comparison; patients with ⩾3 months of treatment pre- and post-switch to rFIXFc were analysed.

DESIGN

B-SURE was a 24-month, prospective, non-interventional, real-world study across haemophilia treatment centres in France.

RESULTS

Ninety-one male patients enrolled across 21 centres (34% <18 years, 89% severe haemophilia B). Eighty-four patients received prophylaxis at rFIXFc initiation; mean prospective observation period was 21.5 months. Sixty-eight of 84 patients had prior FIX prophylaxis; on rFIXFc prophylaxis, these patients achieved low median ABR (1.2), IF (47.45 injections/year) and mean FC (2844 IU/kg/year). Compared with previous FIX, mean ABR was reduced by 40% ( = 63); mean IF and FC were reduced by 38.20 injections/year and 1008 IU/kg/year ( = 57). In patients with prior FIX on-demand ( = 15), mean ABR reduced by 84% on rFIXFc prophylaxis ( = 14), mean IF reduced by 2.13 injections/year and mean FC increased by 381.8 IU/kg/year ( = 15). Most physicians and patients were satisfied/highly satisfied with rFIXFc prophylaxis. rFIXFc was well tolerated with no new safety concerns.

CONCLUSION

Findings support the safety and effectiveness of rFIXFc, with reduced IF and FC while maintaining/improving bleed protection. NCT03655340.

摘要

背景

需要更多真实世界的数据来补充现有的关于重组因子IX Fc融合蛋白（rFIXFc）在B型血友病患者中的疗效和安全性的III期研究。

目的

我们报告了B-SURE研究的最终数据，评估了rFIXFc在法国的真实世界使用情况和有效性。

方法

既往接受过治疗的患者（所有年龄/严重程度）在B-SURE研究期间接受按需或预防性rFIXFc治疗。对接受rFIXFc预防治疗的患者前瞻性评估年化出血率（ABR）、注射频率（IF）和因子消耗（FC）（主要终点）。收集六个月的回顾性因子IX（FIX）数据进行比较；分析了在转换为rFIXFc前后有≥3个月治疗时间的患者。

设计

B-SURE是一项在法国血友病治疗中心进行的为期24个月的前瞻性、非干预性真实世界研究。

结果

21个中心共纳入91例男性患者（34%＜18岁，89%为重度B型血友病）。84例患者在开始使用rFIXFc时接受预防治疗；平均前瞻性观察期为21.5个月。84例患者中有68例曾接受过FIX预防治疗；在接受rFIXFc预防治疗时，这些患者的ABR中位数较低（1.2）、IF（每年47.45次注射）和平均FC（每年2844IU/kg）。与之前使用FIX相比，平均ABR降低了40%（P = 63）；平均IF和FC分别降低了每年38.20次注射和每年1008IU/kg（P = 57）。在之前接受按需FIX治疗的患者中（n = 15），接受rFIXFc预防治疗时平均ABR降低了84%（P = 14），平均IF降低了每年2.13次注射，平均FC增加了每年381.8IU/kg（P = 15）。大多数医生和患者对rFIXFc预防治疗感到满意/非常满意。rFIXFc耐受性良好，没有新的安全问题。