Efficacy of emicizumab in patients with severe haemophilia A without factor VIII inhibitors in Germany: evaluation of real-life data documented by the smart medication eDiary.

IF 3.4 3区 医学 Q2 HEMATOLOGY
Therapeutic Advances in Hematology Pub Date : 2024-11-29 eCollection Date: 2024-01-01 DOI:10.1177/20406207241295653
Carmen Escuriola Ettingshausen, Wolfgang Eberl, Hermann Eichler, Ronald Fischer, Christina Hart, Katharina Holstein, Ralf Knöfler, Jürgen Kreutz, Caspar David Kühnöl, Wolfgang A Miesbach, Christian Pfrepper, Andreas Rösch, Ulrich J Sachs, Karolin Trautmann-Grill, Wolfgang Mondorf
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引用次数: 0

Abstract

Background: Systematically documented data on real-world use of emicizumab, a bispecific antibody factor (F)VIII mimetic, are still lacking in people with severe haemophilia A (PwSHA). Smart medication, a real-time, online platform, monitors treatment administration and outcomes for people with haemophilia A in Germany.

Objective: To evaluate annualised bleeding rates (ABRs) and annualised joint bleeding rates (AJBRs), using data documented in the smart medication eDiary, for PwSHA receiving emicizumab.

Design: Data for 97 PwSHA without FVIII inhibitors who started emicizumab treatment between 1 January 2018 and 31 March 2023, with >24 weeks of documentation after switching from FVIII replacement, were collected in the smart medication eDiary. Those with ⩾24 weeks of pre-emicizumab data were included for analysis 24 weeks before and after switching.

Methods: The primary objective was to evaluate ABR and AJBR for treated bleeds. The proportion of bleed-free participants was calculated and administration frequency for FVIII and emicizumab were collected. The mean dosing frequencies for FVIII replacement and emicizumab were also evaluated.

Results: The mean calculated ABR and AJBR were 0.64 and 0.39, respectively, after initiating emicizumab. For those with documentation before starting emicizumab (n = 58), ABR decreased by 79.6% and AJBR decreased by 90.8%. The proportion of bleed-free participants increased by 21.3%, and joint bleed-free participants increased by 18.2%. The median FVIII dosing frequency was every 3.5 days (n = 54; range: 1.0-20.8); median emicizumab dosing frequency was every 11.2 days (N = 97; range: 6.6-29.4).

Conclusion: Real-world data collected using the smart medication eDiary provide insights into efficacy outcomes after switching from FVIII replacement to emicizumab prophylaxis. Bleeds, including joint bleeds, decreased after switching.

emicizumab在德国无因子VIII抑制剂的严重血友病A患者中的疗效:对智能药物eDiary记录的真实数据的评估
背景:emicizumab是一种双特异性抗体因子(F)VIII模拟物,目前在严重血友病a (PwSHA)患者中仍缺乏实际使用emicizumab的系统记录数据。智能药物是一个实时在线平台,监测德国a型血友病患者的治疗管理和结果。目的:利用智能药物eDiary中记录的数据,评估接受emicizumab治疗的PwSHA的年化出血率(ABRs)和年化关节出血率(ajbr)。设计:在智能药物eDiary中收集了97例无FVIII抑制剂的PwSHA的数据,这些PwSHA在2018年1月1日至2023年3月31日期间开始接受emicizumab治疗,从FVIII替代药物切换后有bbbb24周的记录。那些具有小于24周emicizumab前数据的患者在转换前后24周被纳入分析。方法:主要目的是评价ABR和AJBR在治疗后出血中的作用。计算无出血参与者的比例,并收集FVIII和emicizumab的给药频率。还评估了FVIII替代和emicizumab的平均给药频率。结果:开始使用emicizumab后,计算的平均ABR和AJBR分别为0.64和0.39。对于开始使用emicizumab前有记录的患者(n = 58), ABR下降79.6%,AJBR下降90.8%。无出血的参与者比例增加了21.3%,关节无出血的参与者比例增加了18.2%。中位FVIII给药频率为每3.5天一次(n = 54;范围:1.0 - -20.8);中位半珠单抗给药频率为每11.2天一次(N = 97;范围:6.6 - -29.4)。结论:使用智能药物eDiary收集的真实世界数据提供了从FVIII替代到emicizumab预防转换后的疗效结果的见解。出血,包括关节出血,在转换后减少。
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来源期刊
CiteScore
4.30
自引率
0.00%
发文量
54
审稿时长
7 weeks
期刊介绍: Therapeutic Advances in Hematology delivers the highest quality peer-reviewed articles, reviews, and scholarly comment on pioneering efforts and innovative studies across all areas of hematology. The journal has a strong clinical and pharmacological focus and is aimed at clinicians and researchers in hematology, providing a forum in print and online for publishing the highest quality articles in this area.
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