Escuriola Ettingshausen Carmen, Eberl Wolfgang, Eichler Hermann, Fischer Ronald, Hart Christina, Holstein Katharina, Knöfler Ralf, Kreutz Jürgen, Kühnöl Caspar David, Miesbach Wolfgang A, Pfrepper Christian, Rösch Andreas, Sachs Ulrich J, Trautmann-Grill Karolin, Mondorf Wolfgang
HZRM Haemophilia Centre Rhine, Main, Stresemannallee, D-60596 Frankfurt, Germany.
Department of Pediatrics, Klinikum Braunschweig, Braunschweig, Germany.
Ther Adv Hematol. 2024 Nov 29;15:20406207241295653. doi: 10.1177/20406207241295653. eCollection 2024.
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.
To evaluate annualised bleeding rates (ABRs) and annualised joint bleeding rates (AJBRs), using data documented in the smart medication eDiary, for PwSHA receiving emicizumab.
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.
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.
The mean calculated ABR and AJBR were 0.64 and 0.39, respectively, after initiating emicizumab. For those with documentation before starting emicizumab ( = 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 ( = 54; range: 1.0-20.8); median emicizumab dosing frequency was every 11.2 days ( = 97; range: 6.6-29.4).
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.
关于双特异性抗体因子（F）VIII模拟物emicizumab在重度A型血友病患者（PwSHA）中的真实世界使用的系统记录数据仍然缺乏。智能药物是一个实时在线平台，可监测德国A型血友病患者的治疗给药情况和治疗结果。
使用智能药物电子日记中记录的数据，评估接受emicizumab治疗的PwSHA的年化出血率（ABR）和年化关节出血率（AJBR）。
在智能药物电子日记中收集了97例无FVIII抑制剂且于2018年1月1日至2023年3月31日开始emicizumab治疗、从FVIII替代治疗转换后有>24周记录的PwSHA的数据。纳入emicizumab治疗前有⩾24周数据的患者，在转换前和转换后24周进行分析。
主要目的是评估治疗性出血的ABR和AJBR。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替代治疗和emicizumab的平均给药频率。
开始使用emicizumab后，计算得出的平均ABR和AJBR分别为0.64和0.39。对于在开始emicizumab治疗前有记录的患者（n = 58），ABR下降了79.6%，AJBR下降了90.8%。无出血参与者的比例增加了21.3%，无关节出血参与者增加了18.2%。FVIII的中位给药频率为每3.5天一次（n = 54；范围：1.0 - 20.8）；emicizumab的中位给药频率为每11.2天一次（n = 97；范围：6.6 - 29.4）。
使用智能药物电子日记收集的真实世界数据为从FVIII替代治疗转换为emicizumab预防后的疗效结果提供了见解。转换后出血，包括关节出血，有所减少。
