Long-term outcomes with emicizumab prophylaxis for haemophilia A in China: A multicentre, large-cohort retrospective study.

Authors

Yuan Xu, Ying Wang, Ningling Wang, Liya He, Jing Ling, Mingwei Jin, Bingshou Xie, Shaohua Le, Meijuan Huang, Jie Peng, Hongbo Cheng, Jiao Jin, Li Zhou, Zeping Zhou, Can Gai, Yujing Zhang, Mei Sun, Xiaojing Li, Jie Yin, Yanli Cheng, Ailing Xue, Weiqun Xu, Hong Han, Fuhua Zhang, Weiguo Zhu, Shen He, Hua Zhou, Lei Zhang, Feng Xue, Renchi Yang

Published in

British journal of haematology. Jun 15, 2026. Epub Jun 15, 2026.

Abstract

Emicizumab is an established prophylactic therapy for haemophilia A (HA). However, real-world multicentre evidence on long-term outcomes, particularly in a resource-constrained setting, remains limited. We aimed to evaluate real-world outcomes in patients with HA receiving emicizumab prophylaxis across multiple centres with extended follow-up. We conducted a multicentre retrospective study of patients with HA who received emicizumab prophylaxis for ≥1 year across 25 centres. Outcomes included annualized bleeding rate (ABR), annualized joint bleeding rate (AJBR), bleeding events, target joint status and adverse events. A total of 132 patients (131 males and 1 female) were included, with a median age at switch of 2.0 years (interquartile range [IQR], 1.0-4.8). Over a median follow-up of 26 months (IQR: 16.0-40.0), the negative binomial regression model-based ABR for all bleeds was 0.81 (95% confidence interval [CI], 0.62-1.07). Across 12-month intervals, both calculated mean and negative binomial regression model-based AJBR remained between 0.1 and 0.3. Of the 74 baseline target joints, 95.9% (n = 71) resolved and no new target joints developed. Forty-two patients (31.8%) received factor concentrates during emicizumab treatment. Injection-site reactions occurred in 7.6% of patients, with no thromboembolic or fatal events observed. Long-term emicizumab prophylaxis provides sustained bleeding control, joint protection and a favourable safety profile.

PMID:
42298304
Bibliographic data and abstract were imported from PubMed on 16 Jun 2026.

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