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

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.