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

Summary 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.