The effectiveness and safety of various treatments for hemophilia in China: a literature review and evidence mapping

To evaluate the safety and effectiveness of treatments for hemophilia in China through a literature review and evidence mapping, we systematically searched PubMed, Web of Science, Cochrane Library, CNKI, Wanfang, and CBM. Descriptive statistics were used for data analysis in this evidence mapping and the review concluded on 21 April 2025. Results were compiled in tabular or graphical form. This review included 49 studies (two RCTs and 47 observational studies), involving 2274 patients, 41 articles focusing on hemophilia A, seven on hemophilia B, and one on both. The average (mean or median) age of subjects within the individual studies ranged from 5.77 to 34.6 years. This study revealed a decrease in both annualized bleeding rate (ABR) and annualized joint bleeding rate (AJBR) following prophylaxis treatment and immune tolerance induction (ITI) treatment. The reduction in target joint count post-treatment aligns with previous research indicating successful hemostasis in most people with hemophilia after alternative therapies; long-term follow-up studies consistently showed improved imaging scores at various dosage levels, indicating disease amelioration. Additionally, our findings suggest enhanced quality of life for people with hemophilia following treatment. Alternative therapies, encompassing prophylaxis therapy, on-demand treatment, and ITI therapy, demonstrated both efficacy and safety in the management of hemophilia among Chinese people with hemophilia.