Disease Burden and Treatment Patterns Amongst Patients with Haemophilia A in China: A Retrospective Database Analysis Study.

Introduction: Despite concerted efforts of healthcare agencies, haemophilia treatment coverage remains inadequate in China. This real-world study was conducted to understand patient characteristics, clinical and economic burden, and treatment patterns amongst patients with haemophilia A in China with the aim of improving patient outcomes and quality of life.

Methods: Two data sources, namely the National Haemophilia Registry and Institute of Haematology and Blood Diseases Hospital, were used to analyse the disease burden, treatment patterns and economic burden of haemophilia A in China. The economic burden was assessed from 2017 to 2019.

Results: Overall, 3164 male patients with haemophilia A (mean age 21.5 years) were analysed. Almost half (48.3%) of the patients were children or adolescents; amongst them, 13.5% were enrolled from Guangdong and 10.7% from Hebei. Furthermore, 58.9% of the patients had severe haemophilia A. The most common sites for the first bleeding and bleeding during disease course were skin/mucous membrane (40.0%) and joints (77.9%), respectively. Plasma-derived factor VIII constituted the most frequently used therapy (45.8% of patients) and only 30.1% of patients used a prophylactic treatment. The hospitalisation rate increased from 8% in 2017 to 21% in 2019, whereas 63% to 75% of patients had outpatient visits during each year. The total annual cost of treating haemophilia (hospitalisation plus outpatient) including the cost of FVIII replacement per patient was 57,439.4 CNY.

Conclusion: Suboptimal disease management and low utilisation of prophylaxis are the main challenges for patients with haemophilia A in China. Therefore, continued efforts to improve disease awareness and treatment accessibility are required.