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.