Current status of treatment and disease burden of a cohort of hemophilia B in China.

Objective: Hemophilia B is a rare X-chromosome linked hereditary bleeding disorder. Patients require lifelong treatment and it is costly, but there is a lack of research in China on the treatment and burden for this group. Our aim was to review the actual treatment pattern of hemophilia B patients in China, and describe the financial burden and other disease burden from the patient's perspective. Methods: Using data collected by the Beijing Hemophilia Home Care Center, descriptive statistics were made on the sociodemographic characteristics and treatment of patients. The annual drug costs were calculated according to the actual factor dosage and price. Results: During the study period, 29.9% of the patients only received on-demand treatment, while the rest of the patients received varying numbers of prophylaxis treatment. The total cost of clotting factors for 341 patients in one year was 16.0 million CNY ($2.5 million), with 46990.8 CNY ($7283.7) per patient. The drug cost of prophylaxis was significantly higher than that of on-demand treatment. The amount of prothrombin complex concentrates used by patients was the largest, more than 5 times of recombinant coagulation factor IX. Based on the average annual wage and average working time of Chinese employees in 2021, the average annual wage loss of HB patients reached 31544.2 CNY ($4889.4). The results of the questionnaire showed that 77.1% and 65.3% of patients had chronic pain and acute pain of different frequencies. Conclusion: The level of prophylaxis for Chinese patients is low; safer and more effective recombinant drugs are not widely available. Patients also face a high burden of drug costs, as well as indirect costs that cannot be underestimated. Therefore, continued efforts are needed to improve the quality of life of patients by reducing their financial burden and promote standardized treatment.