OBJECTIVES: Hemophilia is a rare congenital bleeding disorder which has a substantial negative impact on patients’ health-related quality of life (HRQOL). Currently, it is estimated approximately 39,000 patients live with hemophilia in China. However, little is known about Chinese hemophilia patients’ HRQOL. The aim of this study was to measure the HRQOL of patient with hemophilia in China.

METHODS: A nationwide, online, cross-sectional survey was conducted with collaboration with the biggest hemophilia patient organization in China in July 2021. Data on demographic characteristics, health resource utilization, clinical outcomes and HRQOL were collected. HRQOL was measured with the EQ-5D-5L questionnaires completed by patients or their caregivers. Variance analysis and multivariate linear regression model were employed to analyze HRQOL data.

RESULTS: A national sample of 950 patients with hemophilia were included in this study with 412 (43.37%) adults and 538 (56.63%) children patients. The mean EQ-5D-5L utility score was 0.584 (SD=0.292) for adults and 0.868 (SD=0.192) for children, presenting a significant difference in HRQOL between these two groups. The mean of EQ-VAS score for adults and children patient was 59.23 (SD=23.39) and 78.55 (SD=23.07) respectively. The main dimension of HRQOL negatively influenced by hemophilia was mobility for adult and self-care for children respectively. Prophylaxis treatment regimen was examined by regression as a significant influencing factor to achieve better HRQOL both in children and adult patients.

CONCLUSIONS: Hemophilia impaired patients’ HRQOL, especially for adult patients in mobility dimension. It suggested that access to prophylaxis treatment is crucial for patients with hemophilia in China to maintain mobility and gain better HRQOL.