Treatment regimens, patient reported outcomes and health-related quality of life in children with moderate and severe haemophilia A in China: using real-world data

Abstract

Background Prophylaxis therapy for children with moderate and severe haemophilia A (HA) is the optimal treatment regimen. The real-world treatment regimens, patient reported outcomes and health-related quality of life (HRQoL) in children with moderate and severe HA in China are less known.Objective This study aimed to describe the real-world treatment regimens and evaluate the association oftreatment regimens with comprehensive patient reported outcomes including bleeds, chronic pain, target joints, disability and HRQoL in children under 18 years old with HA in China.Methods Real-world data of a nationwide online cross-sectional survey in 2021 and patients’ coagulation factor utilization data from self-management records from 2020 to 2021 were merged. 373 eligible children were included and categorized by treatment regimens according to the Chinese guideline: on-demand, short-term prophylaxis and long-term prophylaxis treatment.Results Currently, in China, 4.8% of children with HA are receiving full-dose long-term prophylaxis treatment. Prophylaxis treatment was a significant positive predictor of better patient reported outcomes and HRQoL. For children with prophylaxis treatment, there were significantly fewer annual bleeds (p < 0.001), lower frequency of chronic pain(p < 0.001), and higher health utility scores(p < 0.01) and EQ-VAS scores(p < 0.05) than children with on-demand treatment.Conclusion Accessible long-term prophylaxis treatment should be promoted for children with moderate and severe HA in China and regular monitoring of their outcomes and HRQoL should be carried out.