OBJECTIVES: Hemophilia is a rare disease hereditary bleeding disorder which needed continuous treatment and life-long care. Regular prophylaxis treatment (RPT) is recommended as a standard treatment regimen, while intermittent prophylaxis treatment (IPT) and on-demand treatment (ODT) is commonly utilized in reality under resource constrain. In China, approximately 39,000 patients live with hemophilia. However, little is known about patients’ treatment model in real life and related clinical outcomes. The aim of this study was to analyze patient-reported clinical outcomes with three hemophilia treatment regimens in real world in China.

METHOD A nationwide, online, cross-sectional survey was conducted with collaboration with the biggest hemophilia patient organization in China. Data on demographic characteristics, health care, clinical outcomes were collected through questionnaires completed by patients or their caregivers.

RESULTS: A national sample of 920 patients with hemophilia were included in this analysis. According to self-reported data, 32.3% of hemophilia patients received RPT regime, while 36.4% of patients received ODT and 31.3% of patients received IPT. The mean of the annual bleeding times for patients receiving RPT, IPT and ODT were21 (SD=43), 38 (SD=53) and 63 (SD=64) respectively, with more bleedings in joints, muscles, digestive system or internal organs occurred on ODT patients. 27.46% of ODT patients tolerated chronic pain lasting more than 50% of the time, which was significantly higher than that among IPT patients (12.50%) and RPT patients (5.72%). It has also been witnessed higher proportion of disability due to hemophilia in ODT patients (51.34%), comparing with IPT patients (30.56%) and RPT patients (19.53%) .

CONCLUSION Only 1/3 of hemophilia patients in China obtained RPT regimen. Inadequate treatment as ODT and IPT resulted in undesired clinical consequences. Measures should be taken to improve hemophilia patients’ accessibility to RPT to gain better clinical outcomes.