Abstract
BACKGROUND: In China, there were over 65,000 hemophilia patients according to estimations requiring overall description. However, former Chinese studies related to hemophilia were based on data of relatively small sample size from single-center or selected multi-centers in specific areas. OBJECTIVE: This study is aimed to provide an overview of patients with hemophilia in China and basic information for optimized hemophilia care and policy decisions in the future. METHODS: We cooperated with the biggest patient organization of hemophilia in China, Hemophilia Home, got access to over ten thousand registered patients with hemophilia and conducted a national representative online survey in 2021. Descriptive statistics were conducted to describe the mean and standard deviation for continuous variables and numbers and proportions for categorical variables. RESULTS: Nine hundred and fifty patients with hemophilia aged 0-71 years were included in our analysis and divided into 538 children and 412 adults. Compared to international research, consistent results were found regarding the proportions of hemophilia types and hemophilia severity, while Chinese patients had less opportunity to receive the formal education and the lower rate to be employed or married. Although children with hemophilia had higher household income, timely treatment, and more prophylaxis treatment leading to better clinical outcomes and higher HRQoL than adults, there were still more annual bleeds, chronic pains, and lower EQ-VAS scores especially in adults than in other countries. CONCLUSIONS AND RECOMMENDATION: Our findings suggest the urgency for promoting hemophilia care and improving the social adaptation of hemophilia patients in China.