https://orcid.org/0009-0007-6516-6860
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.
Data Sharing Statement
The data that support the findings of this study are available from Haemophilia Home but restrictions apply to the availability of these data, which were used under license for the current study, and so are not publicly available. Data are, however, available from the author, LZ, upon reasonable request and with permission of Haemophilia Home.
Ethics Approval and Consent to Participate
This study was approved by the Institutional Review Board Ethics Committee of Fudan University (Ref no.: IRB#2022-02-0951) and conducted in correspondence with regional and relevant guidelines, which complied with the Declaration of Helsinki. Patient consent was obtained via tick box selection for the patient‑reported element of the study.
Acknowledgments
The authors would like to thank Mr. Tao Guan, Mr. Nan Zhao and colleagues from Haemophilia Home for their help and advice with data collection and interpretation. The authors have no competing interests.
Author Contributions
All authors made a significant contribution to the work reported, whether that is in the conception, study design, execution, acquisition of data, analysis and interpretation, or in all these areas; took part in drafting, revising or critically reviewing the article; gave final approval of the version to be published; have agreed on the journal to which the article has been submitted; and agree to be accountable for all aspects of the work.
Disclosure
The authors declare that they have no competing interests.