Demographics and Clinical Profile of Hemophilia Patients in China - Results from a Cross-Sectional, Community-Based Survey
Authors: Xue Feng , Chang Chun , Hsu Karina Chin Po , Sun Jing , Zhang Xinsheng , Wang Shujie , Wu Jingsheng , Chen Rong , Shi Danni , Cao Felix , Yang Renchi 
Published: December 2024

Introduction: The treatment landscape for hemophilia has seen a rapid development of novel therapeutic modalities, with the approach to routine prophylaxis shifting towards non-factor replacement therapies. Currently in China, a vast majority of hemophilia patients manage the condition with factor replacement products, and a snapshot analysis of their demographic information and clinical characteristics would be valuable to gauging how the studied cohort is faring on the journey of hemophilia management with traditional pharmacological options. More importantly, an updated understanding of the current clinical status, disease outcomes and health management aptitude of people with hemophilia (PwH) in China would offer insight into the evolving patient needs and expectations standing to be addressed by the emerging treatments of unique pharmacological features and complementary clinical utility.
Aim: To describe the current clinical profile, hemophilia outcomes and care-seeking behavior of adult PwH in China
Methods: Adult PwH were approached from certified Hemophilia Treatment Centers throughout the country to participate in a web-based, self-administered survey inquiring about clinical profile, hemophilia treatment approaches, non-pharmacological management measures, health-related quality of life, psychological well-being and disease coping capacity. Data analysis was based on descriptive statistics and pos-hoc significance testing. The survey questions incorporated the 36-Item Short Form Health Survey (SF-36), Patient Health Quesionnaire-9 (PHQ-9) and Generalized Anxiety Disorder-7 (GAD-7).
Results: The survey was conducted in 2023 and included 1765 adult PwH (mean [SD] age: 33.8 [10.1] years, range 18-83 years), comprising 1010 (57.2%) aged 18-34 and 755 (42.8%) aged 35 and above.86.8% of survey respondents are diagnosed with hemophilia A and 64.0% have severe disease (&lt;1 IU/dL). 37.7% were diagnosed before age of two, 12.6% developed inhibitors and 50.2% have known family history.Hemophilic arthropathy was the most common complication, reported by 62.8% of participants. Presence of hemophilia-related physical disability was prevalent among 80.5% of participants. Close to half of the whole cohort (48.6%) reported having at least three target joints. The mean annualized bleeding rate (ABR) and mean joint ABR were 16.50±25.57 and 13.13±22.62, respectively.
In terms of treatment choice, the majority of patients (83%) are receiving recombinant factor products, and notably only 1% uses emicizumab, which is currently the only approved non-factor product in China. 42.0% of participants reported attending hemophilia-specific clinical consultations at an interval longer than 12 months, and well over half (64.3%) had never undergone a comprehensive multidisciplinary assessment.
According to PHQ-9 and GAD-7 scores, 80.2% of adult PwH surveyed showed depressive symptoms, with 18.64% being classified as severely depressed; 66.4% exhibited anxiety symptoms, with 9.07% falling into the tier of severe anxiety. The mean overall SF-36 score among all respondents was 47.94±20.03; the social functioning dimension scored 75.53±28.04, while the physical functioning dimension scored only 21.02±35.21, out of a full score of 100 for each dimension.
Conclusion: For adult PwH participating in the survey whose condition is primarily managed with factor-based products, there exists a considerable room for improvement in terms of clinical outcomes and activation in care optimization. Facilitating patient interactions with hemophilia care team and access to multidisciplinary resources may be crucial in encouraging ongoing patient-centered dialogue about an emerging array of treatment options.