An overview of patients with haemophilia A in China: Epidemiology, disease severity and treatment strategies

Abstract

Introduction Haemophilia A (HA) is a rare X chromosome‐linked bleeding disorder resulting in missing or defective clotting factor VIII (FVIII) and causes large disease burden. Aim As a member of World Federation of Hemophilia, China seeks to understand the current epidemiology, disease profile and treatment landscape of patients with HA through the Hemophilia Treatment Center Collaboration Network of China (HTCCNC). Methods The HTCCNC enabled data collection on patients with HA from 166 member hospitals (2007–2019) across China. The distribution of patients across 31 divisions was summarized using a heat map. Patient demographics, disease severity and clinical and treatment information were summarized using descriptive statistics. Results HTCCNC identified 17,779 patients with HA during 2007–2019. Patients were predominantly male (99.99%), and 28.3% had a known family history of haemophilia. Among patients with lab‐measured disease severity (N = 13,116), 6,519 had severe HA (49.7%), 4,788 had moderate HA (36.5%), and 1,809 had mild HA (13.8%). Among patients with information on the delays, delays in diagnosis and in treatment initiation were observed in 1,437 (28.8%) and 1,750 (39.2%) patients, respectively. On average, those patients had an 8.4 years gap between the first bleed and HA diagnosis and a delay of 8.6 years from the first bleed to treatment initiation. Additionally, 44.33% of patients relied solely on episodic treatments, and 16.2% received any prophylaxis treatments. Conclusions Using data from the largest haemophilia registry in China, this study indicated that delayed diagnosis and treatment, together with low utilization of prophylaxis, are key challenges for patients with HA.

Treatment Adherence and Health-Related Quality of Life in Patients with Hemophilia in Hong Kong

“…The distribution of disease severity (43% severe, 33% moderate, and 24% mild) is similar to that of mainland China and Taiwan. Notably, the rate of prophylactic factor infusion (34%) among patient with hemophilia in Hong Kong is slightly higher than the estimated rate (16.2%) in a report from mainland China [ 20 ]. The population factor use of 1.83 units of FVIII per capita [ 19 ] is comparable to factor consumption levels in other developed countries in Asia, such as Korea, Japan, and Singapore [ 21 ].…”

Prophylactic emicizumab for hemophilia A in the Asia‐Pacific region: A randomized study (HAVEN 5)

“…7 In developing countries of the Asia-Pacific region, the disease burden associated with hemophilia A is greater compared with Western countries, with the limited use of prophylaxis being one of the main contributing factors. 8,9 The Asia-Pacific Hemophilia Working Group has developed a Principles of Hemophilia Care document, taking into account variability in regional health care systems and the socioeconomic and cultural diversities, in an effort to drive forward hemophilia care in the region. 10 There remains, however, a high unmet need in reducing disease burden for people with hemophilia A in the Asia-Pacific region.…”

Health‐related quality of life among adults with haemophilia in China: A comparison with age‐matched general population

“…40 Previous studies conducted in China indicated that, late initiation of prophylaxis, coagulation factor deficiency and poor compliance may explain the limited impact of prophylaxis on HRQoL in PwH. 3,5 This study had several limitations. First, the cross-sectional survey was suitable for determining associations but not for determining…”

“…Another reason might be the differences in the level of haemophilia management. A previous study suggested that the prophylaxis of haemophilia in China lagged behind developed countries such as the United Kingdom and the United States, 5 which might have an impact on the HRQoL of patients in China.…”

“…A previous study showed that Chinese PwH had delayed diagnosis and inadequate treatment, with a significant disease burden. 5 Nonetheless, China has made substantial progress in haemophilia care in the past decade, and the gap with developed countries in treatment has begun to narrow. 3 The use of factor concentrates, treatment of blood-borne diseases and improved healthcare increased the life expectancy of PwH.…”