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Objectives <p>We aimed to describe current treatments for haemophilia A in China, focusing on their associated clinical, humanistic, and economic burdens in a real-world context.</p> Methods <p>This was a retrospective cross-sectional study. We investigated the demographics, disease severity, treatment strategies, and clinical outcomes of patients diagnosed with haemophilia A. We also investigated the cost of the treatment of patients with haemophilia A. In the real world, we estimated annual direct medical costs, direct non-medical costs and indirect costs. In addition, we employed the EQ-5D and SF-6D to measure the humanistic burden of patients.</p> Results <p>A total of 60 patients were included in the study, comprising 22 children (&lt; 18 years) and 38 adults (≥ 18 years). The mean age of the children and adults was 9.27 and 33.05 years, respectively. Treatment strategies for adults were primarily on-demand. Patients receiving prophylactic treatment experienced fewer bleeds per year compared to those receiving on-demand treatment (mean ABR: adults 42.91 vs. 20.38; children 20.20 vs. 10.10). The mean EQ-5D utility value reported by children and adults were 0.76 (SD 0.24) and 0.51 (SD 0.34), respectively. For adult patients, the SF-6D utility value was 0.38. The mean total annual direct medical costs associated with haemophilia were ¥429,143 (US$58,666) for children and ¥340,238 (US$46,512) for adults, with medication being the primary cost driver.</p> Conclusions <p>These data document the enormous burden of haemophilia A that persists in the real world of China. While we emphasize incremental direct healthcare expenditures, we must also consider the long-term clinical and socio-economic benefits of prophylactic treatment.</p> Clinical trial number <p>Not applicable.</p>

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Clinical, humanistic, and economic burden of haemophilia A in China: findings from a real-world survey

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年10月24日

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  • Junchao Feng,
  • Lei Dou,
  • Jingdan Chen,
  • Yunhai Fang,
  • Yan Cheng,
  • Shunping Li

摘要

Objectives

We aimed to describe current treatments for haemophilia A in China, focusing on their associated clinical, humanistic, and economic burdens in a real-world context.

Methods

This was a retrospective cross-sectional study. We investigated the demographics, disease severity, treatment strategies, and clinical outcomes of patients diagnosed with haemophilia A. We also investigated the cost of the treatment of patients with haemophilia A. In the real world, we estimated annual direct medical costs, direct non-medical costs and indirect costs. In addition, we employed the EQ-5D and SF-6D to measure the humanistic burden of patients.

Results

A total of 60 patients were included in the study, comprising 22 children (< 18 years) and 38 adults (≥ 18 years). The mean age of the children and adults was 9.27 and 33.05 years, respectively. Treatment strategies for adults were primarily on-demand. Patients receiving prophylactic treatment experienced fewer bleeds per year compared to those receiving on-demand treatment (mean ABR: adults 42.91 vs. 20.38; children 20.20 vs. 10.10). The mean EQ-5D utility value reported by children and adults were 0.76 (SD 0.24) and 0.51 (SD 0.34), respectively. For adult patients, the SF-6D utility value was 0.38. The mean total annual direct medical costs associated with haemophilia were ¥429,143 (US$58,666) for children and ¥340,238 (US$46,512) for adults, with medication being the primary cost driver.

Conclusions

These data document the enormous burden of haemophilia A that persists in the real world of China. While we emphasize incremental direct healthcare expenditures, we must also consider the long-term clinical and socio-economic benefits of prophylactic treatment.

Clinical trial number

Not applicable.