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

## TL;DR

This study examines the clinical, humanistic, and economic burdens of haemophilia A in China using real-world data from 60 patients.

## Contribution

The study provides real-world insights into treatment strategies and costs for haemophilia A in China.

## Key findings

- Prophylactic treatment reduces annual bleeding rates compared to on-demand treatment in haemophilia A patients.
- Children and adults with haemophilia A have significant economic burdens, with medication as the main cost driver.
- Humanistic burden, measured via EQ-5D and SF-6D, shows lower utility values in adult patients compared to children.

## Abstract

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.

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.

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.

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.

Not applicable.

The online version contains supplementary material available at 10.1186/s13023-025-04051-1.