# Assessing the health-state utility values of rare disease-hemophilia B using EQ-5D-5L: a study based on the Chinese population

## TL;DR

This study measures health-state utility values for Chinese hemophilia B patients using EQ-5D-5L and identifies factors affecting these values.

## Contribution

The study is the first to assess health-state utility values specifically for Chinese hemophilia B patients.

## Key findings

- The mean health-state utility value was 0.755 with significant issues in pain/discomfort reported by 57.49% of patients.
- Factors like pain, disability, and drug usage were significantly associated with health-state utility values.
- Proxy assessments may overestimate health-state utility values compared to self-completion.

## Abstract

Obtaining health-state utility values (HSUVs) aids in making scientific decisions in patient health management, especially for rare disease patients. However, there is currently no research specifically measuring the HSUVs of Chinese hemophilia B patients. Therefore, this study aims to assess the HSUVs of hemophilia B patients in China and explore its potential influencing factors.

The sociodemographic characteristics of patients were obtained from the Beijing Hemophilia Home Care Center (BHHCC) database. And the HSUVs were further obtained by reaching hemophilia B patients through an application of BHHCC and the Chinese version of the EQ-5D-5L. The beta regression model was used to explore the potential influencing factors of the HSUVs of patients.

A total of 167 male patients (hemophilia B is an X-chromosome recessive disorder and female patients are rare) were included in the study. The mean age, HSUV and EQ-VAS were 20.01 ± 15.83, 0.755 ± 0.291 and 71.7 ± 22.7, respectively. The ceiling effects was 29.24%, and patients were more likely to experience problems in Pain/discomfort (57.49%). Compared to self-completion, proxy may overestimate HSUVs of patients. Pain (p < 0.000), disability (p < 0.000), complications (p < 0.001), inhibitors (p < 0.01), drug usage (p < 0.001), and bleeds (p < 0.000) were significantly associated with HSUVs in Chinese hemophilia B patients.

This study first assessed the HSUVs of Chinese hemophilia B patients, which provides support for further economic studies. Potential factors that affect the HSUVs of Chinese hemophilia B patients were also explored, which can provide a reference for developing health management measures. However, to enact more comprehensive and reliable disease management decisions, the effects of self-completion and proxy on the HSUVs of hemophilia B patients in China need to be further explored as well as the effects of specific factors.

The online version contains supplementary material available at 10.1186/s13023-025-03894-y.