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EQ-5D and SF-6D health utility scores in patients with spinal and bulbar muscular atrophy

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Abstract

Objective

This study assessed patient-reported health-related quality of life (HRQoL) using two generic preference-based measures in Chinese patients with spinal and bulbar muscular atrophy (SBMA) and identified demographic and clinical determinants of health utility scores in this population.

Methods

This study used cross-sectional data of 212 Chinese patients with SBMA who completed both the EQ-5D and SF-6D. Association between response to EQ-5D and SF-6D dimensions was examined using Spearman’s correlation coefficient, and the association between the two utility scores was assessed using Pearson’s correlation coefficient. The variations in utility scores across patients in different subgroups were compared using one-way ANOVA. Bland–Altman (B–A) plot was used to assess the agreement of utility scores between EQ-5D and SF-6D. A multivariate Tobit regression model was employed to estimate the association between utility scores and the presence of symptoms and chronic conditions.

Results

The mean utility scores for the EQ-5D and SF-6D were 0.54 and 0.56, respectively. The hypothesized correlation between the EQ-5D and SF-6D dimensions ranged from 0.31 to 0.58, and the correlation between their utility scores was 0.64. An acceptable agreement between EQ-5D and SF-6D utility scores was identified by B–A plot. Patients with chronic diseases, misdiagnosis, high financial burden, and several clinical symptoms were highly likely to report a low health utility score.

Conclusions

This study is the first to investigate the HRQoL of patients with SBMA worldwide. The estimated health utility scores for EQ-5D and SF-6D can be utilized as baseline data for future cost-utility analyses of SBMA-related interventions.

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Availability of data and materials

Derived data supporting the findings of this study are available from the corresponding author on request.

Code availability

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Acknowledgements

We thank the Kennedy’s Disease Care Center of China for assisting in questionnaire design and data collection, the patients with spinal and bulbar muscular atrophy for completing the questionnaires, the Beijing Society of Rare Disease Clinical Care and Accessibility and the Illness Challenge Foundation for coordinating this research.

Funding

This work was supported by the National Key Research and Development Program of China (Grant No. 2016YFC0901500) and the Center for Rare Diseases Research, Chinese Academy of Medical Sciences, Beijing, China (Grant No. 2016ZX310174-4).

Author information

Authors and Affiliations

  1. Department of Rehabilitation Sciences, The Hong Kong Polytechnic University, Hong Kong, SAR, China

    Richard Huan Xu

  2. JC School of Public Health and Primary Care, The Chinese University of Hong Kong, Shatin, NT, Hong Kong, SAR, China

    Richard Huan Xu & Dong Dong

  3. Department of Neurology, Beijing Chao-Yang Hospital, Capital Medical University, Beijing, China

    Ming Lu

  4. Department of Cardiology, Peking Union Medical College Hospital, Peking Union Medical College, Chinese Academy of Medical Science, Beijing, 100730, China

    Shuyang Zhang

  5. Shenzhen Research Institute, The Chinese University of Hong Kong, Shenzhen, Guangdong, China

    Dong Dong

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  1. Richard Huan Xu
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  2. Ming Lu
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  3. Shuyang Zhang
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  4. Dong Dong
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Contributions

Study conception and design: DD, SYZ. Material preparation, data collection: RHX, DD. Data analysis: RHX and DD. Draft preparation: RHX and DD. Manuscript revision: RHX, DD, ML. Supervision: DD, SYZ.

Corresponding authors

Correspondence to Shuyang Zhang or Dong Dong.

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The authors declare that they have no conflict of interest.

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Informed consent was obtained from all individual participants included in the study.

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Ethical approval

All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Helsinki Declaration and its later amendments or comparable ethical standards. Both the institutional review board ethics committee of the Chinese University of Hong Kong and Peking Union Medical College Hospital approved the study protocol and informed consent (Ref No. SBRE-18-268 and SK814).

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Xu, R.H., Lu, M., Zhang, S. et al. EQ-5D and SF-6D health utility scores in patients with spinal and bulbar muscular atrophy. Eur J Health Econ 24, 1399–1410 (2023). https://doi.org/10.1007/s10198-022-01551-w

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  • DOI: https://doi.org/10.1007/s10198-022-01551-w

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