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. 2024 Feb;25(1):103-115.
doi: 10.1007/s10198-023-01574-x. Epub 2023 Feb 21.

Assessing validity of the EQ-5D-5L proxy in children and adolescents with Duchenne muscular dystrophy or spinal muscular atrophy

Richard Huan Xu  1 , Yi Dai  2 , Shamay S M Ng  1 , Hector W H Tsang  1   3 , Shuyang Zhang  4 , Dong Dong  5
Affiliations

Assessing validity of the EQ-5D-5L proxy in children and adolescents with Duchenne muscular dystrophy or spinal muscular atrophy

Richard Huan Xu et al. Eur J Health Econ. 2024 Feb.

Abstract

Objective: To assess the psychometric properties of the EuroQol-5-Dimension five-level instrument (EQ-5D-5L) proxy in caregivers of children and adolescents with Duchenne muscular dystrophy (DMD) or spinal muscular atrophy (SMA).

Method: Data were collected using the EQ-5D-5L proxy for individuals with DMD or SMA, as reported by their caregivers. Ceiling and floor effects, reliability (Cronbach's alpha), convergent and divergent validity (Spearman's correlation coefficient and Bland-Altman plot) and known-group validity (analysis of variance) was used to assess the instrument's psychometric properties.

Results: Totally, 855 caregivers completed the questionnaire. Significant floor effects were observed for most dimensions of the EQ-5D-5L in both SMA and DMD samples. The EQ-5D-5L was strongly correlated with the hypothesized subscales of the SF-12, which confirmed satisfactory convergent and divergent validity. The EQ-5D-5L can significantly differentiate between impaired functional groups for individuals, demonstrating satisfactory discriminative ability. The agreement between the EQ-5D-5L utility and EQ-VAS scores was poor.

Conclusions: Based on the measurement properties assessed in this study, the EQ-5D-5L proxy is a valid and reliable tool for measuring the health-related quality of life of individuals with DMD or SMA rated by caregivers. Further studies should examine the content validity of the EQ-5D as well as the performance of its young version in these two patient groups.

Keywords: Caregiver; Duchenne muscular dystrophy; EQ-5D-5L; Proxy; Spinal muscular atrophy; Validity.

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