Evidence map›Paper›PMID 36809586›Full record

ArticleThe European journal of health economics : HEPAC : health economics in prevention and care2024

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

Richard Huan Xu, Yi Dai, Shamay S M Ng, Hector W H Tsang, Shuyang Zhang, Dong Dong

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Article in The European journal of health economics : HEPAC : health economics in prevention and care, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed, 1 pooled it
1.7field-weighted citation impact, top 15% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

4 citing papers in PubMed, 1 synthesis or guideline pooled it, 6 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Measurement properties of the EQ-5D-3L, EQ-5D-5L, and SF-6Dv2 in patients with late-onset Pompe disease.The European journal of health economics : HEPAC : health economics in prevention and care · 2024
    Article
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4 · The record

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5 · Who and what money

Authors and funding

6 authors at 3 institutions in 2 countries.

Richard Huan XuDepartment of Rehabilitation Sciences, The Hong Kong Polytechnic University, Hong Kong, China.
Yi DaiDepartment of Neurology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing, China.
Shamay S M NgDepartment of Rehabilitation Sciences, The Hong Kong Polytechnic University, Hong Kong, China.
Hector W H TsangDepartment of Rehabilitation Sciences, The Hong Kong Polytechnic University, Hong Kong, China.
Shuyang ZhangDepartment of Cardiology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing, China. shuyangzhang103@nrdrs.org.
Dong DongJC School of Public Health and Primary Care, The Chinese University of Hong Kong, Hong Kong, China. dongdong@cuhk.edu.hk.ORCID http://orcid.org/0000-0001-9784-6472
Hong Kong Polytechnic University · HKChinese Academy of Medical Sciences & Peking Union Medical College · CNChinese University of Hong Kong · HK

Funding

Beijing Natural Science Foundation 7192155Chinese Academy of Medical Sciences Innovation Fund for Medical Sciences 2021-I2M-1-003National Key Research and Development Program of China 2016YFC0901500the '13th Five Year' National Science and Technology Major Project for New Drugs 2019ZX09734001
6 · The paper itself

Abstract

objectiveTo 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).

methodData 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.

resultsTotally, 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.

conclusionsBased 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.

Indexed as

Muscular Atrophy, SpinalMuscular Dystrophy, DuchenneAdolescentChildHumansPsychometricsQuality of LifeReproducibility of ResultsSurveys and QuestionnairesCaregiverDuchenne muscular dystrophyEQ-5D-5LProxySpinal muscular atrophyValidity

Identifiers

PMID36809586
OpenAlexW4321435298

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.