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. 2020 Aug 3;18(1):263.
doi: 10.1186/s12955-020-01511-z.

Measuring quality of life in Duchenne muscular dystrophy: a systematic review of the content and structural validity of commonly used instruments

Philip A Powell  1   2 , Jill Carlton  3 , Helen Buckley Woods  3 , Paolo Mazzone  3
Affiliations

Measuring quality of life in Duchenne muscular dystrophy: a systematic review of the content and structural validity of commonly used instruments

Philip A Powell et al. Health Qual Life Outcomes. .

Abstract

Duchenne muscular dystrophy (DMD) is an inherited X-linked neuromuscular disorder. A number of questionnaires are available to assess quality of life in DMD, but there are concerns about their validity. This systematic review aimed to appraise critically the content and structural validity of quality of life instruments for DMD. Five databases (EMBASE, MEDLINE, CINAHL, PsycINFO, and Cochrane Library) were searched, with supplementary searches in Google Scholar. We included articles with evidence on the content and/or structural validity of quality of life instruments in DMD, and/or instrument development. Evidence was evaluated against the Consensus-based Standards for the selection of health Measurement INstruments (COSMIN) criteria. Fifty five articles featured a questionnaire assessing quality of life in DMD. Forty instruments were extracted and 26 underwent assessment. Forty-one articles contained evidence on content or structural validity (including 37 development papers). Most instruments demonstrated low quality evidence and unsatisfactory or inconsistent validity in DMD, with the majority not featuring direct validation studies in this population. Only KIDSCREEN received an adequate rating for instrument design and a satisfactory result for content validity based on its development, yet, like the majority of PROMs, the measure has not been directly validated for use in DMD. Further research is needed on the validity of quality of life instruments in DMD, including content and structural validity studies in this population.

Keywords: Content validity; Duchenne muscular dystrophy; Patient reported outcome measures; Quality of life; Structural validity.

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Conflict of interest statement

Two of the authors (PP and JC) are employed on a related project funded by Duchenne UK that involves developing a new preference-based patient reported outcome measure of quality of life for people with DMD.

Figures

Fig. 1
Fig. 1
Flow diagram of search strategy and selection of papers
See this image and copyright information in PMC

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