Evidence map›Paper›PMID 32746836›Full record

SynthesisHealth and quality of life outcomes2020

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

Philip A Powell, Jill Carlton, Helen Buckley Woods, Paolo Mazzone

Open access · goldAbstract readSystematic Review
PubMed Full text at PMC Publisher
In one paragraph

Synthesis in Health and quality of life outcomes, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers, 5 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
28citing papers in PubMed, 5 pooled it
2.8field-weighted citation impact, top 8% 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

28 citing papers in PubMed, 5 syntheses or guidelines pooled it, 55 citations in OpenAlex.

  1. Pooled it
  2. Health-related quality of life of informal carers in ALS: a systematic review of person reported outcome measures.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2025
    Pooled it
  3. Health-related quality of life in osteoporosis: a systematic review of measurement properties of the QUALEFFO-41.Osteoporosis international : a journal established as result of cooperation between the European Foundation for Osteoporosis and the National Osteoporosis Foundation of the USA · 2024
    Pooled it
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  12. A Qualitative Examination of the Content Validity of the EQ-5D-5L and EQ-5D-Y-3L in Adult and Paediatric Patients With Duchenne Muscular Dystrophy.Health expectations : an international journal of public participation in health care and health policy · 2025
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

4 authors at 1 institution in 1 country.

Philip A PowellSchool of Health and Related Research, University of Sheffield, Regent Court, 30 Regent Street, Sheffield, S1 4DA, UK. p.a.powell@sheffield.ac.uk.ORCID http://orcid.org/0000-0003-1169-3431
Jill CarltonSchool of Health and Related Research, University of Sheffield, Regent Court, 30 Regent Street, Sheffield, S1 4DA, UK.
Helen Buckley WoodsSchool of Health and Related Research, University of Sheffield, Regent Court, 30 Regent Street, Sheffield, S1 4DA, UK.
Paolo MazzoneSchool of Health and Related Research, University of Sheffield, Regent Court, 30 Regent Street, Sheffield, S1 4DA, UK.
University of Sheffield · GB

Funding

Duchenne UK project HERCULES
6 · The paper itself

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.

Indexed as

Quality of LifeFemaleHumansMaleMuscular Dystrophy, DuchenneSurveys and QuestionnairesValidation Studies as TopicContent validityDuchenne muscular dystrophyPatient reported outcome measuresQuality of lifeStructural validity

Identifiers

PMID32746836
PMCPMC7397669
OpenAlexW3047261561

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

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.