Evidence map›Paper›PMID 26483095›Full record

Observational studyDevelopmental medicine and child neurology2016

Health-related quality of life in patients with Duchenne muscular dystrophy: a multinational, cross-sectional study.

Erik Landfeldt, Peter Lindgren, Christopher F Bell, Michela Guglieri, Volker Straub, Hanns Lochmüller, Katharine Bushby

Open access · hybridAbstract readObservational Study
PubMed Full text at PMC Publisher
In one paragraph

Observational study in Developmental medicine and child neurology, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 62 papers, 6 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
62citing papers in PubMed, 6 pooled it
4.2field-weighted citation impact, top 5% 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

62 citing papers in PubMed, 6 syntheses or guidelines pooled it, 123 citations in OpenAlex.

  1. Pooled it
  2. Guideline
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  4. Characterizing health state utilities associated with Duchenne muscular dystrophy: a systematic review.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2020
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2 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

7 authors at 4 institutions in 4 countries.

Erik LandfeldtInstitute of Environmental Medicine, Karolinska Institutet, Stockholm.
Peter LindgrenMedical Management Centre, Department of Learning, Informatics, Management and Ethics, Karolinska Institutet, Stockholm, Sweden.
Christopher F BellGlaxoSmithKline, Research Triangle Park, NC, USA.
Michela GuglieriNewcastle University John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne.
Volker StraubNewcastle University John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne.
Hanns LochmüllerNewcastle University John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne.
Katharine BushbyNewcastle University John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne.
Newcastle University · GBKarolinska Institutet · SECentre for Life · GBResearch Triangle Park Foundation · US

Funding

Medical Research Council G1002274Medical Research Council MR/K000608/1
6 · The paper itself

Abstract

aimTo estimate health-related quality of life (HRQOL) in patients with Duchenne muscular dystrophy (DMD).

methodHRQOL was assessed using the Health Utilities Index Questionnaire (HUI) and the Pediatric Quality of Life Inventory (PedsQL) neuromuscular module version 3.0 online. Results were stratified by disease stage (early/late ambulatory/non-ambulatory) and caregivers' perceptions of patients' health and mental status.

resultsA total of 770 patient-caregiver pairs (173 German, 122 Italian, 191 UK, and 284 USA) participated. Most caregivers (>84%) perceived their patients as happy/somewhat happy and in excellent/very good/good health, irrespective of current ambulatory class. In contrast, mean patient utility (reflecting public preferences: 0, dead; 1, perfect health) deteriorated with disease course, from 0.75 in early ambulatory males to 0.15 in the most severely affected patients. Mean patient PedsQL scores (0-100, higher score indicating better HRQOL) decreased from 80 to 57 across ambulatory classes.

interpretationHRQOL in DMD, measured through public preferences, is substantially impaired in relation to the general population and significantly associated with disease progression. Still, most patients are perceived as happy and in good health by their caregivers, indicating that influential domains of HRQOL remain intact through the disease progression. Our findings emphasize the challenges in measuring HRQOL in a rare, progressive childhood condition such as DMD.

Indexed as

Disease ProgressionAdolescentAdultAgedCaregiversChildChild, PreschoolCross-Sectional StudiesFemaleGermanyHumansItalyMaleMiddle AgedMuscular Dystrophy, DuchenneParents

Identifiers

PMID26483095
PMCPMC4949722
OpenAlexW2120361462

What OpenQuestion holds

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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.