Evidence map›Paper›PMID 34645707›Full record

SynthesisNeurology2021

Life Expectancy in Duchenne Muscular Dystrophy: Reproduced Individual Patient Data Meta-analysis.

Jonathan Broomfield, Micki Hill, Michela Guglieri, Michael Crowther, Keith Abrams

Abstract readMeta-AnalysisSystematic Review
PubMed Full text at PMC Publisher
In one paragraph

Synthesis in Neurology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 125 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
125citing papers in PubMed, 3 pooled it
–field-weighted citation impact
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

125 citing papers in PubMed, 3 syntheses or guidelines pooled it.

  1. Pooled it
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  11. Age-Dependent Effect of Myostatin Blockade in theMuscles (Basel, Switzerland) · 2026
    Article
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  13. Review
  14. Article
  15. Progress on cell therapy for skeletal muscle disorders.Advanced drug delivery reviews · 2026
    Review
  16. Dystrophinopathy with aPCN reports : psychiatry and clinical neurosciences · 2026
    Article
  17. Review
  18. X-Linked Muscular Dystrophy in a Cat with a Putative Variant in theAnimals : an open access journal from MDPI · 2026
    Article
  19. Respiratory function in Becker muscular dystrophy: a comprehensive longitudinal study.Journal of neurology, neurosurgery, and psychiatry · 2026
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  20. Article

65 more citing papers are in PubMed but not listed here.

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

5 authors.

Jonathan BroomfieldFrom the Department of Health Sciences (J.B., M.H.), University of Leicester; Institute of Human Genetics (M.G.), Newcastle University, UK; Department of Medical Epidemiology and Biostatistics (M.C.), Karolinska Institute, Stockholm, Sweden; and Centre for Health Economics (K.A.), University of York, UK. jb781@le.ac.uk.
Micki HillFrom the Department of Health Sciences (J.B., M.H.), University of Leicester; Institute of Human Genetics (M.G.), Newcastle University, UK; Department of Medical Epidemiology and Biostatistics (M.C.), Karolinska Institute, Stockholm, Sweden; and Centre for Health Economics (K.A.), University of York, UK.
Michela GuglieriFrom the Department of Health Sciences (J.B., M.H.), University of Leicester; Institute of Human Genetics (M.G.), Newcastle University, UK; Department of Medical Epidemiology and Biostatistics (M.C.), Karolinska Institute, Stockholm, Sweden; and Centre for Health Economics (K.A.), University of York, UK.
Michael CrowtherFrom the Department of Health Sciences (J.B., M.H.), University of Leicester; Institute of Human Genetics (M.G.), Newcastle University, UK; Department of Medical Epidemiology and Biostatistics (M.C.), Karolinska Institute, Stockholm, Sweden; and Centre for Health Economics (K.A.), University of York, UK.
Keith AbramsFrom the Department of Health Sciences (J.B., M.H.), University of Leicester; Institute of Human Genetics (M.G.), Newcastle University, UK; Department of Medical Epidemiology and Biostatistics (M.C.), Karolinska Institute, Stockholm, Sweden; and Centre for Health Economics (K.A.), University of York, UK.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

BACKGROUND AND

objectivesDuchenne muscular dystrophy (DMD) is a rare progressive disease that is often diagnosed in early childhood and leads to considerably reduced life expectancy; because of its rarity, research literature and patient numbers are limited. To fully characterize the natural history, it is crucial to obtain appropriate estimates of the life expectancy and mortality rates of patients with DMD.

methodsA systematic review of the published literature on mortality in DMD up to July 2020 was undertaken, specifically focusing on publications in which Kaplan-Meier (KM) survival curves with age as a timescale were presented. These were digitized, and individual patient data (IPD) were reconstructed. The pooled IPD were analyzed with the KM estimator and parametric survival analysis models. Estimates were also stratified by birth cohort.

resultsOf 1,177 articles identified, 14 publications met the inclusion criteria and provided data on 2,283 patients, of whom 1,049 had died. Median life expectancy was 22.0 years (95% confidence interval [CI] 21.2, 22.4). Analyses stratified by 3 time periods in which patients were born showed markedly increased life expectancy in more recent patient populations; patients born after 1990 have a median life expectancy of 28.1 years (95% CI 25.1, 30.3). DISCUSSION: This article presents a full overview of mortality across the lifetime of a patient with DMD and highlights recent improvements in survival. In the absence of large-scale prospective cohort studies or trials reporting mortality data for patients with DMD, extraction of IPD from the literature provides a viable alternative to estimating life expectancy for this patient population.

Indexed as

Muscular Dystrophy, DuchenneChild, PreschoolHumansKaplan-Meier EstimateLife ExpectancyProspective Studies

Identifiers

PMID34645707
PMCPMC8665435

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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.