Related Experiment Video
Updated: Oct 17, 2025
Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Life Expectancy in Duchenne Muscular Dystrophy: Reproduced Individual Patient Data Meta-analysis
Jonathan Broomfield1, Micki Hill2, Michela Guglieri2
1From 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.
Background And Objectives:
Duchenne 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.
Methods:
A 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.
Results:
Of 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.
