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Life expectancy in Duchenne Muscular Dystrophy : reproduced individual patient data meta-analysis

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Broomfield, Jonathan, Hill, Micki, Guglieri, Michela, Crowther, Michael and Abrams, Keith R. (2021) Life expectancy in Duchenne Muscular Dystrophy : reproduced individual patient data meta-analysis. Neurology, 97 (23). e2304-e2314. doi:10.1212/WNL.0000000000012910

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Official URL: http://dx.doi.org/10.1212/WNL.0000000000012910

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Abstract

Objective: Duchenne Muscular Dystrophy (DMD) is a rare progressive disease, which is often diagnosed in early childhood, and leads to considerably reduced life-expectancy; due to its rarity, research literature and patient numbers are limited. To fully characterise 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 until July 2020 was undertaken, specifically focusing on publications in which Kaplan-Meier (KM) survival curves with age as a time-scale were presented. These were digitised and individual patient data (IPD) reconstructed. The pooled IPD were analysed using the Kaplan-Meier estimator and parametric survival analysis models. Estimates were also stratified by birth cohort.

Results: Of 1177 articles identified, 14 publications met the inclusion criteria and provided data on 2283 patients, of whom 1049 had died. Median life-expectancy was 22.0 years (95% CI: 21.2, 22.4). Analyses stratifying by three 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).

Conclusions: This paper 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.

Item Type: Journal Article
Subjects: Q Science > QP Physiology
R Medicine > RJ Pediatrics
Divisions: Faculty of Science, Engineering and Medicine > Science > Statistics
Library of Congress Subject Headings (LCSH): Duchenne muscular dystrophy , Duchenne muscular dystrophy -- Patients, Duchenne muscular dystrophy -- Patients -- Mathematical models , Life expectancy -- Statistics
Journal or Publication Title: Neurology
Publisher: Wolters Kluwer Health, Inc.
ISSN: 0028-3878
Official Date: December 2021
Dates:
DateEvent
December 2021Published
13 October 2021Available
27 September 2021Accepted
Volume: 97
Number: 23
Page Range: e2304-e2314
DOI: 10.1212/WNL.0000000000012910
Status: Peer Reviewed
Publication Status: Published
Access rights to Published version: Open Access
RIOXX Funder/Project Grant:
Project/Grant IDRIOXX Funder NameFunder ID
UNSPECIFIEDNational Institute for Health Researchhttp://dx.doi.org/10.13039/501100000272
UNSPECIFIEDAction Duchennehttp://dx.doi.org/10.13039/501100001266

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