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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 . doi:10.1212/WNL.0000000000012910 (In Press)
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WRAP-life-expectancy-duchenne-muscular-dystrophy-Abrams-2021.pdf - Accepted Version - Requires a PDF viewer. Available under License Creative Commons Attribution 4.0. Download (4Mb) | Preview |
Official URL: http://dx.doi.org/10.1212/WNL.0000000000012910
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 | |||||||||
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| Subjects: | Q Science > QP Physiology R Medicine > RJ Pediatrics |
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| Divisions: | Faculty of 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: | 13 October 2021 | |||||||||
| Dates: |
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| DOI: | 10.1212/WNL.0000000000012910 | |||||||||
| Status: | Peer Reviewed | |||||||||
| Publication Status: | In Press | |||||||||
| Access rights to Published version: | Open Access | |||||||||
| RIOXX Funder/Project Grant: |
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