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Meta-Analysis
. 2020 Jul;35(7):643-653.
doi: 10.1007/s10654-020-00613-8. Epub 2020 Feb 27.

Life expectancy at birth in Duchenne muscular dystrophy: a systematic review and meta-analysis

Erik Landfeldt  1 , Rachel Thompson  2 , Thomas Sejersen  3   4 , Hugh J McMillan  2 , Janbernd Kirschner  5   6 , Hanns Lochmüller  2   5
Affiliations
Meta-Analysis

Life expectancy at birth in Duchenne muscular dystrophy: a systematic review and meta-analysis

Erik Landfeldt et al. Eur J Epidemiol. 2020 Jul.

Abstract

Several studies indicate that prognosis for survival in Duchenne muscular dystrophy (DMD) has improved in recent decades. However, published evidence is inconclusive and some estimates may be obsolete due to improvements in standards of care, in particular the routine use of mechanical ventilatory support in advanced stages of the disease. In this systematic review and meta-analysis (PROSPERO identifier: CRD42019121800), we searched MEDLINE (through PubMed), CINAHL, Embase, PsycINFO, and Web of Science for studies published from inception up until December 31, 2018, reporting results of life expectancy in DMD. We pooled median survival estimates from individual studies using the median of medians, and weighted median of medians, methods. Risk of bias was established with the Newcastle-Ottawa Scale. Results were stratified by ventilatory support and risk of bias. We identified 15 publications involving 2662 patients from 12 countries from all inhabited continents except Africa. Median life expectancy without ventilatory support ranged between 14.4 and 27.0 years (pooled median: 19.0 years, 95% CI 18.0-20.9; weighted pooled median: 19.4 years, 18.2-20.1). Median life expectancy with ventilatory support, introduced in most settings in the 1990s, ranged between 21.0 and 39.6 years (pooled median: 29.9 years, 26.5-30.8; weighted pooled median: 31.8 years, 29.3-36.2). Risk of bias had little impact on pooled results. In conclusion, median life expectancy at birth in DMD seems to have improved considerably during the last decades. With current standards of care, many patients with DMD can now expect to live into their fourth decade of life.

Keywords: Mechanical ventilation; Mortality; Prognosis; Survival.

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Conflict of interest statement

Dr. Landfeldt is an employee of ICON plc (Stockholm, Sweden), outside the submitted work. Professor Sejersen reports receipt of grants/research supports from Sanofi-Genzyme and PTC, and receipt of honoraria or consultation fees from Sanofi-Genzyme, PTC, Biogen, Roche, and Sarepta, outside the submitted work. Professor Kirschner reports grants, personal fees and non-financial support from Roche, personal fees from Avexis, grants, personal fees and non-financial support from Biogen, grants from Santhera, grants and personal fees from PTC, outside the submitted work. Dr. Thompson and Dr. McMillan report no disclosures. Professor Lochmüller is the Editor-in-chief for the Journal of Neuromuscular Diseases (IOS Press) and reports consultancy and financial support for research projects and clinical trials by AMO Pharma, Biogen, Desitin, GW Pharma, Pfizer, PTC Therapeutics, Roche, Santhera, Sarepta, Satellos, and Ultragenyx, outside the submitted work.

Figures

Fig. 1
Fig. 1
Study selection for meta-analysis. Note: DMD Duchenne muscular dystrophy
Fig. 2
Fig. 2
Meta-analysis of life expectancy at birth in DMD, Note: BP Bisphosphonate, SES socio-economic status, DMD Duchenne muscular dystrophy, IPPV intermittent positive-pressure ventilation, NPV Negative pressure ventilation. In the absence of data, the sample studied by Passamano et al. [12] was assumed to be uniformly distributed across listed strata when calculating pooled estimates
See this image and copyright information in PMC

References

    1. Mendell JR, Shilling C, Leslie ND, et al. Evidence-based path to newborn screening for Duchenne muscular dystrophy. Ann Neurol. 2012;71(3):304–313. - PubMed
    1. Moat SJ, Bradley DM, Salmon R, Clarke A, Hartley L. Newborn bloodspot screening for Duchenne muscular dystrophy: 21 years experience in Wales (UK) Eur J Hum Genet. 2013;21(10):1049–1053. - PMC - PubMed
    1. Emery AE. The muscular dystrophies. Lancet. 2002;359(9307):687–695. - PubMed
    1. Birnkrant DJ, Bushby K, Bann CM, et al. Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management. Lancet Neurol. 2018;17(4):347–361. - PMC - PubMed
    1. Phillips MF, Quinlivan RC, Edwards RH, Calverley PM. Changes in spirometry over time as a prognostic marker in patients with Duchenne muscular dystrophy. Am J Respir Crit Care Med. 2001;164(12):2191–2194. - PubMed

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