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Science News: Life Expectancy in Duchenne Muscular Dystrophy: Reproduced Individual Patient Data Meta-Analysis

Submitted by: Niranjan Singh, MD

Broomfield J, Hill M, Guglieri M, Crowther M, Abrams K. Life expectancy in Duchenne muscular dystrophy: Reproduced individual patient data meta-analysis. Neurology. 2021 Dec 7;97(23):e2304-e2314. doi: 10.1212/WNL.0000000000012910. Epub 2021 Oct 13. PMID: 34645707.
Summary: Duchenne muscular dystrophy (DMD) is a rare progressive disease often diagnosed early childhood with reduced life expectancy. This is a systemic review of published literature on mortality in DMD up to July 2020 which focused on publications with Kaplan-Meier (KM) survival curves with age as a time scale. Of the 1,177 articles identified, 14 publications met the inclusion criteria providing data on 2,283 patients of whom 1,049 had died. Median life expectancy was 22 years. Analysis stratified by 3 time periods showed increased life expectancy in more recent patient populations born after 1990 had a median life expectancy of 28.1 years
Comments: The study shows that there is improvement in survival in patients born after 1990. This improvement may be due to improvement in standard of care, in particular the routine uses of mechanical ventilator. With current standards of care, many patients with DMD can now expect to live into their 4th decade of life. Similar studies have shown median life expectancy with ventilator support between 21- and 39.6-years verses without ventilator support between 14.4 and 27 years.
Articles of similar interest:
Kieny P, Chollet S, Delalande P, et al. Evolution of life expectancy of patients with duchenne muscular dystrophy at AFM Yolaine de Kepper centre between 1981 and 2011. Ann Phys Rehabil Med. 2013 Sep;56(6):443-54. doi: 10.1016/j.rehab.2013.06.002. Epub 2013 Jun 24. PMID: 23876223.
Landfeldt E, Thompson R, Sejersen T, McMillan HJ, Kirschner J, Lochmüller H. Life expectancy at birth in duchenne muscular dystrophy: A systematic review and meta-analysis. Eur J Epidemiol. 2020 Jul;35(7):643-653. doi: 10.1007/s10654-020-00613-8. Epub 2020 Feb 27. PMID: 32107739; PMCID: PMC7387367.

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