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Multicenter Study
. 2020 Apr;41(4):764-771.
doi: 10.1007/s00246-020-02309-y. Epub 2020 Feb 3.

Risk Factors for Cardiac and Non-cardiac Causes of Death in Males with Duchenne Muscular Dystrophy

Carol A Wittlieb-Weber  1   2 , Kenneth R Knecht  3 , Chet R Villa  4 , Chentel Cunningham  5 , Jennifer Conway  5 , Matthew J Bock  6 , Katheryn E Gambetta  7 , Ashwin K Lal  8 , Kurt R Schumacher  9 , Sabrina P Law  10 , Shriprasad R Deshpande  11 , Shawn C West  12 , Joshua M Friedland-Little  13 , Irene D Lytrivi  10 , Michael A McCulloch  14 , Ryan J Butts  15 , David R Weber  16 , Jonathan N Johnson  17
Affiliations
Multicenter Study

Risk Factors for Cardiac and Non-cardiac Causes of Death in Males with Duchenne Muscular Dystrophy

Carol A Wittlieb-Weber et al. Pediatr Cardiol. 2020 Apr.

Abstract

As survival and neuromuscular function in Duchenne muscular dystrophy (DMD) have improved with glucocorticoid (GC) therapy and ventilatory support, cardiac deaths are increasing. Little is known about risk factors for cardiac and non-cardiac causes of death in DMD. A multi-center retrospective cohort study of 408 males with DMD, followed from January 1, 2005 to December 31, 2015, was conducted to identify risk factors for death. Those dying of cardiac causes were compared to those dying of non-cardiac causes and to those alive at study end. There were 29 (7.1%) deaths at a median age of 19.5 (IQR: 16.9-24.6) years; 8 (27.6%) cardiac, and 21 non-cardiac. Those living were younger [14.9 (IQR: 11.0-19.1) years] than those dying of cardiac [18 (IQR 15.5-24) years, p = 0.03] and non-cardiac [19 (IQR: 16.5-23) years, p = 0.002] causes. GC use was lower for those dying of cardiac causes compared to those living [2/8 (25%) vs. 304/378 (80.4%), p = 0.001]. Last ejection fraction prior to death/study end was lower for those dying of cardiac causes compared to those living (37.5% ± 12.8 vs. 54.5% ± 10.8, p = 0.01) but not compared to those dying of non-cardiac causes (37.5% ± 12.8 vs. 41.2% ± 19.3, p = 0.58). In a large DMD cohort, approximately 30% of deaths were cardiac. Lack of GC use was associated with cardiac causes of death, while systolic dysfunction was associated with death from any cause. Further work is needed to ensure guideline adherence and to define optimal management of systolic dysfunction in males with DMD with hopes of extending survival.

Keywords: Duchenne muscular dystrophy; Dystrophic cardiomyopathy; Heart failure; Sudden cardiac death.

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Conflict of interest statement

Conflict of interest No conflict of interest are reported for any of the authors on this manuscript.

Figures

Fig. 1
Fig. 1
Kaplan–Meier curve depicting survival probability at age of last follow-up in 407 boys with Duchenne muscular dystrophy. Number at risk is the number of subjects used in the denominator to determine the proportion of subjects surviving per decade of age at study end. Shaded gray area indicates the 95% confidence interval. The starting number is 1 less than the entire cohort due to an unknown death date for one subject
Fig. 2
Fig. 2
Cardiac function as measured by echocardiogram comparing the last ejection fraction and fractional shortening prior to death or study end
See this image and copyright information in PMC

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