Oral corticosteroids and onset of cardiomyopathy in Duchenne muscular dystrophy

J Pediatr. 2013 Oct;163(4):1080-4.e1. doi: 10.1016/j.jpeds.2013.05.060. Epub 2013 Jul 15.


Objective: To estimate the age when cardiomyopathy develops in boys with Duchenne muscular dystrophy (DMD) and to analyze the effect of corticosteroid treatment on the age of cardiomyopathy onset.

Study design: We identified a population-based sample of 462 boys with DMD, born between 1982 and 2005, in 5 surveillance sites in the US. Echocardiographic and corticosteroid treatment data were collected. Cardiomyopathy was defined by a reduced fractional shortening (<28%) or ejection fraction (<55%). The age of cardiomyopathy onset was determined. Survival analysis was performed to determine the effects of corticosteroid treatment on cardiomyopathy onset.

Results: The mean (SD) age of cardiomyopathy onset was 14.3 (4.2) years for the entire population and 15.2 (3.4) years in corticosteroid-treated vs 13.1 (4.8) in non-treated boys. Survival analysis described a significant delay of cardiomyopathy onset for boys treated with corticosteroids (P < .02). By 14.3 years of age, 63% of non-treated boys had developed cardiomyopathy vs only 36% of those treated. Among boys treated with corticosteroids, there is a significant positive effect of duration of corticosteroid treatment on cardiomyopathy onset (P < .0001). For every year of corticosteroid treatment, the probability of developing cardiomyopathy decreased by 4%.

Conclusions: Oral corticosteroid treatment was associated with delayed cardiomyopathy onset. The duration of corticosteroid treatment also correlated positively with delayed cardiomyopathy onset. Our analysis suggests that a boy with DMD treated for 5 years with corticosteroids might experience a 20% decrease in the likelihood of developing cardiomyopathy compared with untreated boys.

Keywords: ACE; Angiotensin-converting enzyme; DMD; Duchenne muscular dystrophy; EF; Ejection fraction; FS; Fractional shortening; LV; Left ventricle; MD STARnet; Muscular Dystrophy Surveillance, Tracking, and Research Network.

Publication types

  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Administration, Oral
  • Adolescent
  • Adrenal Cortex Hormones / administration & dosage
  • Adrenal Cortex Hormones / therapeutic use*
  • Age of Onset
  • Cardiomyopathies / complications
  • Cardiomyopathies / drug therapy
  • Cardiomyopathies / epidemiology*
  • Child
  • Child, Preschool
  • Echocardiography
  • Humans
  • Male
  • Muscular Dystrophy, Duchenne / complications*
  • Muscular Dystrophy, Duchenne / drug therapy*
  • Regression Analysis
  • Time Factors
  • Treatment Outcome


  • Adrenal Cortex Hormones