Elsevier

The Journal of Pediatrics

Volume 163, Issue 4, October 2013, Pages 1080-1084.e1
The Journal of Pediatrics

Original Article
Oral Corticosteroids and Onset of Cardiomyopathy in Duchenne Muscular Dystrophy

https://doi.org/10.1016/j.jpeds.2013.05.060Get rights and content

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.

Section snippets

Methods

The Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) is a population-based surveillance program that collects standardized information on people with Duchenne and Becker muscular dystrophy from Arizona, Colorado, Georgia, Iowa, and western New York State. Data are collected from inpatient and outpatient records from multiple medical sources. The case definition for DMD is based on clinical phenotype and supportive dystrophin gene mutation and/or muscle biopsy

Results

Table I illustrates the composition of cases from the analytical dataset and compares it with the entire MD STARnet population. No significant differences were found between the sample and the MD STARnet population for race/ethnicity or patient distribution per surveillance site (state).

Of the 462 patients included in the analytical dataset, 291 were corticosteroid-treated with a mean treatment start age of 7.4 (2.5) years and a mean corticosteroid treatment duration of 4.1 (3.4) years. The

Discussion

Progressive cardiac dysfunction is a hallmark of DMD, although the time course for cardiomyopathy is not well characterized.3, 21 In our population-based cohort of 202 boys with DMD who developed cardiomyopathy, we found the mean age of cardiomyopathy onset was 14.3 years. This is comparable with previous studies documenting cardiomyopathy onset in patients with DMD.5, 18, 22 In a single institution study of 62 boys with DMD, Jefferies et al investigated cardiomyopathy as defined by reduced

References (30)

Cited by (110)

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    As most pediatric patients with DMD are asymptomatic from their cardiomyopathy, the timing of initiating treatment is somewhat controversial. Historically, many centers and practitioners have waited until the onset of disease detected by echocardiography before starting medical therapy [34,61–64]. However, there is accumulating data showing that initiation of medications such as angiotensin-converting enzyme inhibitors, angiotensin receptors blockers, mineralocorticoid receptor antagonists, and beta-adrenergic blockers may be beneficial in delaying disease progression when started before overt cardiomyopathy is present or early in the disease.

  • Comparison of the effect of three steroid regimens on cardiac function in Duchenne muscular dystrophy

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    Cardiac damage occurs slowly, most often in the form of dilated cardiomyopathy [1–3]. Studies have suggested that the average age for development of abnormal left ventricular systolic function is around 14 years [4–7]. Administration of steroids and initiation of therapy with ACE inhibitors before echocardiographic evidence of cardiac dysfunction delay the onset of clinical evidence of cardiac dysfunction and are associated with a significant reduction in heart failure related deaths. [5–9].

  • New diagnostic and therapeutic modalities in neuromuscular disorders in children

    2021, Current Problems in Pediatric and Adolescent Health Care
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Supported by the Centers for Disease Control and Prevention (Cooperative Agreement DD000187 for Surveillance and Epidemiologic Research of Duchenne and Becker Muscular Dystrophy). The authors declare no conflicts of interest.

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