Original ArticleOral Corticosteroids and Onset of Cardiomyopathy in Duchenne Muscular Dystrophy
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
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2023, Progress in Pediatric CardiologyCitation Excerpt :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.
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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.