Key result
Enalapril or carvedilol improves LV fractional shortening by ~1.5% in pediatric muscular dystrophy.
Why the study?
Cardiac function deteriorates over time in Duchenne and Becker muscular dystrophies leading to heart failure, and the effect of enalapril and carvedilol on LV dysfunction in this population was evaluated.
RCT (n=23)
Open-label
Randomized
No
Absolute Event Rate: 27.6% vs 26.1%
p-value: p=0.046
Hypothesis-generating for early cardioprotection in pediatric muscular dystrophy; randomized trials needed before practice change.
BACKGROUND AND OBJECTIVES: In Duchenne and Becker muscular dystrophies, cardiac function deteriorates with time resulting in heart failure which is often fatal. We prospectively evaluated the effect of enalapril and carvedilol on left ventricular (LV) dysfunction in middle childhood and adolescent patients with muscular dystrophy. SUBJECTS AND METHODS: Twenty-three patients with LV dysfunction (22 with Duchenne muscular dystrophy, 1 with Becker muscular dystrophy) were enrolled. We prescribed enalapril (13 patients) or carvedilol (10 patients) randomly from July 2008 to August 2010 and followed up the patients until September 2011. The changes in LV function parameters before and after the treatment were evaluated by echocardiography. RESULTS: The mean age at the start of treatment with enalapril or carvedilol was 12.6±3.7 years (median 13 years), and mean follow-up duration was 20.1±8.9 months. In the enalapril group, LV fractional shortening (FS) increased from 25.8±2.1 to 26.6±3.0 (p=0.241). In the carvedilol group, LV FS increased from 26.4±1.1 to 28.6±4.2 (p=0.110). In all 23 patients, LV FS significantly increased from 26.1±1.7 (before) to 27.6±3.7 (after treatment) (p<0.046). Indexed LV dimension at end diastole and LV end-diastolic volume decreased slightly, but without statistical significance by tri-plane volumetry. LV diastolic functional parameters were maintained during follow-up period. CONCLUSION: Enalapril or carvedilol could improve LV systolic function in middle childhood and adolescent patients with muscular dystrophy without significant adverse effects.
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Kwon et al. (2012) conducted an RCT in Duchenne and Becker muscular dystrophies with left ventricular dysfunction (n=23). Enalapril or Carvedilol vs. Baseline (before treatment) was evaluated on Left ventricular fractional shortening (LV FS) (p=0.046). Treatment with enalapril or carvedilol significantly improved left ventricular fractional shortening from 26.1% to 27.6% (p=0.046) in children and adolescents with muscular dystrophy.
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