Early treatment with perindopril delayed the onset of left ventricular dysfunction (LVEF <45%) in children with Duchenne muscular dystrophy compared to delayed treatment (4.2% vs 29.6%; p=0.02).
RCT (n=57)
double-blind
randomized
Yes
Does early treatment with perindopril delay the onset and progression of left ventricular dysfunction in children with Duchenne muscular dystrophy?
Early prophylactic treatment with perindopril in children with Duchenne muscular dystrophy significantly reduces the long-term risk of developing prominent left ventricular dysfunction.
Absolute Event Rate: 4.2% vs 29.6%
p-value: p=0.02
OBJECTIVES: The aim of this research was to examine the effects of perindopril on cardiac function in patients with Duchenne muscular dystrophy (DMD). BACKGROUND: Duchenne muscular dystrophy, an inherited X-linked disease, is characterized by progressive muscle weakness and myocardial involvement. METHODS: In phase I, 57 children with DMD and a left ventricular ejection fraction (LVEF) >55% (mean 65.0 +/- 5.4%), 9.5 to 13 years of age (mean 10.7 +/- 1.2 years), were enrolled in a three-year multicenter, randomized, double-blind trial of perindopril, 2 to 4 mg/day (group 1), versus placebo (group 2). In phase II, all patients received open-label perindopril for 24 more months; LVEF was measured at 0, 36, and 60 months. RESULTS: Phase I was completed by 56 (27 in group 1 and 29 in group 2) and phase II by 51 patients (24 in group 1 and 27 in group 2). There was no difference in baseline characteristics between the treatment groups. At the end of phase I, mean LVEF was 60.7 +/- 7.6% in group 1 versus 64.4 +/- 9.8% in group 2, and was <45% in a single patient in each group (p = NS). At 60 months, LVEF was 58.6 +/- 8.1% in group 1 versus 56.0 +/- 15.5% in group 2 (p = NS). A single patient had an LVEF <45% in group 1 versus eight patients in group 2 (p = 0.02). CONCLUSIONS: Early treatment with perindopril delayed the onset and progression of prominent left ventricle dysfunction in children with DMD.
“For the first time we have shown that it is possible to slow down the progression of this rare degenerative disease. In DMD the heart muscles are affected and heart problems prove fatal in around 40% of children with DMD.”
Duboc et al. (2005) conducted an RCT in Duchenne muscular dystrophy (n=57). perindopril vs. placebo was evaluated on LVEF <45% at 60 months (p=0.02). Early treatment with perindopril delayed the onset of left ventricular dysfunction (LVEF <45%) in children with Duchenne muscular dystrophy compared to delayed treatment (4.2% vs 29.6%; p=0.02).