Effect of perindopril on the onset and progression of left ventricular dysfunction in Duchenne muscular dystrophy
D Duboc, C Meune, G Lerebours, JY Devaux… - Journal of the American …, 2005 - jacc.org
D Duboc, C Meune, G Lerebours, JY Devaux, G Vaksmann, HM Bécane
Journal of the American College of Cardiology, 2005•jacc.orgObjectives: 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 …
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 …
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.
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