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CLINICAL RESEARCH: HEART FAILURE

Effect of perindopril on the onset and progression of left ventricular dysfunction in Duchenne muscular dystrophy

Denis Duboc, MD, PhD^*,*, Christophe Meune, MD^*, Guy Lerebours, MD, Jean-Yves Devaux, MD, PhD^*, Guy Vaksmann, MD^* and Henri-Marc Bécane, MD^*

^* French Working Group of Heart Involvement in Myopathies Investigators, Paris, France
Servier Laboratories, Paris, France

Manuscript received March 2, 2004; revised manuscript received August 5, 2004, accepted September 13, 2004.

^* Reprint requests and correspondence: Dr. Denis Duboc, Department of Cardiology, Cochin Hospital, 27 rue du Faubourg St-Jacques, 75014 Paris, France (Email: denis.duboc{at}cch.ap-hop-paris.fr).

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.

Abbreviations and Acronyms   ACEI = angiotensin-converting enzyme inhibitors   CHF = congestive heart failure   DMD = Duchenne muscular dystrophy   LV = left ventricle/ventricular   LVEF = left ventricular ejection fraction

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