Author + information
- Received March 2, 2004
- Revision received August 5, 2004
- Accepted September 13, 2004
- Published online March 15, 2005.
- Denis Duboc, MD, PhD*,* (, )
- Christophe Meune, MD*,
- Guy Lerebours, MD†,
- Jean-Yves Devaux, MD, PhD*,
- Guy Vaksmann, MD* and
- Henri-Marc Bécane, MD*
- ↵*Reprint requests and correspondence:
Dr. Denis Duboc, Department of Cardiology, Cochin Hospital, 27 rue du Faubourg St-Jacques, 75014 Paris, France
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.
This study was supported by grants from the French Association Against Myopathies and from Servier Laboratories.
- Received March 2, 2004.
- Revision received August 5, 2004.
- Accepted September 13, 2004.
- American College of Cardiology Foundation