title: Effect of perindopril on the onset and progression of left ventricular dysfunction in Duchenne muscular dystrophy.
abstract: OBJECTIVE
  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.
authors.affiliation: French Working Group of Heart Involvement in Myopathies Investigators, Paris, France.
authors.forename: Denis
authors.initials: D
authors.lastname: Duboc
authors.forename: Christophe
authors.initials: C
authors.lastname: Meune
authors.forename: Guy
authors.initials: G
authors.lastname: Lerebours
authors.forename: Jean-Yves
authors.initials: JY
authors.lastname: Devaux
authors.forename: Guy
authors.initials: G
authors.lastname: Vaksmann
authors.forename: Henri-Marc
authors.initials: HM
authors.lastname: Bécane
mesh_terms: D000293:Adolescent; D000806:Angiotensin-Converting Enzyme Inhibitors / Q000009:adverse effects / Q000627:therapeutic use*; D002648:Child; D002669:Child Welfare; D018450:Disease Progression; D004311:Double-Blind Method; D005500:Follow-Up Studies; D006801:Humans; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000503:physiopathology; D020913:Perindopril / Q000009:adverse effects / Q000627:therapeutic use*; D013318:Stroke Volume / Q000187:drug effects; D016896:Treatment Outcome; D018487:Ventricular Dysfunction, Left / Q000150:complications* / Q000188:drug therapy / Q000503:physiopathology; D016277:Ventricular Function, Left / Q000187:drug effects
chemical_list: D000806:Angiotensin-Converting Enzyme Inhibitors; D020913:Perindopril