Perindopril preserves left ventricular function in X-linked Duchenne muscular dystrophy

yDuchenne muscular dystrophy (DMD), an X-linked disorder due to a lack of dystrophin, is associated with muscle weakness and myocardial dysfunction. DMD children between the ages of 9.5 and 13 years with normal left ventricular ejection fraction were included in this prospective study. They were randomly assigned for 3 years to perindopril 2-4 mg (group 1) or placebo (group 2) in a double-blind protocol, followed by open-label treatment with perindopril for all patients. Left ventricular function was compared between the two groups at 5 years. A total of 28 patients were assigned to group 1 and 29 patients were assigned to group 2. Baseline characteristics were similar in both groups. At the end of the 5-year follow-up period, eight patients had an ejection fraction under 45% in the placebo group vs. one patient in the perindopril group (P < 0.02). All patients were alive in the perindopril group and three had died in the placebo group (P = 0.07). Early initiation of treatment with perindopril is associated with a preservation of left ventricular function in DMD children and with a trend towards a lower mortality.