Perindopril preventive treatment on mortality in Duchenne muscular dystrophy: 10 years' follow-up.

Background: Duchenne muscular dystrophy (DMD), an X-linked disorder due to lack of dystrophin, is associated with muscle weakness and myocardial dysfunction. Although preliminary data support the efficacy of angiotensin-converting enzyme inhibitors on left ventricular (LV) function, our aim was to examine the long-term impact of a preventive treatment with perindopril on mortality in children with DMD.

Methods: Patients with DMD between the ages of 9.5 and 13 years presenting with normal LV ejection fraction were included in this prospective study. They were randomly assigned for 3 years to perindopril, 2 to 4 mg (group 1), or placebo (group 2) in a double-blind protocol, followed by open-label treatment with perindopril for up to 10 years. Survival rate at 10 years in each group is reported.

Results: There were 28 patients assigned to group 1 and 29 to group 2. Baseline characteristics were similar in both groups. At the end of the 10 years' follow-up period, survival status was available for all included patients: 26 (92.9%) of 28 patients in group 1 were alive at 10 years versus 19 (65.5%) of 29 in group 2 (P = .02). Kaplan-Meier cumulative survival was significantly lower in group 2 than in group 1 (P = .013).

Conclusion: Early initiation of treatment with perindopril is associated with a lower mortality in patients with DMD with normal LV ejection fraction at study entry.