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).


CONCLUSIONS
Early initiation of treatment with perindopril is associated with a lower mortality in patients with DMD with normal LV ejection fraction at study entry.
Duboc
Denis
D
Department of Cardiology, Cochin Hospital, APHP, Paris V René Descartes University, Paris, France.
Meune
Christophe
C
Pierre
Bertrand
B
Wahbi
Karim
K
Eymard
Bruno
B
Toutain
Annick
A
Berard
Carole
C
Vaksmann
Guy
G
Weber
Simon
S
Bécane
Henri-Marc
HM
D000293:Adolescent; D000806:Angiotensin-Converting Enzyme Inhibitors / Q000627:therapeutic use*; D002648:Child; D004311:Double-Blind Method; D005500:Follow-Up Studies; D006801:Humans; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000401:mortality* / Q000517:prevention & control; D020913:Perindopril / Q000627:therapeutic use*; D015996:Survival Rate; D013997:Time Factors
D000806:Angiotensin-Converting Enzyme Inhibitors; D020913:Perindopril