Clinical InvestigationCongestive Heart FailurePerindopril preventive treatment on mortality in Duchenne muscular dystrophy: 10 years' follow-up
Section snippets
Methods
The protocol of this study has been previously described in detail.14 Briefly, among 80 children screened at 10 medical centers (see Appendix A), 57 between the ages of 9.5 and 13 years, who had genetically confirmed DMD and an LVEF >55% measured by radionuclide ventriculography, were included in a 2-phase prospective study. Left ventricular ejection fraction was ≤55% in 20 patients, and DMD was not confirmed in 3 patients. Additional inclusion criteria included tolerance of a 1-mg test dose of
Results
The baseline characteristics of the study groups were similar (Table I). No other pharmaceutical agent than the study drugs was administered during phase I. During phase II, treatment with perindopril was continued; exact doses of perindopril were not recorded during that period, but all physicians reported that it was prescribed at maximum tolerated doses in all patients, as recommended. In addition, in the beginning of phase II, 4 patients in group 1 (initially allocated to perindopril) and 5
Discussion
This study documented a survival benefit conferred by the early, instead of delayed, administration of perindopril in patients with DMD between the ages of 9.5 and 13 years presenting with normal LVEF at entry in the study. Although several other studies have reported a lowering of mortality by ACEI in patients with congestive heart failure10 or a preventive effect in patients at high risk of adverse cardiovascular events,15 ours is the first to demonstrate a survival benefit conferred by ACEI
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2022, Molecular Genetics and MetabolismCitation Excerpt :This is of specific importance, as start of cardiac therapy after detection of CM via conventional echocardiography did not improve systolic left ventricular function [7]. Interestingly, for patients with Duchenne muscular dystrophy, there is evidence that if cardiac treatment is initiated before cardiac dysfunction is detected, onset of LV dysfunction may be delayed, and even mortality can be reduced [40–42]. Therefore, one could speculate that such prophylactic treatment may be beneficial also in PA patients.