PT  - JOURNAL ARTICLE
AU  - John P. Bourke
AU  - Gillian Watson
AU  - Stefan Spinty
AU  - Andrew Bryant
AU  - Helen Roper
AU  - Thomas Chadwick
AU  - Ruth Wood
AU  - Elaine McColl
AU  - Kate Bushby
AU  - Francesco Muntoni
AU  - Michela Guglieri
AU  - for the DMD Heart Study Group
TI  - Preventing Cardiomyopathy in DMD
AID  - 10.1212/CPJ.0000000000001023
DP  - 2021 Oct 01
TA  - Neurology: Clinical Practice
PG  - e661--e668
VI  - 11
IP  - 5
4099  - http://cp.neurology.org/content/11/5/e661.short
4100  - http://cp.neurology.org/content/11/5/e661.full
AB  - Objective To determine whether a combination of 2 heart medications would be tolerated and could prevent/delay the onset of cardiomyopathy in boys with Duchenne muscular dystrophy (DMD) compared with placebo.Methods This multicenter, parallel group, 1:1 patient randomized, placebo-controlled study of prophylactic perindopril and bisoprolol recruited boys with DMD aged 5–13 years, with normal ventricular function. Repeat assessments of left ventricular (LV) function, electrocardiogram, and adverse event reporting were performed 6 monthly. The primary outcome was change in ejection fraction between arms after 36 months. The study was approved by the National Research Ethics Service Committee East Midlands—Derby.Results Eighty-five boys were recruited (76% on steroid therapy) and randomized to combination heart drugs or matched placebo. Group change in left ventricular ejection fraction (LVEF%) at 36 months from baseline was −2.2% ± 6.0% and −2.9% ± 6.1% in active and placebo arms (adjusted mean difference: −2.1, 95% CI −5.2 to 1.1). There was no difference between treatment arms over repeated assessments (analysis of variance) up to 36 months (trial arms p = 0.53); arm-over-time (p = 0.44). Four participants on placebo but none on active therapy were withdrawn due to deteriorations in LV function. Secondary outcomes did not differ between arms either. Thirty-six serious adverse events occurred none due to cardiac events or trial medication.Conclusions Combination therapy was well tolerated. Consistent with the previous prophylactic perindopril heart study, there was no evidence of group benefit after 36-month treatment.Classification of Evidence This study provides Class I evidence that combination perindopril-bisoprolol therapy was well tolerated but did not change decline in LVEF significantly in boys with DMD.