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Preventing Cardiomyopathy in DMD

Lookup NU author(s): Dr John Bourke, Gillian WatsonORCiD, Andrew Bryant, Dr Thomas Chadwick, Ruth Wood, Emerita Professor Elaine McCollORCiD, Emerita Professor Katherine Bushby, Professor Michela GuglieriORCiD


Full text for this publication is not currently held within this repository. Alternative links are provided below where available.


© 2022 American Academy of Neurology.ObjectiveTo 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.MethodsThis 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.ResultsEighty-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 Four participants on placebo but none on active therapy were withdrawn due to deteriorations in LV function.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.ConclusionsCombination 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 EvidenceThis 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.

Publication metadata

Author(s): Bourke JP, Watson G, Spinty S, Bryant A, Roper H, Chadwick T, Wood R, Mccoll E, Bushby K, Muntoni F, Guglieri M

Publication type: Article

Publication status: Published

Journal: Neurology: Clinical Practice

Year: 2021

Volume: 11

Issue: 5

Pages: E661-E668

Print publication date: 01/10/2021

Online publication date: 03/12/2020

Acceptance date: 06/11/2020

ISSN (print): 2163-0402

ISSN (electronic): 2163-0933

Publisher: Lippincott Williams and Wilkins


DOI: 10.1212/CPJ.0000000000001023


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