Randomised placebo-controlled trial of combination ACE inhibitor and beta-blocker therapy to prevent cardiomyopathy in children with Duchenne muscular dystrophy? (DMD Heart Protection Study): a protocol study.

Bourke, John P and Watson, Gillian and Muntoni, Francesco and Spinty, Stefan and Roper, Helen and Guglieri, Michela and Speed, Chris and McColl, Elaine and Chikermane, Ashish and Jayawant, Sandeep and Adwani, Satish and Willis, Tracey and Wilkinson, Jennifer and Bryant, Andrew and Chadwick, Thomas and Wood, Ruth and Bushby, Kate (2018) Randomised placebo-controlled trial of combination ACE inhibitor and beta-blocker therapy to prevent cardiomyopathy in children with Duchenne muscular dystrophy? (DMD Heart Protection Study): a protocol study. BMJ open, 8 (12). e022572. ISSN 2044-6055. (In Press) This article is available to all HEFT staff and students via ASK Discovery tool http://tinyurl.com/z795c8c by using their HEFT Athens login IDs

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Abstract

INTRODUCTION

Although cardiologists were 'late-comers' to the multidisciplinary team-contributing to the complex care of patients with Duchenne muscular dystrophy (DMD), they now recognise the importance of systematic cardiac surveillance and timely therapy to prolonged survival in patients with DMD. Empirical deployment of cardioactive medications has already improved outcomes, but the evidence base for clinical decision making is weak. Fundamental questions remain as to whether prophylactic therapy is justified and convincingly superior to prompt deployment of the same therapies once left ventricular (LV) dysfunction is detected. Even if it were, at what age should therapy be introduced and with what specific drugs?

METHODS AND ANALYSIS

We are conducting a multicentre, parallel group, randomised, placebo-controlled study of combination therapy with an ACE inhibitor (perindopril) and a beta-blocker (bisoprolol) in boys with DMD aged 5-13 years, with normal LV function by echocardiographic criteria at the time of recruitment. Boys are being followed-up for a minimum of 3 years and a maximum of 5 years and undergo repeat assessments of LV function, heart rate and ECG, forced expiratory volume in the 1 s and forced vital capacity, adverse event reporting and quality of life at 6 monthly intervals.The primary outcome is change in LV function between active and placebo-treated participants over the course of the study.

ETHICS AND DISSEMINATION

The study was approved by 'NRES Committee East Midlands - Derby'. The results will be disseminated through manuscript publications, an international workshop and presentations to scientific meetings and parent forums.

TRANSLATIONAL ASPECTS

The study seeks to establish the evidence for prophylactic heart therapies for children with DMD, define the optimum age for their introduction and identify any safety concerns.

ARTICLE SUMMARY

The protocol describes the design of an ongoing multicentre, double-blind, randomised placebo-controlled study to establish the evidence for the use of prophylactic heart therapies in children with DMD, define the optimum age for their introduction and identify any safety concerns.

TRIAL REGISTRATION NUMBERS

EudraCT2007-005932-10 and ISRCTN50395346; Pre-results.

Item Type: Article
Additional Information: This article is available to all HEFT staff and students via ASK Discovery tool http://tinyurl.com/z795c8c by using their HEFT Athens login IDs
Subjects: WG Cardiovascular system. Cardiology
WS Paediatrics. Child health
Divisions: Womens and Childrens > Paediatrics
Related URLs:
Depositing User: Miss Emily Johnson
Date Deposited: 31 Dec 2018 10:36
Last Modified: 31 Dec 2018 10:36
URI: http://www.repository.heartofengland.nhs.uk/id/eprint/1807

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