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

• Published in: BMJ open Vol. 8; no. 12; p. e022572
• Main Authors: Bourke, John P, Watson, Gillian, Muntoni, Francesco, Spinty, Stefan, Roper, Helen, Guglieri, Michela, Speed, Chris, McColl, Elaine, Chikermane, Ashish, Jayawant, Sandeep, Adwani, Satish, Willis, Tracey, Wilkinson, Jennifer, Bryant, Andrew, Chadwick, Thomas, Wood, Ruth, Bushby, Kate
• Format: Journal Article
• Language: English
• Published: England British Medical Journal Publishing Group 19.12.2018; BMJ Publishing Group LTD; BMJ Publishing Group
• Subjects: ace-inhibitor; Adolescent; Adrenergic beta-Antagonists - therapeutic use; Age; Angiotensin-Converting Enzyme Inhibitors - therapeutic use; Beta blockers; beta-blocker; cardiac dystrophinopathy; Cardiology; Cardiomyopathies - etiology; Cardiomyopathies - prevention & control; Cardiomyopathy; Cardiovascular Medicine; Child; Child, Preschool; Children & youth; Clinics; Drug Therapy, Combination; duchenne muscular dystrophy; Echocardiography; Electrocardiography; Evidence-based medicine; Forced Expiratory Volume; Health surveillance; Heart failure; Heart Ventricles - diagnostic imaging; Humans; Male; Medical prognosis; Mortality; Multicenter Studies as Topic; Muscular dystrophy; Muscular Dystrophy, Duchenne - complications; Patients; prophylactic therapy; Randomized Controlled Trials as Topic; Vital Capacity; United Kingdom > UK; prophylactic therapy; duchenne muscular dystrophy; ace-inhibitor; beta-blocker; cardiac dystrophinopathy

IntroductionAlthough 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....