Corticosteroid treatment retards development of ventricular dysfunction in Duchenne muscular dystrophy

Approaching a New Age in Duchenne Muscular Dystrophy Treatment

“…10,22 Recently, a historical cohort study of DMD cases undergoing serial cardiac evaluations over 3 years showed that corticosteroid treatment begun prior to ventricular dysfunction was protective against development of ventricular dysfunction, defined as a shortening fraction less than 28%. 23 The probability of freedom from ventricular dysfunction by Kaplan-Meier estimates was a striking 93% for the steroid-treated cohort versus 53% for the untreated cohort at 1500 days. 23 Early angiotensin-converting enzyme inhibitor (ACEi) treatment may also be indicated in DMD as suggested by a trial of perindopriol by Duboc and colleagues.…”

“…23 The probability of freedom from ventricular dysfunction by Kaplan-Meier estimates was a striking 93% for the steroid-treated cohort versus 53% for the untreated cohort at 1500 days. 23 Early angiotensin-converting enzyme inhibitor (ACEi) treatment may also be indicated in DMD as suggested by a trial of perindopriol by Duboc and colleagues. 24 In this double-blind study, children from 9.5 to 13 years of age with DMD and normal cardiac function were treated with peridopril or placebo for 3 years, and then all participants received open-label peridopriol for an additional 2 years.…”

Psychosocial Support Needs of Families of Boys with Duchenne Muscular Dystrophy

“…Therapeutic interventions include the use of corticosteroids (such as prednisone or deflazacort) for skeletal muscle weakness and afterload reduction (such as angiotensin converting enzyme inhibitor or beta-blocker) for cardiomyopathy. Corticosteroid therapy offers benefit to DMD boys by improving muscle strength and function [Mendell et al, 1989;Griggs et al, 1991], prolonging independent ambulation [Biggar et al, 2001;Schara et al, 2001], plus slowing the progression of cardiomyopathy [Markham et al, 2008] and scoliosis [Kinali et al, 2007]. As well, the introduction of noninvasive positive pressure ventilation has prolonged the survival of individuals with DMD [Bach & Martinez, 2011].…”

Duchenne muscular dystrophy; a cardiomyopathy that can be prevented?

“…The prognostic significance of these findings warrants further longitudinal follow-up studies to determine whether late gadolinium enhancement precedes a decrease in cardiac function, and whether early pharmaceutical interventions are useful in preventing progression of Duchenne muscular dystrophy. Recent clinical studies with corticosteriods have already shown promising results by their potential to retard development of left ventricular dysfunction [33,34]. Mavrogeni et al [33] showed that Duchenne patients on deflazacort are characterized by better preservation of the CMR T2 relaxation time of the myocardium and an improved left ventricular systolic function.…”

“…Mavrogeni et al [33] showed that Duchenne patients on deflazacort are characterized by better preservation of the CMR T2 relaxation time of the myocardium and an improved left ventricular systolic function. Markham et al [34] demonstrated that treatment of Duchenne patients with steroids was protective against left ventricular dysfunction. This indicates that steroid treatment, when started prior to ventricular dysfunction, retarded the anticipated development of ventricular dysfunction in Duchenne patients.…”