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. 2014 Aug;50(2):250-6.
doi: 10.1002/mus.24163. Epub 2014 May 14.

Cooperative International Neuromuscular Research Group Duchenne Natural History Study demonstrates insufficient diagnosis and treatment of cardiomyopathy in Duchenne muscular dystrophy

Christopher Spurney  1 , Reiko Shimizu, Lauren P Morgenroth, Hanna Kolski, Heather Gordish-Dressman, Paula R Clemens; CINRG Investigators
Collaborators, Affiliations

Cooperative International Neuromuscular Research Group Duchenne Natural History Study demonstrates insufficient diagnosis and treatment of cardiomyopathy in Duchenne muscular dystrophy

Christopher Spurney et al. Muscle Nerve. 2014 Aug.

Abstract

Introduction: Cardiomyopathy is a common cause of morbidity and death in patients with Duchenne muscular dystrophy (DMD).

Methods: This investigation was a cross-sectional cross-sectional analysis of clinical data from the multi-institutional Cooperative International Neuromuscular Research Group (CINRG) DMD Natural History Study of 340 DMD patients aged 2-28 years. Cardiomyopathy was defined as shortening fraction (SF) <28% or ejection fraction (EF) <55%.

Results: Two hundred thirty-one participants reported a prior clinical echocardiogram study, and 174 had data for SF or EF. The prevalence of cardiomyopathy was 27% (47 of 174), and it was associated significantly with age and clinical stage. The association of cardiomyopathy with age and clinical stage was not changed by glucocorticoid use as a covariate (P > 0.68). In patients with cardiomyopathy, 57% (27 of 47) reported not taking any cardiac medications. Cardiac medications were used in 12% (15 of 127) of patients without cardiomyopathy.

Conclusions: We found that echocardiograms were underutilized, and cardiomyopathy was undertreated in this DMD natural history cohort.

Keywords: Duchenne muscular dystrophy; cardiomyopathy; echocardiogram; glucocorticoid; natural history.

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Conflict of interest statement

Conflict of Interest: On behalf of all authors, the corresponding author states that there is no conflict of interest.

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