This project was funded through grants from the U.S. Department of Education/NIDRR (#H133B031118, #H133B090001), the NIH/NINDS Paul D. Wellstone Muscular Dystrophy Research Centers (U54HD053177), the U.S. Department of Defense (#W81XWH-09-1-0592) and NIH/NCRR (#UL1RR031988).
Cooperative international neuromuscular research group duchenne natural history study demonstrates insufficient diagnosis and treatment of cardiomyopathy in duchenne muscular dystrophy
Version of Record online: 14 MAY 2014
Copyright © 2014 Wiley Periodicals, Inc.
Muscle & Nerve
Volume 50, Issue 2, pages 250–256, August 2014
How to Cite
Spurney, C., Shimizu, R., Morgenroth, L. P., Kolski, H., Gordish-Dressman, H., Clemens, P. R. and the CINRG Investigators (2014), Cooperative international neuromuscular research group duchenne natural history study demonstrates insufficient diagnosis and treatment of cardiomyopathy in duchenne muscular dystrophy. Muscle Nerve, 50: 250–256. doi: 10.1002/mus.24163
Disclosure: The authors take full responsibility for the contents of this paper, which do not represent the views of the Department of Veterans Affairs or the United States Government.
- Issue online: 21 JUL 2014
- Version of Record online: 14 MAY 2014
- Accepted manuscript online: 3 JAN 2014 01:12AM EST
- Manuscript Accepted: 31 DEC 2013
- Manuscript Revised: 18 DEC 2013
- Manuscript Received: 22 MAR 2013
- Duchenne muscular dystrophy;
- natural history
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. Muscle Nerve 50:250–256, 2014