Clinical InvestigationCongestive Heart FailureCharacteristics and outcomes of cardiomyopathy in children with Duchenne or Becker muscular dystrophy: A comparative study from the Pediatric Cardiomyopathy Registry☆
Section snippets
The Pediatric Cardiomyopathy Registry
This report is based on PCMR data as of April 2005 on 3045 patients (newborn to 18 years at diagnosis) who presented with cardiomyopathy to a pediatric cardiologist in 1990 or later. Each case is classified according to morphology as dilated, hypertrophic, restrictive, mixed, or other type of cardiomyopathy. Cases are further classified as “definite” if specified quantitative strict echocardiographic criteria of left ventricular (LV) dilation and systolic dysfunction are met, if the pattern of
Comparison of DMD and BMD Groups
The DMD and BMD groups had a similar age at cardiomyopathy diagnosis (14.4 ± 2.3 vs 14.6 ± 2.0 years) and rates of CHF at diagnosis (30% vs 33%) (Table I).
Echocardiographic findings
At the time of cardiomyopathy diagnosis, the BMD group had larger LV end-diastolic and LV end-systolic dimension Z-scores than did the DMD group (Table II) but similar LV fractional shortening (FS) and LV wall thickness. Although qualitative echocardiographic data were available for only 40 of the 143 children with DMD or BMD, the BMD group
Echocardiographic findings
Patients with BMD do not usually experience marked cardiomyopathy as children, but when they do, they present with a higher degree of mitral and tricuspid regurgitation, LV dilation, and CHF than do children with DMD. Because BMD children have fewer neuromuscular symptoms until they reach older ages, they may not be diagnosed until they present with CHF or cardiac symptoms.
Children and adolescents with ODCM had a higher incidence of CHF at the time of diagnosis than did those in the DMD + BMD
Conclusion
We have shown that a subset of patients with DMD and BMD has clinically important cardiac dysfunction during childhood and that BMD patients have more severe LV dilation and valvular regurgitation at the time of presentation with cardiomyopathy than do DMD patients. The mortality rate for DMD patients with cardiomyopathy is significantly worse than that of BMD patients (who often undergo transplant) and similarly aged myocarditis and idiopathic dilated cardiomyopathy patients; however, rates of
References (19)
- et al.
107th ENMC International Workshop: the management of cardiac involvement in muscular dystrophy and myotonic dystrophy. 7th-9th June 2002, Naarden, the Netherlands
Neuromuscul Disord
(2003) - et al.
Cardiomyopathy in neuromuscular disorders
Prog Pediatr Cardiol
(2007) - et al.
Cardioprotection for Duchenne's muscular dystrophy
Am Heart J
(1999) - et al.
Effect of perindopril on the onset and progression of left ventricular dysfunction in Duchenne muscular dystrophy
J Am Coll Cardiol
(2005) - et al.
Improved outcomes of pediatric dilated cardiomyopathy with utilization of heart transplantation
J Am Coll Cardiol
(2004) - et al.
Myocardial delayed enhancement by magnetic resonance imaging in patients with muscular dystrophy
J Am Coll Cardiol
(2007) Realizing optimal care for children with cardiovascular disease: Funding challenges and research approaches
Prog Pediatr Cardiol
(2005)- et al.
Dystrophies and heart disease
Curr Opin Cardiol
(1997) - et al.
The heart in human dystrophinopathies
Cardiology
(2003)
Cited by (174)
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2022, Journal of Cardiovascular Magnetic ResonanceCardiac MR Imaging of Muscular Dystrophies
2022, Current Problems in Diagnostic Radiology
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This study was supported by the National Heart, Lung, and Blood Institute/Department of Health and Human Services grant R01 HL53392, Bethesda, MD, the Children's Cardiomyopathy Foundation, Tenafly, NJ, and the Muscular Dystrophy Association, Tuscon, AZ.