Characteristics and outcomes of cardiomyopathy in children with Duchenne or Becker muscular dystrophy: a comparative study from the Pediatric Cardiomyopathy Registry.
OBJECTIVE
The aim of this study was to determine in pediatric Duchenne (DMD) and Becker muscular dystrophy (BMD) or other dilated cardiomyopathies (ODCM) whether outcomes differ by diagnosis.


BACKGROUND
Children with dilated cardiomyopathy are treated as a single undifferentiated group.


METHODS
This cohort study of 128 children with DMD, 15 with BMD, and 312 with ODCM uses outcome measures of left ventricular (LV) size and function, death, heart transplant, and death or transplant.


RESULTS
At cardiomyopathy diagnosis, the DMD and BMD groups had similar mean ages (14.4 and 14.6 years), prevalence of congestive heart failure (CHF) (30% and 33%), and LV fractional shortening (FS) Z-scores (median, -5.2 for DMD and -6.7 for BMD). The BMD group had more severe mitral regurgitation (P = .05) and a higher mean LV end-diastolic dimension Z-score than the DMD group (2.9 +/- 1.5 vs 1.2 +/- 1.9, P = .002). Duchenne muscular dystrophy group survival was lower than in BMD or ODCM groups (P = .06) at 5 years (57%, 100%, and 71%, respectively). In BMD, 25% received cardiac transplants within 0.4 years of cardiomyopathy diagnosis. The combined DMD and BMD group had less LV dilation and a closer-to-normal LV FS at cardiomyopathy diagnosis than the ODCM group. After 2 years, LV dilation increased, and LV FS did not change in the combined DMD and BMD group; for ODCM patients, LV dilation did not progress, and LV FS improved.


CONCLUSIONS
Children with DMD and cardiomyopathy have a higher mortality. Becker muscular dystrophy has a high heart transplantation rate in the 5 years after diagnosis of cardiomyopathy. Serial echocardiography demonstrates a different disease course for DMD and BMD patients compared with ODCM patients.
Connuck
David M
DM
Janet Weis Children's Hospital, Geisinger Medical Center, Danville, PA, USA.
Sleeper
Lynn A
LA
Colan
Steven D
SD
Cox
Gerald F
GF
Towbin
Jeffrey A
JA
Lowe
April M
AM
Wilkinson
James D
JD
Orav
E John
EJ
Cuniberti
Leigha
L
Salbert
Bonnie A
BA
Lipshultz
Steven E
SE
D000293:Adolescent; D002311:Cardiomyopathy, Dilated / Q000000981:diagnostic imaging* / Q000209:etiology* / Q000401:mortality; D016022:Case-Control Studies; D002648:Child; D015331:Cohort Studies; D004452:Echocardiography; D005260:Female; D006801:Humans; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000150:complications* / Q000401:mortality