Characteristics and outcomes of cardiomyopathy in children with Duchenne or Becker muscular dystrophy: A comparative study from the Pediatric Cardiomyopathy Registry

David M. Connuck, Lynn A. Sleeper, Steven D. Colan, Gerald F. Cox, Jeffrey A. Towbin, April M. Lowe, James D. Wilkinson, E. John Orav, Leigha Cuniberti, Bonnie A. Salbert, Steven E Lipshultz

Research output: Contribution to journalArticle

117 Citations (Scopus)

Abstract

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.

Original languageEnglish
Pages (from-to)998-1005
Number of pages8
JournalAmerican Heart Journal
Volume155
Issue number6
DOIs
StatePublished - Jun 1 2008

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Duchenne Muscular Dystrophy
Cardiomyopathies
Registries
Pediatrics
Dilated Cardiomyopathy
Dilatation
Transplants
Mitral Valve Insufficiency
Heart Transplantation
Left Ventricular Function
Echocardiography
Cohort Studies
Heart Failure
Heart Rate
Outcome Assessment (Health Care)

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine

Cite this

Characteristics and outcomes of cardiomyopathy in children with Duchenne or Becker muscular dystrophy : A comparative study from the Pediatric Cardiomyopathy Registry. / Connuck, David M.; Sleeper, Lynn A.; Colan, Steven D.; Cox, Gerald F.; Towbin, Jeffrey A.; Lowe, April M.; Wilkinson, James D.; Orav, E. John; Cuniberti, Leigha; Salbert, Bonnie A.; Lipshultz, Steven E.

In: American Heart Journal, Vol. 155, No. 6, 01.06.2008, p. 998-1005.

Research output: Contribution to journalArticle

Connuck, DM, Sleeper, LA, Colan, SD, Cox, GF, Towbin, JA, Lowe, AM, Wilkinson, JD, Orav, EJ, Cuniberti, L, Salbert, BA & Lipshultz, SE 2008, 'Characteristics and outcomes of cardiomyopathy in children with Duchenne or Becker muscular dystrophy: A comparative study from the Pediatric Cardiomyopathy Registry', American Heart Journal, vol. 155, no. 6, pp. 998-1005. https://doi.org/10.1016/j.ahj.2008.01.018
Connuck, David M. ; Sleeper, Lynn A. ; Colan, Steven D. ; Cox, Gerald F. ; Towbin, Jeffrey A. ; Lowe, April M. ; Wilkinson, James D. ; Orav, E. John ; Cuniberti, Leigha ; Salbert, Bonnie A. ; Lipshultz, Steven E. / Characteristics and outcomes of cardiomyopathy in children with Duchenne or Becker muscular dystrophy : A comparative study from the Pediatric Cardiomyopathy Registry. In: American Heart Journal. 2008 ; Vol. 155, No. 6. pp. 998-1005.
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abstract = "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.",
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T1 - Characteristics and outcomes of cardiomyopathy in children with Duchenne or Becker muscular dystrophy

T2 - A comparative study from the Pediatric Cardiomyopathy Registry

AU - Connuck, David M.

AU - Sleeper, Lynn A.

AU - Colan, Steven D.

AU - Cox, Gerald F.

AU - Towbin, Jeffrey A.

AU - Lowe, April M.

AU - Wilkinson, James D.

AU - Orav, E. John

AU - Cuniberti, Leigha

AU - Salbert, Bonnie A.

AU - Lipshultz, Steven E

PY - 2008/6/1

Y1 - 2008/6/1

N2 - 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.

AB - 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.

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