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Lessons learned from the Pediatric Cardiomyopathy Registry (PCMR) Study Group*

Published online by Cambridge University Press:  17 September 2015

James D. Wilkinson
Affiliation:
Department of Pediatrics, Wayne State University School of Medicine, Children’s Research Center of Michigan, Detroit, Michigan, United States of America
Joslyn A. Westphal
Affiliation:
Department of Pediatrics, Wayne State University School of Medicine, Children’s Research Center of Michigan, Detroit, Michigan, United States of America
Neha Bansal
Affiliation:
Department of Pediatrics, Detroit Medical Center, Children’s Hospital of Michigan, Detroit, Michigan, United States of America
Jason D. Czachor
Affiliation:
Department of Pediatrics, Wayne State University School of Medicine, Children’s Research Center of Michigan, Detroit, Michigan, United States of America
Hiedy Razoky
Affiliation:
Department of Pediatrics, Wayne State University School of Medicine, Children’s Research Center of Michigan, Detroit, Michigan, United States of America
Steven E. Lipshultz*
Affiliation:
Department of Pediatrics, Wayne State University School of Medicine, Children’s Research Center of Michigan, Detroit, Michigan, United States of America University of Miami Miller School of Medicine, Miami, Florida, United States of America
*
Correspondence to: S. E. Lipshultz, MD, Department of Pediatrics, Wayne State University School of Medicine, 3901 Beaubien Boulevard, 1K40, Detroit, MI 48201, United States of America.Tel: +313 745 5870; Fax: +313 993 0390; E-mail: slipshultz@med.wayne.edu

Abstract

Cardiomyopathy is a rare disorder of the heart muscle, affecting 1.13 cases per 100,000 children, from birth to 18 years of age. Cardiomyopathy is the leading cause of heart transplantation in children over the age of 1. The Pediatric Cardiomyopathy Registry funded in 1994 by the National Heart, Lung, and Blood Institute was established to examine the epidemiology of the disease in children below 18 years of age. More than 3500 children across the United States and Canada have been enrolled in the Pediatric Cardiomyopathy Registry, which has followed-up these patients until death, heart transplantation, or loss to follow-up. The Pediatric Cardiomyopathy Registry has provided the most in-depth illustration of this disease regarding its aetiology, clinical course, associated risk factors, and patient outcomes. Data from the registry have helped in guiding the clinical management of cardiomyopathy in children under 18 years of age; however, questions still remain regarding the most clinically effective diagnostic and treatment approaches for these patients. Future directions of the registry include the use of next-generation whole-exome sequencing and cardiac biomarkers to identify aetiology-specific treatments and improve diagnostic strategies. This article provides a brief synopsis of the work carried out by the Pediatric Cardiomyopathy Registry since its inception, including the current knowledge on the aetiologies, outcomes, and treatments of cardiomyopathy in children.

Type
Original Articles
Copyright
© Cambridge University Press 2015 

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Footnotes

*

Presented at Johns Hopkins All Children’s Heart Institute, International Pediatric Heart Failure Summit, Saint Petersburg, Florida, United States of America, 4–5 February, 2015.

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