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The Value of National Institutes of Health (NIH) Registry-Based Research in Identifying Childhood Cardiac Disease Outcomes: The Pediatric Cardiomyopathy Registry Experience

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Pediatric and Congenital Cardiac Care

Abstract

Cardiomyopathy is a serious, but rare, disease of the heart muscle. Cardiomyopathy commonly results in heart failure and is the leading cause of heart transplantation in children older than 1 year of age. The Pediatric Cardiomyopathy Registry (PCMR), which has been funded by the National Heart Lung and Blood Institute (NHBLI) since 1994, has enrolled and followed more than 3,500 children with cardiomyopathy in the US and Canada. Results from the registry have established the incidence of pediatric cardiomyopathy in North America, determined the prevalence of heart failure, have identified causes of cardiomyopathy, as well as transplant and survival patterns. In this chapter, we will describe the design and operation of the PCMR and present a summary of results. The PCMR is an example of how a well-designed and well-conducted patient registry can provide important insights into the etiologies, clinical course, and patient outcomes for a rare disease, in this case cardiomyopathy in children.

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Acknowledgements

The work of the PCMR would not have been possible without the collaboration of many physicians and other health professionals, scientists, and research staff from the United States and Canada. Special acknowledgement should be given to our PCMR investigators: Steven D. Colan, MD; Gerald Cox MD, PhD; Jane Messere, RN; Lynn Sleeper, ScD; Jeffrey A. Towbin, MD; Stephanie Ware, MD, PhD; John Lynn Jefferies, MD, MPH; E. John Orav, PhD; Daphne Hsu, MD; Steven Webber, MBChB; Charles Canter, MD; Linda Addonizio, MD; Beth Kaufman, MD; Melanie Everitt, MD; Elfriede Pahl, MD; Paul Kantor, MBBCh; Paolo Rusconi, MD; Robert E. Shaddy, MD; Paul R. Lurie, MD; David Connuck, MD; April Lowe, MS; Bonnie Salbert, DO; Tajinder Singh, MD, MSc; Jorge Alvarez, AB; Biagio Pietra, MD; Ranae Larsen, MD; Jacqueline Lamour, MD; Renee Margossian, MD; Beth Kaufman, MD; Susan Foerster, MD; Wendy Chung, MD; Rakesh Singh, MD, MS; and Juanita Hunter, MD.

We would also like to acknowledge Mrs. Lisa Yue and the Children’s Cardiomyopathy Foundation for their continuing support of the PCMR.

And finally, we would like to express our most sincere gratitude to the children with cardiomyopathy and their families whose participation has made the PCMR possible.

Author information

Authors and Affiliations

  1. Department of Pediatrics, Children’s Research Center of Michigan, Wayne State University School of Medicine, 3901 Beaubien Blvd, 5195 Carls Bldg, Detroit, MI, 48201, USA

    James D. Wilkinson MD, MPH & Joslyn A. Westphal BA, MPH

  2. Department of Pediatrics, University of Miami Miller School of Medicine, 1601 NW 12th Ave, 9th Floor, Miami, FL, 33133, USA

    Samuel W. Ross BA & Danielle D. Dauphin BA

  3. Carman and Ann Adams Department of Pediatrics, Wayne State University School of Medicine, University Pediatricians, Children’s Hospital of Michigan, Detroit Medical Center, Children’s Research Center of Michigan, 3901 Beaubien Boulevard, 1K40, Detroit, MI, 48201-2196, USA

    Steven E. Lipshultz MD, FAAP, FAHA

Authors
  1. James D. Wilkinson MD, MPH
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  2. Joslyn A. Westphal BA, MPH
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  3. Samuel W. Ross BA
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  4. Danielle D. Dauphin BA
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  5. Steven E. Lipshultz MD, FAAP, FAHA
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Corresponding author

Correspondence to Steven E. Lipshultz MD, FAAP, FAHA .

Editor information

Editors and Affiliations

  1. School of Medicine,, University of Oslo, Oslo, Norway

    Paul R. Barach

  2. Division of Cardiac Surgery, Department of Surgery, Johns Hopkins University, Division of Cardiac Surgery, Department of Surgery, Johns Hopkins All Children’s Heart Institute, All Children’s Hospital and Florida Hospital for Children, Johns Hopkins University, St. Petersburg, Tampa, Orlando, FL, USA,, Baltimore, Maryland, USA

    Jeffery P. Jacobs

  3. Wayne State University, Detroit, Michigan, USA

    Steven E. Lipshultz

  4. The Hospital for Sick Children, Toronto, Ontario, Canada

    Peter C. Laussen

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© 2015 Springer-Verlag London

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Wilkinson, J.D., Westphal, J.A., Ross, S.W., Dauphin, D.D., Lipshultz, S.E. (2015). The Value of National Institutes of Health (NIH) Registry-Based Research in Identifying Childhood Cardiac Disease Outcomes: The Pediatric Cardiomyopathy Registry Experience. In: Barach, P., Jacobs, J., Lipshultz, S., Laussen, P. (eds) Pediatric and Congenital Cardiac Care. Springer, London. https://doi.org/10.1007/978-1-4471-6587-3_34

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  • DOI: https://doi.org/10.1007/978-1-4471-6587-3_34

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  • Online ISBN: 978-1-4471-6587-3

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