The value of national institutes of health (NIH) registry-based research in identifying childhood cardiac disease outcomes

The pediatric cardiomyopathy registry experience

James D. Wilkinson, Joslyn A. Westphal, Samuel W. Ross, Danielle D. Dauphin, Steven E Lipshultz

Research output: Chapter in Book/Report/Conference proceedingChapter

1 Citation (Scopus)

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.

Original languageEnglish (US)
Title of host publicationPediatric and Congenital Cardiac Care: Volume 1: Outcomes Analysis
PublisherSpringer-Verlag London Ltd
Pages445-463
Number of pages19
ISBN (Print)9781447165873, 9781447165866
DOIs
StatePublished - Jan 1 2015
Externally publishedYes

Fingerprint

National Institutes of Health (U.S.)
Cardiomyopathies
Registries
Heart Diseases
Pediatrics
Research
Rare Diseases
Heart Failure
National Heart, Lung, and Blood Institute (U.S.)
Heart Transplantation
North America
Canada
Myocardium
Transplants
Survival
Incidence

Keywords

  • Cardiomyopathy
  • Heart failure
  • Patient registry
  • Pediatric cardiomyopathy registry
  • Pediatrics

ASJC Scopus subject areas

  • Medicine(all)

Cite this

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 Pediatric and Congenital Cardiac Care: Volume 1: Outcomes Analysis (pp. 445-463). Springer-Verlag London Ltd. https://doi.org/10.1007/978-1-4471-6587-3_34

The value of national institutes of health (NIH) registry-based research in identifying childhood cardiac disease outcomes : The pediatric cardiomyopathy registry experience. / Wilkinson, James D.; Westphal, Joslyn A.; Ross, Samuel W.; Dauphin, Danielle D.; Lipshultz, Steven E.

Pediatric and Congenital Cardiac Care: Volume 1: Outcomes Analysis. Springer-Verlag London Ltd, 2015. p. 445-463.

Research output: Chapter in Book/Report/Conference proceedingChapter

Wilkinson, JD, Westphal, JA, Ross, SW, Dauphin, DD & Lipshultz, SE 2015, The value of national institutes of health (NIH) registry-based research in identifying childhood cardiac disease outcomes: The pediatric cardiomyopathy registry experience. in Pediatric and Congenital Cardiac Care: Volume 1: Outcomes Analysis. Springer-Verlag London Ltd, pp. 445-463. https://doi.org/10.1007/978-1-4471-6587-3_34
Wilkinson JD, Westphal JA, Ross SW, Dauphin DD, Lipshultz SE. The value of national institutes of health (NIH) registry-based research in identifying childhood cardiac disease outcomes: The pediatric cardiomyopathy registry experience. In Pediatric and Congenital Cardiac Care: Volume 1: Outcomes Analysis. Springer-Verlag London Ltd. 2015. p. 445-463 https://doi.org/10.1007/978-1-4471-6587-3_34
Wilkinson, James D. ; Westphal, Joslyn A. ; Ross, Samuel W. ; Dauphin, Danielle D. ; Lipshultz, Steven E. / The value of national institutes of health (NIH) registry-based research in identifying childhood cardiac disease outcomes : The pediatric cardiomyopathy registry experience. Pediatric and Congenital Cardiac Care: Volume 1: Outcomes Analysis. Springer-Verlag London Ltd, 2015. pp. 445-463
@inbook{e2a32a06cc1b47319477c221f971acdb,
title = "The value of national institutes of health (NIH) registry-based research in identifying childhood cardiac disease outcomes: The pediatric cardiomyopathy registry experience",
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.",
keywords = "Cardiomyopathy, Heart failure, Patient registry, Pediatric cardiomyopathy registry, Pediatrics",
author = "Wilkinson, {James D.} and Westphal, {Joslyn A.} and Ross, {Samuel W.} and Dauphin, {Danielle D.} and Lipshultz, {Steven E}",
year = "2015",
month = "1",
day = "1",
doi = "10.1007/978-1-4471-6587-3_34",
language = "English (US)",
isbn = "9781447165873",
pages = "445--463",
booktitle = "Pediatric and Congenital Cardiac Care: Volume 1: Outcomes Analysis",
publisher = "Springer-Verlag London Ltd",

}

TY - CHAP

T1 - The value of national institutes of health (NIH) registry-based research in identifying childhood cardiac disease outcomes

T2 - The pediatric cardiomyopathy registry experience

AU - Wilkinson, James D.

AU - Westphal, Joslyn A.

AU - Ross, Samuel W.

AU - Dauphin, Danielle D.

AU - Lipshultz, Steven E

PY - 2015/1/1

Y1 - 2015/1/1

N2 - 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.

AB - 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.

KW - Cardiomyopathy

KW - Heart failure

KW - Patient registry

KW - Pediatric cardiomyopathy registry

KW - Pediatrics

UR - http://www.scopus.com/inward/record.url?scp=84947422708&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84947422708&partnerID=8YFLogxK

U2 - 10.1007/978-1-4471-6587-3_34

DO - 10.1007/978-1-4471-6587-3_34

M3 - Chapter

SN - 9781447165873

SN - 9781447165866

SP - 445

EP - 463

BT - Pediatric and Congenital Cardiac Care: Volume 1: Outcomes Analysis

PB - Springer-Verlag London Ltd

ER -