Cardiac biomarkers in pediatric cardiomyopathy

Study design and recruitment results from the Pediatric Cardiomyopathy Registry

Pediatric Cardiomyopathy Registry Investigators

Research output: Contribution to journalArticle

Abstract

Background: Cardiomyopathies are a rare cause of pediatric heart disease, but they are one of the leading causes of heart failure admissions, sudden death, and need for heart transplant in childhood. Reports from the Pediatric Cardiomyopathy Registry (PCMR) have shown that almost 40% of children presenting with symptomatic cardiomyopathy either die or undergo heart transplant within 2 years of presentation. Little is known regarding circulating biomarkers as predictors of outcome in pediatric cardiomyopathy. Study design: The Cardiac Biomarkers in Pediatric Cardiomyopathy (PCM Biomarkers) study is a multi-center prospective study conducted by the PCMR investigators to identify serum biomarkers for predicting outcome in children with dilated cardiomyopathy (DCM) and hypertrophic cardiomyopathy (HCM). Patients < 21 years of age with either DCM or HCM were eligible. Those with DCM were enrolled into cohorts based on time from cardiomyopathy diagnosis: categorized as new onset or chronic. Clinical endpoints included sudden death and progressive heart failure. Results: There were 288 children diagnosed at a mean age of 7.2 ± 6.3 years who enrolled in the PCM Biomarkers Study at a median time from diagnosis to enrollment of 1.9 years. There were 80 children enrolled in the new onset DCM cohort, defined as diagnosis at or 12 months prior to enrollment. The median age at diagnosis for the new onset DCM was 1.7 years and median time from diagnosis to enrollment was 0.1 years. There were 141 children enrolled with either chronic DCM or chronic HCM, defined as children ≥2 years from diagnosis to enrollment. Among children with chronic cardiomyopathy, median age at diagnosis was 3.4 years and median time from diagnosis to enrollment was 4.8 years. Conclusion: The PCM Biomarkers study is evaluating the predictive value of serum biomarkers to aid in the prognosis and management of children with DCM and HCM. The results will provide valuable information where data are lacking in children.

Original languageEnglish (US)
JournalProgress in Pediatric Cardiology
DOIs
StatePublished - Jan 1 2019

Fingerprint

Cardiomyopathies
Registries
Dilated Cardiomyopathy
Biomarkers
Pediatrics
Hypertrophic Cardiomyopathy
Sudden Death
Heart Failure
Transplants
Serum
Heart Diseases
Research Personnel
Prospective Studies

Keywords

  • Biomarkers
  • Cardiomyopathy
  • Heart failure
  • Pediatrics

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Cardiology and Cardiovascular Medicine

Cite this

Cardiac biomarkers in pediatric cardiomyopathy : Study design and recruitment results from the Pediatric Cardiomyopathy Registry. / Pediatric Cardiomyopathy Registry Investigators.

In: Progress in Pediatric Cardiology, 01.01.2019.

Research output: Contribution to journalArticle

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title = "Cardiac biomarkers in pediatric cardiomyopathy: Study design and recruitment results from the Pediatric Cardiomyopathy Registry",
abstract = "Background: Cardiomyopathies are a rare cause of pediatric heart disease, but they are one of the leading causes of heart failure admissions, sudden death, and need for heart transplant in childhood. Reports from the Pediatric Cardiomyopathy Registry (PCMR) have shown that almost 40{\%} of children presenting with symptomatic cardiomyopathy either die or undergo heart transplant within 2 years of presentation. Little is known regarding circulating biomarkers as predictors of outcome in pediatric cardiomyopathy. Study design: The Cardiac Biomarkers in Pediatric Cardiomyopathy (PCM Biomarkers) study is a multi-center prospective study conducted by the PCMR investigators to identify serum biomarkers for predicting outcome in children with dilated cardiomyopathy (DCM) and hypertrophic cardiomyopathy (HCM). Patients < 21 years of age with either DCM or HCM were eligible. Those with DCM were enrolled into cohorts based on time from cardiomyopathy diagnosis: categorized as new onset or chronic. Clinical endpoints included sudden death and progressive heart failure. Results: There were 288 children diagnosed at a mean age of 7.2 ± 6.3 years who enrolled in the PCM Biomarkers Study at a median time from diagnosis to enrollment of 1.9 years. There were 80 children enrolled in the new onset DCM cohort, defined as diagnosis at or 12 months prior to enrollment. The median age at diagnosis for the new onset DCM was 1.7 years and median time from diagnosis to enrollment was 0.1 years. There were 141 children enrolled with either chronic DCM or chronic HCM, defined as children ≥2 years from diagnosis to enrollment. Among children with chronic cardiomyopathy, median age at diagnosis was 3.4 years and median time from diagnosis to enrollment was 4.8 years. Conclusion: The PCM Biomarkers study is evaluating the predictive value of serum biomarkers to aid in the prognosis and management of children with DCM and HCM. The results will provide valuable information where data are lacking in children.",
keywords = "Biomarkers, Cardiomyopathy, Heart failure, Pediatrics",
author = "{Pediatric Cardiomyopathy Registry Investigators} and Everitt, {Melanie D.} and Wilkinson, {James D.} and Ling Shi and Towbin, {Jeffrey A.} and Colan, {Steven D.} and Kantor, {Paul F.} and Canter, {Charles E.} and Webber, {Steven A.} and Hsu, {Daphne T.} and Elfriede Pahl and Addonizio, {Linda J.} and Dodd, {Debra A.} and Jefferies, {John L.} and Rossano, {Joseph W.} and Brian Feingold and Ware, {Stephanie M.} and Lee, {Teresa M.} and Justin Godown and Simpson, {Kathleen E.} and Sleeper, {Lynn A.} and Czachor, {Jason D.} and Hiedy Razoky and Ashley Hill and Joslyn Westphal and Molina, {Kimberly M.} and Lipshultz, {Steven E.}",
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T2 - Study design and recruitment results from the Pediatric Cardiomyopathy Registry

AU - Pediatric Cardiomyopathy Registry Investigators

AU - Everitt, Melanie D.

AU - Wilkinson, James D.

AU - Shi, Ling

AU - Towbin, Jeffrey A.

AU - Colan, Steven D.

AU - Kantor, Paul F.

AU - Canter, Charles E.

AU - Webber, Steven A.

AU - Hsu, Daphne T.

AU - Pahl, Elfriede

AU - Addonizio, Linda J.

AU - Dodd, Debra A.

AU - Jefferies, John L.

AU - Rossano, Joseph W.

AU - Feingold, Brian

AU - Ware, Stephanie M.

AU - Lee, Teresa M.

AU - Godown, Justin

AU - Simpson, Kathleen E.

AU - Sleeper, Lynn A.

AU - Czachor, Jason D.

AU - Razoky, Hiedy

AU - Hill, Ashley

AU - Westphal, Joslyn

AU - Molina, Kimberly M.

AU - Lipshultz, Steven E.

PY - 2019/1/1

Y1 - 2019/1/1

N2 - Background: Cardiomyopathies are a rare cause of pediatric heart disease, but they are one of the leading causes of heart failure admissions, sudden death, and need for heart transplant in childhood. Reports from the Pediatric Cardiomyopathy Registry (PCMR) have shown that almost 40% of children presenting with symptomatic cardiomyopathy either die or undergo heart transplant within 2 years of presentation. Little is known regarding circulating biomarkers as predictors of outcome in pediatric cardiomyopathy. Study design: The Cardiac Biomarkers in Pediatric Cardiomyopathy (PCM Biomarkers) study is a multi-center prospective study conducted by the PCMR investigators to identify serum biomarkers for predicting outcome in children with dilated cardiomyopathy (DCM) and hypertrophic cardiomyopathy (HCM). Patients < 21 years of age with either DCM or HCM were eligible. Those with DCM were enrolled into cohorts based on time from cardiomyopathy diagnosis: categorized as new onset or chronic. Clinical endpoints included sudden death and progressive heart failure. Results: There were 288 children diagnosed at a mean age of 7.2 ± 6.3 years who enrolled in the PCM Biomarkers Study at a median time from diagnosis to enrollment of 1.9 years. There were 80 children enrolled in the new onset DCM cohort, defined as diagnosis at or 12 months prior to enrollment. The median age at diagnosis for the new onset DCM was 1.7 years and median time from diagnosis to enrollment was 0.1 years. There were 141 children enrolled with either chronic DCM or chronic HCM, defined as children ≥2 years from diagnosis to enrollment. Among children with chronic cardiomyopathy, median age at diagnosis was 3.4 years and median time from diagnosis to enrollment was 4.8 years. Conclusion: The PCM Biomarkers study is evaluating the predictive value of serum biomarkers to aid in the prognosis and management of children with DCM and HCM. The results will provide valuable information where data are lacking in children.

AB - Background: Cardiomyopathies are a rare cause of pediatric heart disease, but they are one of the leading causes of heart failure admissions, sudden death, and need for heart transplant in childhood. Reports from the Pediatric Cardiomyopathy Registry (PCMR) have shown that almost 40% of children presenting with symptomatic cardiomyopathy either die or undergo heart transplant within 2 years of presentation. Little is known regarding circulating biomarkers as predictors of outcome in pediatric cardiomyopathy. Study design: The Cardiac Biomarkers in Pediatric Cardiomyopathy (PCM Biomarkers) study is a multi-center prospective study conducted by the PCMR investigators to identify serum biomarkers for predicting outcome in children with dilated cardiomyopathy (DCM) and hypertrophic cardiomyopathy (HCM). Patients < 21 years of age with either DCM or HCM were eligible. Those with DCM were enrolled into cohorts based on time from cardiomyopathy diagnosis: categorized as new onset or chronic. Clinical endpoints included sudden death and progressive heart failure. Results: There were 288 children diagnosed at a mean age of 7.2 ± 6.3 years who enrolled in the PCM Biomarkers Study at a median time from diagnosis to enrollment of 1.9 years. There were 80 children enrolled in the new onset DCM cohort, defined as diagnosis at or 12 months prior to enrollment. The median age at diagnosis for the new onset DCM was 1.7 years and median time from diagnosis to enrollment was 0.1 years. There were 141 children enrolled with either chronic DCM or chronic HCM, defined as children ≥2 years from diagnosis to enrollment. Among children with chronic cardiomyopathy, median age at diagnosis was 3.4 years and median time from diagnosis to enrollment was 4.8 years. Conclusion: The PCM Biomarkers study is evaluating the predictive value of serum biomarkers to aid in the prognosis and management of children with DCM and HCM. The results will provide valuable information where data are lacking in children.

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KW - Cardiomyopathy

KW - Heart failure

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