Abstract 4346346: Biomarkers in Patients with Newly Diagnosed Dilated Cardiomyopathy: A Study from the Pediatric Cardiomyopathy Registry

Background: Children with dilated cardiomyopathy (DCM) are at high risk for morbidity and death. There are few data on the utility of biomarkers in this population. Research Question/Hypothesis: We aimed to evaluate a panel of cardiac biomarkers as predictors of death or heart transplant within 2 years of diagnosis. Methods: Patients <21 years of age at DCM diagnosis were enrolled at 15 North American pediatric cardiomyopathy centers within one year of diagnosis with collection of serum, echocardiographic and clinical data at enrollment. Results: Data from 74 subjects were analyzed, median age 2.1 years at enrollment [interquartile range (IQR 0.7-12.8)], 40 (54%) female, and 56 (76%) enrolled within 90 days of diagnosis. The primary endpoint of death or heart transplant was met in 13 subjects (18%). Several cardiac biomarkers of heart failure (HF), N-terminal pro B-type natriuretic peptide, growth differentiation factor-15, interleukin-6, and C-reactive protein were associated with the primary endpoint (p <0.05 for all) in the univariate analysis. In multivariable analysis, growth differentiation factor-15 was independently associated with the primary endpoint (hazard ratio 5.23, 95% confidence interval 2.20, 12.44; p <0.001). Conclusion: Growth differentiation factor-15, an emerging circulating biomarker for HF, was independently associated with death or heart transplantation within 2 years in children with a recent diagnosis of DCM. Traditional biomarkers such as NT pro-BNP were found to be less predictive on single point assessment. If validated in further studies, this biomarker could serve as an endpoint for prospective clinical trials.

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Journal
Circulation
Published
2025-11-03
DOI
https://doi.org/10.1161/circ.152.suppl_3.4346346
Primary Topic
GDF15 and Related Biomarkers
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article
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article

Abstract 4346346: Biomarkers in Patients with Newly Diagnosed Dilated Cardiomyopathy: A Study from the Pediatric Cardiomyopathy Registry

Steven D. Colan, Charles E. Canter, Stephanie M. Ware, Brian Feingold et al.
Circulation
GDF15 and Related Biomarkers
article

Abstract 4346346: Biomarkers in Patients with Newly Diagnosed Dilated Cardiomyopathy: A Study from the Pediatric Cardiomyopathy Registry

Steven D. Colan, Charles E. Canter, Stephanie M. Ware, Brian Feingold, Jean A. Ballweg, Joseph William Rossano, Taye H. Hamza, Paul F. Kantor, Irene D. Lytrivi, Melanie D. Everitt, Elfriede Pahl, Sonya Kirmani, Neha M. Bansal, Steven E. Lipshultz, Jeffrey Allen Towbin, Teresa Lee, James L. Wilkinson, Justin Godown, Steven Webber
article en

Abstract

Background: Children with dilated cardiomyopathy (DCM) are at high risk for morbidity and death. There are few data on the utility of biomarkers in this population. Research Question/Hypothesis: We aimed to evaluate a panel of cardiac biomarkers as predictors of death or heart transplant within 2 years of diagnosis. Methods: Patients <21 years of age at DCM diagnosis were enrolled at 15 North American pediatric cardiomyopathy centers within one year of diagnosis with collection of serum, echocardiographic and clinical data at enrollment. Results: Data from 74 subjects were analyzed, median age 2.1 years at enrollment [interquartile range (IQR 0.7-12.8)], 40 (54%) female, and 56 (76%) enrolled within 90 days of diagnosis. The primary endpoint of death or heart transplant was met in 13 subjects (18%). Several cardiac biomarkers of heart failure (HF), N-terminal pro B-type natriuretic peptide, growth differentiation factor-15, interleukin-6, and C-reactive protein were associated with the primary endpoint (p <0.05 for all) in the univariate analysis. In multivariable analysis, growth differentiation factor-15 was independently associated with the primary endpoint (hazard ratio 5.23, 95% confidence interval 2.20, 12.44; p <0.001). Conclusion: Growth differentiation factor-15, an emerging circulating biomarker for HF, was independently associated with death or heart transplantation within 2 years in children with a recent diagnosis of DCM. Traditional biomarkers such as NT pro-BNP were found to be less predictive on single point assessment. If validated in further studies, this biomarker could serve as an endpoint for prospective clinical trials.

CirculationVol. 152(Suppl_3)
Northwestern University (US), Boston Children's Hospital (US), Children's Hospital of Philadelphia (US), Children's Hospital of Los Angeles (US), University of Wisconsin–Madison (US), Vanderbilt University (US), Washington University in St. Louis (US), Columbia University Irving Medical Center (US), Children's Hospital Colorado (US), Mount Sinai Hospital (US), Michigan United (US), Alexion Pharmaceuticals (United States) (US), Helen DeVos Children's Hospital (US), Children's Hospital of Pittsburgh (US), Cytokinetics (United States) (US), Jacobs Institute (US), Boston Children's Museum (US), Le Bonheur Children's Hospital (US), Indiana University (US), University of Arkansas for Medical Sciences (US), University of Colorado Denver (US)
Openalex Percentile: Top 54%
GDF15 and Related Biomarkers
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