Kathleen E. Simpson, MD
Distinction of Serum Biomarkers of Heart Failure Between Children and Adults with Dilated Cardiomyopathy
Washington University in Saint Louis, Saint Louis, MO
2019 Amount Awarded – $24,400
Dilated cardiomyopathy (DCM), characterized by weak and enlarged heart muscle, is a major cause of death and heart transplantation in children. Only about 50% of children survive without a heart transplant after 5 years from diagnosis. Outside of heart transplantation, few options exist for children suffering from DCM and associated heart failure symptoms. Unfortunately, medical therapy specifically for children with DCM is lacking, and doctors typically have to use medications that have only been studied in adults. Increasingly, evidence supports that DCM in children is different from adults. Recent study results show that the amount of scar (remodeling) and signals that contribute to scarring in heart tissue varies between children and adults, suggesting that how the heart remodels in DCM is not the same across the age spectrum. These results question the effectiveness of therapies to target remodeling in children. The goal of this study is to build on this knowledge and define the different patterns of specific proteins in the blood (biomarkers) associated with children and adult DCM. The study will evaluate an extensive panel of biomarkers from blood samples from children with DCM and compare them to adults with DCM. By identifying biomarkers that can be measured in the blood and are distinct to children with DCM, it will help to identify possible targets for further heart failure therapy specific to children.