Non invasive risk assessment tool for pediatric heart failure

ABSTRACT Heart Failure HF in pediatric patients is a devastating disease that is difficult to evaluate even with invasive hospital procedures Importantly and unique to the pediatric population approximately of pediatric HF patients spontaneously recover normal heart function within year of diagnosis Unfortunately there is no reliable clinical parameter or biomarker that allows pediatric cardiologists to correctly predict which of their HF patients will recover normal heart function without surgical intervention forcing clinicians to place their these children on transplant lists even though surgical intervention would be unnecessary in some cases Offering a potential solution to this problem a recent study performed at the University of Colorado Denver has identified unique expression signatures of microRNAs found in circulating blood that correlate with a subpopulation of pediatric HF patients who recover ventricular function without surgical intervention It is the objective of this Phase I project to complete initial proof of concept experiments related to the development of a commercial test based on the measurement of these circulating miRNAs The work proposed to complete this project is designed to provide the groundwork for a prospective Phase II trial By establishing and validating the test protocols with control samples this project will create the basis for a test that can then be evaluated in a subsequent prospective study designed to tests newly diagnosed pediatric HF patients and follow their treatment outcomes The successful development of a non invasive test for pediatric HF patients would have significant patient and commercial value By providing clinicians with a non invasive risk stratification tool for pediatric patients with HF a test of this kind would help clinicians patients and families understand health related prognosis for appropriate long term planning reduce the number of invasive procedures performed on these compromised patients prevent unnecessary transplantations improving survival for decades beyond the current year lifespan of a transplanted heart and decrease health care expenditures associated with cardiac transplantation NARRATIVE A recent study performed at the University of Colorado Denver has identified unique expression signatures of microRNAs found in circulating blood that correlate with a subpopulation of pediatric Heart Failure patients who recover ventricular function without a need for transplantation It is the objective of this Phase I project to complete proof of concept experiments related to the development of a biomedical test based on the measurement of these circulating miRNAs that will help clinicians risk stratify their patients