# Prognostic Significance of microRNA Expression in Children with Cardiomyopathy

> **NIH NIH R01** · STATE UNIVERSITY OF NEW YORK AT BUFFALO · 2021 · $799,064

## Abstract

Project Summary
Pediatric cardiomyopathies encompass a heterogeneous group of disorders including dilated, hypertrophic,
and less commonly, restrictive cardiomyopathies. Emerging experimental evidence and epidemiologic data
suggest that the pediatric heart failure population is distinctly different from adult patients The most common
cause of heart failure and reason for cardiac transplantation in children older than 1 year is dilated
cardiomyopathy (DCAmong these etiologies, pediatric dilated cardiomyopathy (DCM) has an estimated 40%
five year transplant-free survival and remains the most common diagnosis leading to heart transplant in
children greater than 1 year of age. The central hypothesis is that circulating microRNAs (miRs) will be useful
biomarkers for risk stratification, will correlate with outcomes and may represent novel therapeutic targets in
children with DCM. The aims of this study are: 1) Determine the profile of circulating miRs in a cohort of
children with DCM and acute systolic heart failure. The circulating miR expression patterns will then be
stratified based on reaching a primary end-point by 1 year defined as: [1] death or transplantation, [2] recovery
(normalization of ventricular size and function) or [3] stable DCM (persistent ventricular dilation or dysfunction);
2) Define the miR profile in a separate cohort of children with chronic, stable DCM to determine if those
patients with stable DCM who progress to death/transplant within 1 year will be similar to the profile of the Aim
1 cohort of children with acute heart failure who also progress to death/transplant; and 3) Analyze the
expression of circulating and heart tissue miRs in pediatric controls (non-failing donors whose heart could not
be placed [NF]) and pediatric DCM patients. The results of this study could improve clinicians prognostic’
assessment at diagnosis of DCM in children, improve decision-making regarding listing children for heart
transplant as opposed to identifying those who are expected to recover ventricular function, and could provide
insight into cellular mechanisms of disease and define novel targets for future treatment of DCM in children.

## Key facts

- **NIH application ID:** 10126049
- **Project number:** 5R01HL139968-04
- **Recipient organization:** STATE UNIVERSITY OF NEW YORK AT BUFFALO
- **Principal Investigator:** STEVEN EDWARD LIPSHULTZ
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $799,064
- **Award type:** 5
- **Project period:** 2018-02-17 → 2024-01-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10126049

## Citation

> US National Institutes of Health, RePORTER application 10126049, Prognostic Significance of microRNA Expression in Children with Cardiomyopathy (5R01HL139968-04). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10126049. Licensed CC0.

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