Heart failure (HF) is one of the leading causes of hospitalization in the United States and is associated with substantial adverse effects on quality of life as well as a mortality rate on the order of the most lethal forms of cancer. Treatment for adults with HF varies from well- established neurohormonal inhibitors to device therapy. However, despite the demonstrated benefits of current therapy, the societal and economic burdens of HF are increasing. In pediatric HF from dilated cardiomyopathy (DCM) five-year freedom from death or transplant remains low, ranging from 54%-63%. This suggests that there are underlying myocardial cellular mechanisms uniquely regulated in children with HF. Irrespective of age or etiology of HF, new therapies for HF are necessary to improve morbidity and mortality and decrease health care costs. The proposed studies will use meticulously preserved pediatric serum along with all relevant clinical data, and are designed to integrate patient samples with an in vitro system that is a surrogate for pathological changes observed in the myocardium. In addition, this award will provide protected time for Dr. Sucharov to continue her successful mentorship of junior faculty and fellows trainees. The translational aspect of this work will be relevant to MD fellows and junior faculty, and will allow patient oriented research without risk to children with HF.
Heart failure is the most expensive medical problem in the United States, and a major cause of death and disability. While the disease is less common in children, the clinical consequences are similarly severe. Emerging experimental evidence and epidemiologic data suggest that the pediatric heart failure population is distinctly different from adult patients. However, the treatment paradigms remain similar, based almost entirely on data derived from adults. This application will address potential novel treatments for pediatric heart failure.