Investigations of Pathologic Remodeling Using Pediatric Heart Failure Serum

摘要 心力衰竭（HF）是美国住院的主要原因之一， 与对生活质量的实质性不利影响以及 最致命的癌症类型成人HF的治疗从良好的- 已建立的神经激素抑制剂用于器械治疗。然而，尽管显示 随着当前治疗的益处，HF的社会和经济负担正在增加。儿科 扩张型心肌病（DCM）导致的HF五年内免于死亡或移植的比率仍然很低， 范围为54%-63%。这表明心肌细胞 在HF儿童中独特调节的机制。不考虑年龄或HF病因，新发 有必要对HF进行治疗，以改善发病率和死亡率， 成本 拟议的研究将使用精心保存的儿科血清沿着与所有相关的 临床数据，并设计用于将患者样本与体外系统整合， 心肌中观察到的病理变化的替代物。此外，该奖项将 为Sucharov博士提供受保护的时间，以继续她对初级教师的成功指导 和学员。这项工作的翻译方面将与MD研究员有关， 初级教师，并将允许病人为导向的研究没有风险的儿童与HF。

Abstract 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.