Cardiac Biomarkers in Pediatric Cardiomyopathy

描述(申请人提供)：这是一个研究项目拨款(R01)申请的心脏生物标记物在儿科心肌病。在这项拨款提案中，将有三个具体目标。第一项将尝试确定已建立的和新的临床重要性 新诊断(事件)扩张型心肌病儿童的心脏生物标志物。第二项研究旨在评估心脏胶原代谢生物标记物在确定心肌纤维化和舒张期功能障碍方面的临床应用，如心脏MRI所确定的，以及超声心动图在儿童特发性或家族性肥厚型心肌病的新诊断和现有病例中所确定的。第三个也是最终目标将集中于确定心脏生物标志物的纵向变化是否与临床稳定的扩张型或肥厚型心肌病儿童心力衰竭分级恶化有关。这些研究将显著增加我们对儿科心肌病的了解，提出新的研究领域，并改善患者管理，因为它涉及到为儿科心肌病患者确定最合适的循证临床护理。 公共卫生相关性：心肌病(心肌疾病)是导致儿童心力衰竭、死亡和心脏移植的主要原因，然而，在过去35年中，心肌病儿童的移植计划或死亡时间并没有改善，经济最发达的国家没有比发展中国家的结果更好。心肌病儿童接受心脏移植的比例在过去10年中没有下降，心肌病仍然是一岁以上儿童接受心脏移植的主要原因。儿科R01中的心脏生物标记物将致力于识别特定的心脏生物标记物和生物标记物小组，这些生物标记物将有助于确定对儿童心肌病患者最合适的循证临床护理，包括何时考虑将心脏移植作为一种治疗选择，并将提供侵入性最小(这在儿童中尤其重要)和最具成本效益的方法来早期发现心脏功能障碍。

DESCRIPTION (provided by applicant): This is a research project grant (R01) application for Cardiac Biomarkers in Pediatric Cardiomyopathy. In this grant proposal, there will be three specific aims. The first will attempt to determine the clinical importance of established and novel cardiac biomarkers in children with newly diagnosed (incident) dilated cardiomyopathy. The second will aim to assess the clinical utility of cardiac biomarkers of collagen metabolism in determining the presence of myocardial fibrosis, as established by cardiac MRI, and diastolic dysfunction, as established by echocardiography in both newly diagnosed and existing cases of idiopathic or familial hypertrophic cardiomyopathy in children. The third and final aim will focus on determining whether longitudinal changes in cardiac biomarkers are associated with worsening heart failure class in clinically stable children with dilated or hypertrophic cardiomyopathy. These studies will significantly increase our understanding of pediatric cardiomyopathy, suggest new areas of research, and improve patient management as it relates to determining the most appropriate evidence-based clinical care for pediatric cardiomyopathy patients. PUBLIC HEALTH RELEVANCE: Cardiomyopathies (diseases of the heart muscle) are a leading cause of heart failure, death, and heart transplantation in children, yet time to transplan or death for children with cardiomyopathy has not improved during the past 35 years, with the most economically advanced nations having no better outcomes than developing nations. The percentage of children with cardiomyopathy who received a heart transplant has not declined over the past 10 years and cardiomyopathy remains the leading cause of transplantation for children over one year of age. The Cardiac Biomarker in Pediatrics R01 will aim to identify specific cardiac biomarkers and panels of biomarkers that will help determine the most appropriate evidence- based clinical care for pediatric cardiomyopathy patients, including when to consider heart transplantation as a therapeutic option, and will offer the least invasive (a particularly important consideration in children) and most-cost-effective approach to the early detection of cardiac dysfunction.