Cardiac Biomarkers in Pediatric Cardiomyopathy

小儿心肌病的心脏生物标志物

• 批准号: 8295233
• 负责人: STEVEN EDWARD LIPSHULTZ
• 金额: $ 143.06万
• 依托单位: UNIVERSITY OF MIAMI SCHOOL OF MEDICINE
• 依托单位国家: 美国
• 财政年份: 2012
• 资助国家: 美国
• 起止时间: 2012-08-06 至 2016-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8295233
• 关键词: Acute; Age-Years; Agreement; Applications Grants; Area; Biological Markers; Cardiac; Cardiomyopathies; Cessation of life; Child; Childhood; Clinical; Collagen; Developing Countries; Diagnosis; Dilated Cardiomyopathy; Disease; Early Diagnosis; Echocardiography; Familial Hypertrophic Cardiomyopathy; Fibrosis; Functional disorder; Heart Transplantation; Heart failure; Hypertrophic Cardiomyopathy; Hypertrophy; Left; Left Ventricular Mass; MRI Scans; Magnetic Resonance Imaging; Measurement; Measures; Metabolism; Myocardial; Myocardium; Newly Diagnosed; Outcome; Patients; Pediatrics; Research; Research Project Grants; Serum; Therapeutic; Time; Transplantation; Ventricular; clinical care; cost effective; evidence base; experience; improved; novel

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.

描述（由申请人提供）：这是一个研究项目资助（R01）申请心脏生物标志物在小儿心肌病。在这份拨款建议书中，将有三个具体目标。第一个将试图确定临床重要性的既定和新的 新诊断（偶发）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胶原代谢的心脏生物标志物在确定心肌纤维化（如心脏MRI所示）和舒张功能障碍（如超声心动图所示）中的临床效用，这些心肌纤维化和舒张功能障碍均见于新诊断和现有的特发性或家族性肥厚型心肌病儿童病例。第三个也是最后一个目标将集中在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相关。这些研究将显著增加我们对小儿心肌病的理解，提出新的研究领域，并改善患者管理，因为它涉及到确定小儿心肌病患者最合适的循证临床护理。 公共卫生关系：心肌病（心肌疾病）是儿童心力衰竭、死亡和心脏移植的主要原因，但在过去35年中，心肌病儿童的移植或死亡时间没有改善，经济最发达的国家的结果并不比发展中国家好。在过去的10年里，接受心脏移植的心肌病儿童的比例没有下降，心肌病仍然是一岁以上儿童移植的主要原因。儿科心脏生物标志物R01将旨在确定特定的心脏生物标志物和生物标志物组，这些生物标志物将有助于确定儿科心肌病患者最合适的循证临床护理，包括何时考虑心脏移植作为治疗选择，并将提供最少的侵入性（在儿童中特别重要的考虑因素）和最具成本效益的方法来早期检测心功能不全。