CHOP Pediatric Center of Excellence in Nephrology

CHOP 儿科肾病卓越中心

• 批准号: 10241465
• 负责人: SUSAN L. FURTH
• 金额: $ 83.51万
• 依托单位: CHILDREN'S HOSP OF PHILADELPHIA
• 依托单位国家: 美国
• 财政年份: 2017
• 资助国家: 美国
• 起止时间: 2017-09-18 至 2022-09-14
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10241465
• 关键词: Address; Adolescent; Adult; Agreement; Biomedical Research; Biometry; Blood Vessels; Caring; Cessation of life; Child; Childhood; Clinical; Clinical Data; Clinical Practice Guideline; Clinical Research; Clinical Trials; Clinical Trials Design; Collaborations; Core Facility; Data; Derivation procedure; Diagnosis; Direct Costs; Disease; Educational Status; Electronic Health Record; Enrollment; Evaluation; Family; Fostering; Foundations; Funding; Goals; Growth and Development function; Health; Health system; Infrastructure; Institution; Institutional Review Boards; Kidney Diseases; Learning; Logistics; Mentors; Methods; Minerals; Multi-Institutional Clinical Trial; Nephrology; Nutritional; Observational Study; Outcome; Outcome Assessment; Patients; Pediatric Hospitals; Peer Review; Pennsylvania; Philadelphia; Pilot Projects; Population; Quality Control; Renal glomerular disease; Research; Research Design; Research Methodology; Research Personnel; Research Project Grants; Resources; Risk Factors; Services; Standardization; System; Translations; Universities; Urinary Calculi; Validation; base; bone; bone health; bone quality; cardiovascular disorder risk; clinical center; clinical epidemiology; clinical phenotype; clinical trial enrollment; clinical trial implementation; design; disease diagnosis; evidence base; imaging biomarker; implementation framework; improved; multiple data sources; nutrition; programs; recruit; skeletal; translational scientist; trial design; virtual; young adult

CHOP PEDIATRIC CENTER OF EXCELLENCE IN NEPHROLOGY ABSTRACT We propose to establish a Pediatric Center of Excellence in Nephrology (PCEN) at the Children’s Hospital of Philadelphia (CHOP). Our theme is to address barriers to clinical trials implementation in children with kidney disease. As kidney disease in children is uncommon, identification of adequate numbers of children with specific disorders and recruitment for trials is difficult. Additionally, methods to clinically phenotype children with kidney disease in terms of growth, development, nutritional issues, cardiovascular disease risk factors, bone and mineral disorders often vary from study to study, and quality control is variable. Expertise in study design and analysis is needed to achieve appropriate inferences from observational data, and to design clinical trials, however, frequently pediatric centers in nephrology lack this expertise. We will address these challenges through a regional and national collaboration of clinical and translational researchers at CHOP, Johns Hopkins, and children’s hospitals participating in PEDSnet. PEDSnet is a consortium of eight academic pediatric health centers that collectively provide care for >4.5 million children. PEDSnet has established a common institutional review board and has harmonized the diverse EHR systems of its participating centers to create a standardized multi-institutional data network in order to facilitate the efficient conduct of observational research, quality improvement and clinical trials. The CHOP-PCEN will have three biomedical research cores; a Design and Analysis core, a Clinical Phenotyping Core focused on CVD risk factors and Nutrition/Bone health, a Learning Health System (LHS) Core, as well as a pilot and feasibility program and an enrichment core. We include 2 research project proposals utilizing the cores: “Bone Quality and Vascular Health in Adolescents with Urinary Stone Disease”, “Derivation and Validation of Imaging Biomarkers for CKD Progression” and a third, integrated into the LHS core, “Skeletal Outcomes in Children and Young Adults with Glomerular Disease”. The research base is comprised of 39 investigators, who are PI’s, co-investigators or collaborators on over 150 funded projects totaling over $35 million in annual direct costs, over $12 million of this funding is in research projects relevant to pediatric nephrology led by likely users of the core services. The PCEN will build upon the strong foundation of clinical research at CHOP and Hopkins, as well as the University of Penn Adult Nephrology Division and Center for Clinical Epidemiology and Biostatistics to catalyze the design and implementation of clinical trials in children with kidney disease.

CHOP 儿科肾病卓越中心 摘要 我们建议在儿科肾病学卓越中心 (PCEN) 费城儿童医院 (CHOP) 我们的主题是解决临床试验的障碍。 儿童肾病的实施 由于儿童肾病并不常见， 确定足够数量的患有特定疾病的儿童并招募试验对象 此外，对患有肾脏疾病的儿童进行临床表型的方法也很困难。 生长、发育、营养问题、心血管疾病危险因素、骨骼和矿物质 疾病通常因研究而异，并且研究的质量控制也各不相同。 需要设计和分析才能从观测数据中得出适当的推论，并 然而，设计临床试验时，儿科肾脏病中心常常缺乏这方面的专业知识。 将通过区域和国家临床和技术合作来应对这些挑战 CHOP、约翰霍普金斯大学和儿童医院的转化研究人员参与 PEDSnet 是一个由八个学术儿科健康中心组成的联盟。 PEDSnet 已建立了共同的机构审查，为超过 450 万儿童提供护理。 董事会并协调了其参与中心的不同 EHR 系统，以创建一个 标准化的多机构数据网络，以促进高效开展 观察性研究、质量改进和临床试验。 CHOP-PCEN 将拥有三个生物医学研究核心；一个设计和分析核心，一个 临床表型核心专注于 CVD 危险因素和营养/骨骼健康，一门学习 卫生系统 (LHS) 核心，以及试点和可行性计划以及丰富核心。 我们包括 2 个利用核心的研究项目提案：“骨质量和血管健康 患有尿路结石病的青少年”，“成像生物标志物的推导和验证 CKD 进展”，第三个纳入 LHS 核心，“儿童骨骼结果” 和患有肾小球疾病的年轻人”该研究基地由 39 名成员组成。 研究人员，他们是 150 多个资助项目的 PI、共同研究人员或合作者 年度直接成本总计超过 3500 万美元，其中超过 1200 万美元用于研究 PCEN 将由核心服务的可能用户领导与儿科肾病学相关的项目。 建立在 CHOP 和霍普金斯大学强大的临床研究基础以及 宾夕法尼亚大学成人肾脏科和临床流行病学中心 生物统计学促进肾病儿童临床试验的设计和实施 疾病。