CHOP Pediatric Center of Excellence in Nephrology

CHOP儿科肾脏病卓越中心摘要 我们建议在以下地点设立儿科肾病卓越中心(PCEN) 费城儿童医院(CHOP)。我们的主题是解决临床试验的障碍 在肾病儿童中实施。由于儿童肾脏疾病并不常见， 确定足够数量的患有特定障碍的儿童并招募参加试验 很难。此外，在肾脏疾病的临床表型方面的方法 生长、发育、营养问题、心血管疾病危险因素、骨骼和矿物质 不同的研究往往有不同的障碍，质量控制也是不同的。学习方面的专业知识 需要设计和分析，以从观测数据中得出适当的推论，并 设计临床试验，然而，肾病儿科中心往往缺乏这方面的专业知识。我们 将通过临床和国家的区域和国家合作来应对这些挑战 CHOP、约翰·霍普金斯大学和儿童医院的翻译研究人员参与了 PEDS网。PEDSnet是由八个学术儿科健康中心组成的联盟，这些中心共同 照顾450万儿童。PEDSnet建立了一个共同的机构审查 董事会并协调了其参与中心的各种电子健康记录系统，以创建 标准化的多机构数据网络，以便利有效地开展 观察性研究、质量改进和临床试验。 CHOP-PCEN将有三个生物医学研究核心；一个设计和分析核心，一个 临床表型核心侧重于心血管疾病危险因素和营养/骨健康，一项学习 卫生系统(LHS)核心，以及一个试点和可行性计划以及一个浓缩核心。 我们包括两个利用核心的研究项目建议：“骨骼质量和血管健康 在患有尿路结石疾病的青少年中，影像生物标记物的推导和验证 CKD进展“和第三个纳入LHS核心的”儿童骨骼结局“ 和患有肾小球疾病的年轻人“。该研究基地由39个 调查员，他们是PI的、合作调查员或150多个资助项目的合作者 每年的直接成本总计超过3500万美元，其中超过1200万美元用于研究 由核心服务的潜在用户领导的与儿科肾病相关的项目。PCEN将 建立在CHOP和霍普金斯大学坚实的临床研究基础上，以及 宾夕法尼亚大学成人肾脏病学部和临床流行病学中心 生物统计学促进儿童肾脏病临床试验的设计和实施 疾病。

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.