CHOP Pediatric Center of Excellence in Nephrology

中心概况项目总结 随着这次更新，在儿童医院肾脏病的儿科卓越中心， 费城（CHOP PCEN）将继续促进围绕 儿童肾脏疾病的病因、诊断和治疗。提高效率和 有效性，CHOP PCEN将继续专注于打破临床试验的障碍 在我们的患者中实施。在最初的四年资助中，CHOP PCEN与 通过学习健康系统（LHS）核心与PEDSnet合作， 互联的、多机构的基础设施，重点关注儿童肾脏疾病。LHS的 核心扩展了PEDSnet的数据科学工作，以建立一个嵌入式儿科 肾脏病学数据资源，以数据元素和数据质量优化为中心 肾脏疾病的研究。在下一个融资周期，LHS核心将提供扩大的 在儿科肾病学中进行比较有效性和实用性试验的服务。通过 除了分子精密肾病（MPN）核心，我们将促进新的识别 目标是扩大儿童和成年肾病患者的治疗选择。的MPN 核心是唯一准备解决关键障碍的临床和研究实施 儿科肾脏学的分子精密工具。行政核心促进协商 与研究设计和分析方面的专家合作，从观察结果中得出适当的推论 数据，并在此建议，设计比较有效性研究和实用的临床 审判行政核心将支持机会池试点和可行性计划， 丰富计划和两个研究项目，将创新方法应用于数据 LHS核心和NIDDK联盟（CKiD和CureGN）的资源，以解决临床 重要的证据缺口。其中一个项目将检查平衡液的比较有效性 与生理盐水相比，降低脓毒症儿童急性和慢性肾脏疾病的风险。 第二个项目将开发、测试和应用一类新的边际结构模型， 估计对不同类型的复发时间至事件结局的时变治疗效应， 包括蛋白尿缓解、感染相关的急性护理和骨折。PCEN将 建立在CHOP和约翰霍普金斯儿科肾脏病学研究的坚实基础上 以及与其他儿科肾病中心和成人的成功合作 宾夕法尼亚大学的肾脏学同事。我们将扩大这些相互作用， 加速将发现转化为儿童和年轻人的治疗方法的机构 患有肾病

CENTER OVERVIEW PROJECT SUMMARY With this renewal, the Pediatric Center of Excellence in Nephrology at the Children’s Hospital of Philadelphia (CHOP PCEN) will continue to facilitate extensive collaborative research around the causes, diagnoses, and treatment of childhood kidney diseases. Increasing efficiency and effectiveness, the CHOP PCEN will continue its focus to break down barriers to clinical trials implementation in our patients. In its initial four years of funding, the CHOP PCEN has partnered with PEDSnet through the Learning Health System (LHS) Core to establish a national interconnected, multi-institutional infrastructure focused on childhood kidney disease. The LHS Core has extended the data science work of PEDSnet to establish an embedded Pediatric Nephrology Data Resource that emphasizes data elements and data quality optimization central to studies of kidney disease. In the next funding cycle, the LHS Core will provide expanded services for comparative effectiveness and pragmatic trials in pediatric nephrology. Through the addition of the Molecular Precision Nephrology (MPN) Core, we will facilitate identification of novel targets to expand therapeutic options for children and youngadults with kidney disease. The MPN Core is uniquely poised to address critical barriers to the clinical and research implementation of molecular precision tools in pediatric nephrology. The Administrative Core facilitates consultation with experts in study design and analysis to achieve appropriate inferences from observational data, and in this proposal, to design comparative effectiveness studies and pragmatic clinical trials. The Administrative Core will support the Opportunity Pool Pilot and Feasibility Program, the Enrichment Program, and two Research Projects that apply innovative approaches to the data resources of the LHS Core and NIDDK consortia (CKiD and CureGN) to address clinically important evidence gaps. One project will examine comparative effectiveness of balanced fluids versus normal saline to reduce the risk of acute and chronic kidney diseasein children with sepsis. The second project will develop, test, and apply a novel class of marginal structural models to estimate time-varying treatment effects on different types of recurrent time-to-event outcomes, including proteinuria remission, infection-related acute care, and skeletal fracture. The PCEN will build upon the strong foundation of pediatric nephrology research at CHOP and Johns Hopkins University as well as successful collaborations with other pediatric nephrology centers and adult nephrology colleagues at the University of Pennsylvania. We will amplify these interactions across institutions to accelerate translation of discoveries into therapies for children and young adults with kidney disease.