CHOP Pediatric Center of Excellence in Nephrology

中心概述项目总结 随着这次更新，儿童医院的儿科肾脏病卓越中心 费城(CHOP PCEN)将继续推动围绕 儿童肾脏疾病的病因、诊断和治疗。提高效率和 为了提高有效性，CHOP PCEN将继续致力于打破临床试验的障碍 在我们的病人身上实施。在最初四年的资助中，CHOP PCEN与 以PEDSnet通过学习健康系统(LHS)为核心建立全国 以儿童肾病为重点的相互关联的多机构基础设施。劳合社 CORE扩展了PEDSnet的数据科学工作，建立了嵌入式儿科 强调数据元素和数据质量优化中心的肾脏病数据资源 肾脏疾病的研究。在下一个供资周期中，LHS核心将提供扩大的 为儿科肾病的比较有效性和实用试验提供服务。通过 增加了分子精密肾病(MPN)核心，我们将有助于识别新的 目标是扩大患有肾脏疾病的儿童和年轻人的治疗选择。MPN CORE独一无二地准备解决临床和研究实施的关键障碍 儿科肾病中的分子精密仪器。行政核心促进协商 与研究设计和分析专家一起从观察中获得适当的推论 数据，并在这项建议中，设计比较有效性研究和务实的临床 审判。管理核心将支持机会池试点和可行性计划，即 丰富计划和两个将创新方法应用于数据的研究项目 LHS Core和NIDDK联盟(CKiD和CureGN)要在临床上解决的资源 重要的证据空白。其中一个项目将检验平衡液的比较效果。 与生理盐水相比，可降低脓毒症儿童患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缘结构模型 评估不同类型的复发时间-事件结局的时变治疗效果； 包括蛋白尿缓解、感染相关的急性护理和骨骼骨折。PCEN将 建立在CHOP和约翰霍普金斯大学儿科肾病学研究的坚实基础上 大学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病学同事。我们将扩大这些互动的范围 机构加速将发现转化为儿童和年轻人的治疗方法 患有肾脏疾病。

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.