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CHOP Pediatric Center of Excellence in Nephrology

CHOP 儿科肾病卓越中心

基本信息

批准号：
10705295
负责人：
Michelle Denburg
金额：
$ 90.04万
依托单位：
CHILDREN'S HOSP OF PHILADELPHIA
依托单位国家：
美国
项目类别：
财政年份：
2017
资助国家：
美国
起止时间：
2017-09-18 至 2027-08-31
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10705295
关键词：
Acceleration Acute Address Adult Atlases Bioinformatics Biometry Cells Child Childhood Chronic Chronic Kidney Failure Clinical Clinical Practice Guideline Clinical Research Clinical Trials Clinical Trials Design Clinical effectiveness Collaborations Comparative Effectiveness Research Consultations Core Facility Data Data Element Data Science Dedications Diagnosis Disease Disease Progression Disease remission Educational Status Effectiveness Evaluation Event Faculty Family Fluid Balance Focal and Segmental Glomerulosclerosis Fostering Foundations Fracture Funding Generations Health system Human Infection Infrastructure Institution Intervention Kidney Kidney Diseases Learning Maps Mentors Methodology Methods Mission Molecular Molecular Target National Institute of Diabetes and Digestive and Kidney Diseases Natural History Nephrology Normal saline Observational Study Outcome Outcomes Research Pathogenesis Patients Pediatric Hospitals Pennsylvania Philadelphia Pragmatic clinical trial Process Proteinuria Proteome Recurrence Research Research Design Research Institute Research Methodology Research Personnel Research Project Grants Resources Risk Reduction Sampling Sepsis Services Structural Models Testing Therapeutic Time Tissue Banks Tissue Sample Translations Universities Work acute care base clinical phenotype clinical trial implementation cohort comparative effectiveness comparative effectiveness study comparative effectiveness trial computable phenotypes data quality data resource design epigenome evidence base improved innovation molecular phenotype nephrogenesis new therapeutic target novel pediatric department phenotyping algorithm pragmatic trial programs skeletal therapeutic target tool treatment effect virtual young adult

项目摘要

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.

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。