CHOP Pediatric Center of Excellence in Nephrology

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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.