About PCEN

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Kidney disease in children is uncommon, making it difficult to identify an adequate number of children with specific disorders and to recruit for clinical trials. Additionally, methods to clinically phenotype children with kidney disease in terms of growth, development, nutritional issues, cardiovascular disease risk factors, and bone and mineral disorders often vary from study to study, and quality control is variable. CHOP PCEN's Cores allow researchers to directly address these challenges to trial new therapies for children with kidney disease.

CHOP PCEN is funded through the National Institute of Diabetes and Digestive Kidney Diseases (NIDDK) to support basic, translational, and clinical research in pediatric diseases. There are three funded PCENs with goals to attract new scientific expertise, to encourage multidisciplinary research, and to develop the pediatric nephrology research community. In addition to CHOP, the other two Centers are at the University of Virginia and Washington University in St. Louis.

One of the critical barriers to studying childhood kidney disease is the lack of a national interconnected, multi-institutional infrastructure able to reach adequate numbers of affected children and provide the depth of information necessary to characterize their kidney disease. The Learning Health System Core will formally establish a Pediatric Nephrology Learning Network to address fundamental questions of clinical effectiveness of therapies for children and their families.

This will be accomplished in partnership with PEDSnet, a national multispecialty collaboration across eight pediatric academic health systems. PEDSnet currently includes 11 member health systems and has forged relationships with disease- and specialty-specific networks, health plans, and national data partners such as ExpressScripts and IMS Health. PEDSnet has harmonized the diverse electronic health record systems of participating institutions to assemble a longitudinal data resource that currently comprises more than five million children across 23 states.

The PCEN Learning Health System Core will extend PEDSnet resources to construct, maintain, and mediate access to structured data as part of a new Pediatric Nephrology Database Resource; develop capacity to address unstructured data elements in the Database Resource through knowledge extraction from pediatric kidney biopsy reports; and share technical and methodological resources for use of PEDSnet data across the research development continuum.

Core services:

  • Data science consultation
  • Methodological consultation on design and analysis
  • Access to the electronic health record data captured by PEDSnet to conduct descriptive/observational studies

Examples for potential pilot projects:

  • Epidemiology of renal disorders and their treatments
  • Clinical insight report for renal sub-population
  • Comparative effectiveness of interventions for well-defined cohorts
  • Case identification algorithms for enrollment into prospective clinical research

Previously funded pilot projects:

  • Learning Health Systems for Pediatric Lupus and Lupus Nephritis Research
  • Developing and Validating a Computable Phenotype for ARPKD
  • Treatment Practices and Outcomes in Children with IgA Vasculitis with Nephritis 
  • Feasibility of N-of-1 Trials in Children with Hypertension and Chronic Kidney Disease (NICHE-CKD) study
  • Comparative effectiveness of treatments for recurrent focal segmental glomerulosclerosis in pediatric kidney transplantation
  • Variation in the Care of Adolescents and Young Adults with Chronic Kidney Disease between Internal Medicine and Pediatric Nephrologists
  • Impact of Social Drivers of Health on Pediatric Kidney Transplant Outcomes

The Molecular Precision Nephrology Core (MPN Core) was established as a CHOP PCEN Core in September 2022. The objective of the MPN Core is to provide a pediatrics kidney cell reference atlas and molecular phenotyping services for PCEN investigators and for the pediatric nephrology community. Molecular phenotyping services include single cell multi-omics experiments, and data analysis.

The MPN Core aims to develop and maintain a tissue bank of human kidney samples that can be analyzed and translated into a kidney tissue reference resource atlas. Healthy kidney samples from across the childhood age spectrum will be used to generate cell level definition for healthy pediatric kidney tissues. This can be used as a reference that sampled diseased tissue can be compared against to identify key cell types and pathways for specific disease manifestations. This will improve mechanistic understanding and therapeutics development for pediatric kidney diseases as it will generate the identification of pathways that can be targeted for treatment.

The MPN Core is led by Katalin Susztak, MD, PhD. She has extensive expertise in single cell sequencing, epigenomics, proteomics, and metabolomics in the study of kidney disease and her work is focused on the molecular pathways that govern chronic kidney disease development. This core expands CHOP and Penn collaborations and the research base to promote life course research in nephrology.

Core Services:

  • Analyze, store and distribute unique data and samples
  • Provide specialized tools and technologies and access to specialized expertise
  • Develop, standardize, and share reference data for use by the research base and the broader nephrology research community