Personalizing Outcomes of Nephrolithiasis in Youth (PONY)

PROJECT SUMMARY - OVERALL Kidney stone disease (nephrolithiasis) is characterized by painful, recurrent symptomatic events and is rising disproportionately in youth. Structural and scientific barriers have impeded strengthening the evidence base for management of pediatric kidney stone disease. The overarching objective of the Personalizing Outcomes of Nephrolithiasis in Youth (PONY) P20 Center is to generate knowledge that improves outcomes for youth with nephrolithiasis through an intentional and focused initiative to accelerate the research careers of early-stage investigators and thereby enhance the benign urology research community. To achieve this mission, this proposal strengthens partnerships among Dr. Gregory Tasian (PI at Children’s Hospital of Philadelphia, Dr Jonathan Ellison (early-stage investigator at Children’s Wisconsin), Dr. David Chu (early-stage investigator at Lurie Children’s Hospital of Chicago), and Dr. Jing Huang (biostatistician at Children’s Hospital of Philadelphia). In the proposed PONY P20 Center, we address critical structural and scientific barriers that have impeded strengthening the evidence base for management of pediatric nephrolithiasis. We will accomplish our objectives by leveraging the resources generated by Dr. Tasian’s research program, in particular the robust infrastructure of the Pediatric KIDney Stone (PKIDS) Care Improvement Network and data generated by the NIDDK-supported Urinary Stone Disease Research Network. Central to the PONY P20 Center is the PKIDS Care Improvement Network, which will support the Research Project and provide the means to disseminate the knowledge generated by the research directly into real-world care. In Aim 1, we will generate new knowledge responsive to patient-identified gaps in kidney stone research by conducting two distinct but complementary research studies that use data from PKIDS trial, the Prevention of Urinary Stones with Hydration (PUSH) trial, and the STudy to Enhance uNderstanding of sTent-associated Symptoms (STENTS). In Aim 2, we will accelerate the research careers of early-stage investigators in benign urology. The Administrative Core will create opportunities for Drs. Ellison and Chu to lead the execution of the Research Project through operational and logistical support as well as data access. PKIDS will also provide the scientific environment to support the R01 applications led by Drs. Ellison and Chu that will arise from the Research Project. In Aim 3, the PONY P20 Center will enhance the benign urology research community. We will share our expertise in stakeholder (particularly patient and caregiver) engagement with members of CAIRIBU other benign urologic researchers. We will participate in existing CAIRIBU forums by contributing our lessons learned on stakeholder engagement in clinical investigation, patient-centered trial design, and conceptual innovations in learning health systems. Overall, the PONY P20 Center will generate knowledge that addresses critical priorities identified by youth with nephrolithiasis and accelerates the career of early-stage investigators who will be leaders in benign urological research.