MP58-17 LEVERAGING CLINICAL RESEARCH NETWORKS TO IDENTIFY PATIENTS WITH PRIMARY HYPEROXALURIA FOR CLINICAL TRIALS

Abstract

You have accessJournal of UrologyHealth Services Research: Quality Improvement & Patient Safety IV (MP58)1 Sep 2021MP58-17 LEVERAGING CLINICAL RESEARCH NETWORKS TO IDENTIFY PATIENTS WITH PRIMARY HYPEROXALURIA FOR CLINICAL TRIALS Christina Ching, Kim Dickinson, Hanieh Razzaghi, Matthew Lorenzo, Levon Utidjian, John Karafilidis, Lisa Mucha, Christopher Forrest, and Gregory Tasian Christina ChingChristina Ching More articles by this author , Kim DickinsonKim Dickinson More articles by this author , Hanieh RazzaghiHanieh Razzaghi More articles by this author , Matthew LorenzoMatthew Lorenzo More articles by this author , Levon UtidjianLevon Utidjian More articles by this author , John KarafilidisJohn Karafilidis More articles by this author , Lisa MuchaLisa Mucha More articles by this author , Christopher ForrestChristopher Forrest More articles by this author , and Gregory TasianGregory Tasian More articles by this author View All Author Informationhttps://doi.org/10.1097/JU.0000000000002088.17AboutPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareFacebookLinked InTwitterEmail Abstract INTRODUCTION AND OBJECTIVE: Challenges to performing clinical trials include identifying and recruiting eligible participants across institutions. These challenges increase for rare diseases such as primary hyperoxaluria (PH), a genetic disorder of liver oxalate overproduction that affects up to 1 in 40,000 persons. Here, we leverage data from a clinical research network (CRN) to identify one of the largest cohorts of children with PH for future clinical trials. METHODS: PEDSnet is a pediatric CRN comprised of 8 US health systems, engaging clinicians, researchers, and other stakeholders to conduct research to improve health outcomes in children. PEDSnet has harmonized the diverse EHR systems of participating institutions to assemble a longitudinal database for >6 million children across 23 states by transforming source institutional EHR data into the OMOP and PCORnet common data model (CDM). The CDM organizes data into a standard structure, which facilitates centralized queries. PEDSnet’s data network also provides a path back to the full patient record, enabling contact for prospective studies. We developed and executed an algorithm to identify children <18 years with PH seen at 6 PEDSnet institutions from January 1, 2009 - December 15, 2020 and characterized the cohort to determine feasibility of clinical trial recruitment. RESULTS: We identified 286 patients with likely PH (median age 9.4 years; IQR 5.1,12.9) of whom 263 had at least one diagnostic code for PH 1, 2, or 3, hyperoxaluria, oxalate nephropathy, or oxalosis, and an additional 23 had ≥2 diagnosis codes for disorder of carbohydrate metabolism and ≥1 code for kidney stones. 70% had kidney stones and 24% had ≥1 stone surgery. The median follow-up time was 2.8 years (IQR 1.1, 6.2). The median number of days since the most recent encounter was 190 days (IQR 36, 988) with 160 (56%) having been seen in the last year (Figure 1). CONCLUSIONS: We demonstrate the utility of PEDSnet to establish a large PH cohort for meaningful research and discovery. Specifically, 160 children with PH had recent encounters in 6 health systems which could indicate their accessibility for HIPAA compliant recruitment in clinical trials. This model could improve the efficiency, effectiveness, and cost of conducting clinical trials for rare urologic diseases. Source of Funding: Dicerna Pharmaceuticals © 2021 by American Urological Association Education and Research, Inc.FiguresReferencesRelatedDetails Volume 206Issue Supplement 3September 2021Page: e994-e995 Advertisement Copyright & Permissions© 2021 by American Urological Association Education and Research, Inc.MetricsAuthor Information Christina Ching More articles by this author Kim Dickinson More articles by this author Hanieh Razzaghi More articles by this author Matthew Lorenzo More articles by this author Levon Utidjian More articles by this author John Karafilidis More articles by this author Lisa Mucha More articles by this author Christopher Forrest More articles by this author Gregory Tasian More articles by this author Expand All Advertisement Loading ...