Design and Basic Characteristics of a National Patient-Powered Registry in ADPKD. Issue 8 (25th August 2022)

• Record Type: Journal Article
• Authors: Hoover, Elise; Perrone, Ronald D.; Rusconi, Chris; Benson, Beverly; Dahl, Neera K.; Gitomer, Berenice; Manelli, Amy; Mrug, Michal; Park, Meyeon; Seliger, Stephen L.; Phadnis, Milind A.; Thewarapperuma, Nadeesha; Watnick, Terry J.
• Is Part Of: Kidney360. Volume 3:Issue 8(2022)
• Page Start: 1350
• Page End: 1358
• Publication Date: 2022-08-25
• Subjects: cystic kidney disease -- ADPKD -- kidney disease -- outcomes -- polycystic kidney disease -- quality of life -- registries; 616.61
• DOI: 10.34067/KID.0002372022
• Languages: English
• ISSNs: 2641-7650

Abstract

Key Points: As of October 2021, more than 1500 autosomal dominant polycystic kidney disease (ADPKD) patients have signed up to participate in the ADPKD Registry, which collects patient-reported data. The Registry is a valuable tool for dissemination of information about studies for which individuals may qualify—seven studies so far. By incorporating two validated outcome measures, the ADPKD-IS and ADPKD-PDS, the Registry holds unique data on a national scale. Visual Abstract: Abstract : Background: Autosomal dominant polycystic kidney disease (ADPKD) is the most common form of inherited kidney disease worldwide. Over the past five years, the therapeutic pipeline for ADPKD has expanded, leading to a growing need for patient enrollment in clinical trials and improved understanding of patient-centered outcomes that can be used in trial design. To advance these goals, the Polycystic Kidney Disease Foundation (PKDF) established a national web-based ADPKD Registry. Methods: The ADPKD Registry is hosted on a secure, HIPAA-compliant, online platform (IQVIA, oc-meridian.com/pkdcure). Participants are consented through the online system and complete a series of modules. The Core Questionnaire includes patient-reported diagnosis, latest creatinine values, and comorbidities. Additional modules include surveys of family history, diet, quality of life, extrarenal manifestations, and attitudes surrounding research participation. Results: As of October 2021, 1563 ADPKD patients across the United States have registered and completed the Core Questionnaire. Participants have a median age of 44 years and are 72% women, 93% White, with 4% self-identifying as Hispanic/Latino and 2% as Black. All CKD stages are present, including post kidney transplant. To date, seven clinical studies have used the Registry as a recruitment tool. Additionally, quality-of-life burden scores revealed a correlation with disease stage as determined by kidney function. Conclusions: The Registry described here is the only one of its kind and is a valuable longitudinal research tool encompassing all stages of ADPKD. The registry will allow investigators to pursue a range of research questions related to the management of ADPKD, including definition of health-related quality of life (HRQoL) outcomes and recruitment for a variety of observational and therapeutic clinical protocols.