Design and Basic Characteristics of a National Patient-Powered Registry in ADPKD

Kidney360. 2022 May 20;3(8):1350-1358. doi: 10.34067/KID.0002372022. eCollection 2022 Aug 25.

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.

Keywords: ADPKD; cystic kidney disease; kidney disease; outcomes; polycystic kidney disease; quality of life; registries.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Creatinine / therapeutic use
  • Female
  • Humans
  • Male
  • Polycystic Kidney, Autosomal Dominant* / diagnosis
  • Quality of Life
  • Registries
  • Surveys and Questionnaires
  • United States / epidemiology

Substances

  • Creatinine