Core Outcome Domains for Trials in Autosomal Dominant Polycystic Kidney Disease: An International Delphi Survey

Yeoungjee Cho, Gopala Rangan, Charlotte Logeman, Hyunjin Ryu, Benedicte Sautenet, Ronald D. Perrone, Annie Claire Nadeau-Fredette, Reem A. Mustafa, Htay Htay, Michel Chonchol, Tess Harris, Talia Gutman, Jonathan C. Craig, Albert C.M. Ong, Arlene Chapman, Curie Ahn, Helen Coolican, Juliana Tze Wah Kao, Ron T. Gansevoort, Vicente TorresYork Pei, David W. Johnson, Andrea K. Viecelli, Armando Teixeira-Pinto, Martin Howell, Angela Ju, Karine E. Manera, Allison Tong

Research output: Contribution to journalArticlepeer-review

4 Scopus citations


Rationale & Objective: Outcomes reported in trials involving patients with autosomal dominant polycystic kidney disease (ADPKD) are heterogeneous and rarely include patient-reported outcomes. We aimed to identify critically important consensus-based core outcome domains to be reported in trials in ADPKD. Study Design: An international 2-round online Delphi survey was conducted in English, French, and Korean languages. Setting & Participants: Patients/caregivers and health professionals completed a 9-point Likert scale (7-9 indicating critical importance) and a Best-Worst Scale. Analytical Approach: The absolute and relative importance of outcomes were assessed. Comments were analyzed thematically. Results: 1,014 participants (603 [60%] patients/caregivers, 411 [40%] health professionals) from 56 countries completed round 1, and 713 (70%) completed round 2. The prioritized outcomes were kidney function (importance score, 8.6), end-stage kidney disease (8.6), death (7.9), blood pressure (7.9), kidney cyst size/growth (7.8), and cerebral aneurysm (7.7). Kidney cyst–related pain was the highest rated patient-reported outcome by both stakeholder groups. Seven themes explained the prioritization of outcomes: protecting life and health, directly encountering life-threatening and debilitating consequences, specificity to ADPKD, optimizing and extending quality of life, hidden suffering, destroying self-confidence, and lost opportunities. Limitations: Study design precluded involvement from those without access to internet or limited computer literacy. Conclusions: Kidney function, end-stage kidney disease, and death were the most important outcomes to patients, caregivers, and health professionals. Kidney cyst–related pain was the highest rated patient-reported outcome. Consistent reporting of these top prioritized outcomes may strengthen the value of trials in ADPKD for decision making.

Original languageEnglish (US)
Pages (from-to)361-373
Number of pages13
JournalAmerican Journal of Kidney Diseases
Issue number3
StatePublished - Sep 2020


  • Autosomal dominant polycystic kidney disease (ADPKD)
  • Delphi
  • chronic kidney disease (CKD)
  • chronic pain
  • clinical trial design
  • core outcome domains
  • patient priorities
  • patient-reported outcomes (PROs)
  • qualitative research
  • quality of life (QoL)

ASJC Scopus subject areas

  • Nephrology


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