• 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, Ong Albert C M ACM Academic Nephrology Unit, Department of Infection Immunity & Cardiovascular Disease, University of Sheffield, Sheffield, United Kingdom., Arlene Chapman, Curie Ahn, Helen Coolican, Juliana Tze-Wah Kao, Ron T Gansevoort, Vicente Torres, York Pei, David W Johnson, Andrea K Viecelli, Armando Teixeira-Pinto, Martin Howell, Angela Ju, Karine E Manera, and Allison Tong.
• Department of Nephrology, Princess Alexandra Hospital, Brisbane, Australia; Australasian Kidney Trials Network, University of Queensland, Brisbane, Australia; Translational Research Institute, Brisbane, Australia. Electronic address: yeoungjee.cho@health.qld.gov.au.
• Am. J. Kidney Dis. 2020 Sep 1; 76 (3): 361-373.

Rationale & ObjectiveOutcomes 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 DesignAn international 2-round online Delphi survey was conducted in English, French, and Korean languages.Setting & ParticipantsPatients/caregivers and health professionals completed a 9-point Likert scale (7-9 indicating critical importance) and a Best-Worst Scale.Analytical ApproachThe absolute and relative importance of outcomes were assessed. Comments were analyzed thematically.Results1,014 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.LimitationsStudy design precluded involvement from those without access to internet or limited computer literacy.ConclusionsKidney 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.Copyright © 2020 National Kidney Foundation, Inc. Published by Elsevier Inc. All rights reserved.

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