1. Core Outcome Domains for Trials in Autosomal Dominant Polycystic Kidney Disease: An International Delphi Survey.
- Author
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Cho Y, Rangan G, Logeman C, Ryu H, Sautenet B, Perrone RD, Nadeau-Fredette AC, Mustafa RA, Htay H, Chonchol M, Harris T, Gutman T, Craig JC, Ong ACM, Chapman A, Ahn C, Coolican H, Kao JT, Gansevoort RT, Torres V, Pei Y, Johnson DW, Viecelli AK, Teixeira-Pinto A, Howell M, Ju A, Manera KE, and Tong A
- Subjects
- Adolescent, Adult, Africa epidemiology, Aged, Asia epidemiology, Caregivers psychology, Child, Consensus, Delphi Technique, Female, Health Personnel psychology, Humans, Intracranial Aneurysm etiology, Kidney Failure, Chronic epidemiology, Kidney Failure, Chronic etiology, Male, Middle Aged, Pain etiology, Patient Reported Outcome Measures, Patients psychology, Polycystic Kidney, Autosomal Dominant complications, Polycystic Kidney, Autosomal Dominant epidemiology, Polycystic Kidney, Autosomal Dominant psychology, Qualitative Research, Quality of Life, Self Concept, Socioeconomic Factors, Stress, Psychological, Young Adult, Polycystic Kidney, Autosomal Dominant therapy
- Abstract
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., (Copyright © 2020 National Kidney Foundation, Inc. Published by Elsevier Inc. All rights reserved.)
- Published
- 2020
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