TY - JOUR
T1 - Core Outcome Domains for Trials in Autosomal Dominant Polycystic Kidney Disease
T2 - An International Delphi Survey
AU - Cho, Yeoungjee
AU - Rangan, Gopala
AU - Logeman, Charlotte
AU - Ryu, Hyunjin
AU - Sautenet, Benedicte
AU - Perrone, Ronald D
AU - Nadeau-Fredette, Annie-Claire
AU - Mustafa, Reem A
AU - Htay, Htay
AU - Chonchol, Michel
AU - Harris, Tess
AU - Gutman, Talia
AU - Craig, Jonathan C
AU - Ong, Albert C M
AU - Chapman, Arlene
AU - Ahn, Curie
AU - Coolican, Helen
AU - Kao, Juliana Tze-Wah
AU - Gansevoort, Ron T
AU - Torres, Vicente
AU - Pei, York
AU - Johnson, David W
AU - Viecelli, Andrea K
AU - Teixeira-Pinto, Armando
AU - Howell, Martin
AU - Ju, Angela
AU - Manera, Karine E
AU - Tong, Allison
N1 - Copyright © 2020 National Kidney Foundation, Inc. Published by Elsevier Inc. All rights reserved.
PY - 2020/9
Y1 - 2020/9
N2 - 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.
AB - 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.
U2 - 10.1053/j.ajkd.2020.01.005
DO - 10.1053/j.ajkd.2020.01.005
M3 - Article
C2 - 32359822
SN - 0272-6386
VL - 76
SP - 361
EP - 373
JO - American Journal of Kidney Diseases
JF - American Journal of Kidney Diseases
IS - 3
ER -