Cho, Y., Rangan, G., Logeman, C. et al. (25 more authors) (2020) Core outcome domains for trials in autosomal dominant polycystic kidney disease: An international Delphi survey. American Journal of Kidney Diseases, 76 (3). pp. 361-373. ISSN 0272-6386
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.
Metadata
Item Type: | Article |
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Authors/Creators: |
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Copyright, Publisher and Additional Information: | © 2020 by the National Kidney Foundation, Inc. This is an author produced version of a paper subsequently published in American Journal of Kidney Diseases. Uploaded in accordance with the publisher's self-archiving policy. Article available under the terms of the CC-BY-NC-ND licence (https://creativecommons.org/licenses/by-nc-nd/4.0/). |
Dates: |
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Institution: | The University of Sheffield |
Academic Units: | The University of Sheffield > Sheffield Teaching Hospitals |
Depositing User: | Symplectic Sheffield |
Date Deposited: | 07 May 2020 08:35 |
Last Modified: | 22 Nov 2021 14:17 |
Status: | Published |
Publisher: | Elsevier BV |
Refereed: | Yes |
Identification Number: | 10.1053/j.ajkd.2020.01.005 |
Open Archives Initiative ID (OAI ID): | oai:eprints.whiterose.ac.uk:160349 |
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