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

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

Edité par CCSD ; Elsevier -

International audience. 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.

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