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

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  • 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.


  • Cho, Yeoungjee
  • Rangan, Gopala
  • Logeman, Charlotte
  • Ryu, Hyunjin
  • Sautenet, Benedicte
  • Perrone, Ronald D
  • Nadeau-Fredette, Annie-Claire
  • Mustafa, Reem Adel
  • Htay, Htay
  • Chonchol, Michel
  • Harris, Tess
  • Gutman, Talia
  • Craig, Jonathan C
  • Ong, Albert CM
  • 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

publication date

  • September 2020

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