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

Cho, Y., Rangan, G., Logeman, C., Ryu, H., Sautenet, B., Perrone, R. D., Nadeau-Fredette, A-C., Mustafa, R. A., Htay, H., Chonchol, M., Harris, T., Gutman, T., Craig, J. C., Ong, A. C. M., Chapman, A., Ahn, C., Coolican, H., Kao, J. T-W., Gansevoort, R. T., Torres, V., Pei, Y., Johnson, D. W., Viecelli, A. K., Teixeira-Pinto, A., Howell, M., Ju, A., Manera, K. E. & Tong, A., Sep-2020, In : American Journal of Kidney Diseases. 76, 3, p. 361-373 13 p.

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  • Core Outcome Domains for Trials in Autosomal Dominant Polycystic Kidney Disease

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

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.

Original languageEnglish
Pages (from-to)361-373
Number of pages13
JournalAmerican Journal of Kidney Diseases
Issue number3
Early online date28-Apr-2020
Publication statusPublished - Sep-2020

ID: 126888822