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Article Dans Une Revue American Journal of Kidney Diseases Année : 2020

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

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

Résumé

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.

Dates et versions

hal-03159462 , version 1 (04-03-2021)

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Citer

Yeoungjee Cho, Gopala Rangan, Charlotte Logeman, Hyunjin Ryu, Benedicte Sautenet, et al.. Core Outcome Domains for Trials in Autosomal Dominant Polycystic Kidney Disease: An International Delphi Survey. American Journal of Kidney Diseases, 2020, ⟨10.1053/j.ajkd.2020.01.005⟩. ⟨hal-03159462⟩
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