Developing Consensus-Based Outcome Domains for Trials in Children and Adolescents With CKD: An International Delphi Survey

Charlotte Logeman, Chandana Guha, Martin Howell, Camilla S. Hanson, Jonathan C. Craig, Susan Samuel, Michael Zappitelli, Mina Matsuda-Abedini, Allison Dart, Susan Furth, Allison Eddy, Jaap Groothoff, Hui Kim Yap, Detlef Bockenhauer, Aditi Sinha, Stephen I. Alexander, Stuart L. Goldstein, Debbie S. Gipson, Mini Michael, Amanda WalkerJoshua Kausman, Segolene Gaillard, Justine Bacchetta, Michelle N. Rheault, Bradley A. Warady, Alicia Neu, Martin Christian, Steven McTaggart, Isaac Liu, Sharon Teo, Benedicte Sautenet, Talia Gutman, Simon Carter, Armando Teixeira-Pinto, Allison Tong

Research output: Contribution to journalArticlepeer-review


Rationale & Objective: The inconsistency in outcomes reported and lack of patient-reported outcomes across trials in children with chronic kidney disease (CKD) limits shared decision making. As part of the Standardized Outcomes in Nephrology (SONG)-Kids initiative, we aimed to generate a consensus-based prioritized list of critically important outcomes to be reported in all trials in children with CKD. Study Design: An online 2-round Delphi survey in English, French, and Hindi languages. Settings & Participants: Patients (aged 8-21 years), caregivers/family, and health care professionals (HCPs) rated the importance of outcomes using a 9-point Likert scale (7-9 indicating critical importance) and completed a Best-Worst Scale. Analytical Approach: We assessed the absolute and relative importance of outcomes. Comments were analyzed thematically. Results: 557 participants (72 [13%] patients, 132 [24%] caregivers, and 353 [63%] HCPs) from 48 countries completed round 1 and 312 (56%) participants (28 [40%] patients, 64 [46%] caregivers, and 220 [56%] HCPs) completed round 2. Five outcomes were common in the top 10 for each group: mortality, kidney function, life participation, blood pressure, and infection. Caregivers and HCPs rated cardiovascular disease higher than patients. Patients gave lower ratings to all outcomes compared with caregivers/HCPs except they rated life participation (round 2 mean difference, 0.1), academic performance (0.1), mobility (0.4), and ability to travel (0.4) higher than caregivers and rated ability to travel (0.4) higher than HCPs. We identified 3 themes: alleviating disease and treatment burden, focusing on the whole child, and resolving fluctuating and conflicting goals. Limitations: Most participants completed the survey in English. Conclusions: Mortality, life participation, kidney function, and blood pressure were consistently highly prioritized by patients, caregivers, and HCPs. Patients gave higher priority to some lifestyle-related outcomes compared with caregivers/HCPs. Establishing critically important outcomes for all trials in children with CKD may improve consistent reporting of survival, kidney health, and clinical and life impact outcomes that are meaningful for decision making.

Original languageEnglish (US)
Pages (from-to)533-545
Number of pages13
JournalAmerican Journal of Kidney Diseases
Issue number4
StatePublished - Oct 2020


  • Chronic kidney disease (CKD)
  • Delphi survey
  • adolescents
  • blood pressure
  • caregivers
  • children
  • clinical trial design
  • consensus
  • core outcome set
  • kidney function
  • life participation
  • mortality
  • outcome measures
  • outcomes
  • patient-centered outcomes
  • pediatrics
  • priority ranking
  • treatment goals

ASJC Scopus subject areas

  • Nephrology


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