TY - JOUR
T1 - Developing Consensus-Based Outcome Domains for Trials in Children and Adolescents With CKD: An International Delphi Survey
AU - Logeman, Charlotte
AU - Guha, Chandana
AU - Howell, Martin
AU - Hanson, Camilla S.
AU - Craig, Jonathan C.
AU - Samuel, Susan
AU - Zappitelli, Michael
AU - Matsuda-Abedini, Mina
AU - Dart, Allison
AU - Furth, Susan
AU - Eddy, Allison
AU - Groothoff, Jaap
AU - Yap, Hui-Kim
AU - Bockenhauer, Detlef
AU - Sinha, Aditi
AU - Alexander, Stephen I.
AU - Goldstein, Stuart L.
AU - Gipson, Debbie S.
AU - Michael, Mini
AU - Walker, Amanda
AU - Kausman, Joshua
AU - Gaillard, Segolene
AU - Bacchetta, Justine
AU - Rheault, Michelle N.
AU - Warady, Bradley A.
AU - Neu, Alicia
AU - Christian, Martin
AU - McTaggart, Steven
AU - Liu, Isaac
AU - Teo, Sharon
AU - Sautenet, Benedicte
AU - Gutman, Talia
AU - Carter, Simon
AU - Teixeira-Pinto, Armando
AU - Tong, Allison
PY - 2020/10
Y1 - 2020/10
N2 - 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.
AB - 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.
KW - Chronic kidney disease (CKD)
KW - Delphi survey
KW - adolescents
KW - blood pressure
KW - caregivers
KW - children
KW - clinical trial design
KW - consensus
KW - core outcome set
KW - kidney function
KW - life participation
KW - mortality
KW - outcome measures
KW - outcomes
KW - patient-centered outcomes
KW - pediatrics
KW - priority ranking
KW - treatment goals
UR - http://www.scopus.com/inward/record.url?scp=85087711562&partnerID=8YFLogxK
U2 - https://doi.org/10.1053/j.ajkd.2020.03.014
DO - https://doi.org/10.1053/j.ajkd.2020.03.014
M3 - Article
C2 - 32654889
SN - 0272-6386
VL - 76
SP - 533
EP - 545
JO - American Journal of Kidney Diseases
JF - American Journal of Kidney Diseases
IS - 4
ER -