Abstract
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 language | English (US) |
|---|---|
| Pages (from-to) | 533-545 |
| Number of pages | 13 |
| Journal | American Journal of Kidney Diseases |
| Volume | 76 |
| Issue number | 4 |
| DOIs | |
| State | Published - Oct 2020 |
Bibliographical note
Funding Information:Ms Logeman and Dr Hanson are supported by the National Health and Medical Research Council (NHMRC) Program grant ( ID1092957 ). Dr Tong is supported by an NHMRC Fellowship ( ID1106716 ). The study was funded by The University of Sydney Research Accelerator Grant and the NHMRC Program Grant Better Evidence and Translation in Chronic Kidney Disease (BEAT-CKD; ID1092957). The funders had no role in the study design, data collection, analysis, reporting, or decision to submit for publication.
Funding Information:
Charlotte Logeman, MPH, Chandana Guha, MPhil, Martin Howell, PhD, Camilla S. Hanson, PhD, Jonathan C. Craig, PhD, Susan Samuel, MD, Michael Zappitelli, MD, Mina Matsuda-Abedini, MD, Allison Dart, MD, Susan Furth, PhD, Allison Eddy, MD, Jaap Groothoff, PhD, Hui-Kim Yap, MD, Detlef Bockenhauer, PhD, Aditi Sinha, MD, Stephen I. Alexander, PhD, Stuart L. Goldstein, MD, Debbie S. Gipson, MD, Mini Michael, MD, Amanda Walker, MD, Joshua Kausman, PhD, Segolene Gaillard, MD, Justine Bacchetta, MD, Michelle N. Rheault, MD, Bradley A. Warady, MD, Alicia Neu, MD, Martin Christian, MD, Steven McTaggart, PhD, Isaac Liu, MD, Sharon Teo, MD, Benedicte Sautenet, PhD, Talia Gutman, MPH, Simon Carter, MD, Armando Teixeira-Pinto, PhD, and Allison Tong, PhD. Research idea and study design: all authors; data acquisition: CL, CSH, JCC, SS, MZ, MM-A, AD, SF, AE, HY, DB, AS, SIA, DSG, MM, AW, JK, SG, JB, MNR, BAW, AN, MC, SM, IL, ST, BS, TG, SC, AT; data analysis/interpretation: CL, CG, MH, CSH, JCC, SS, MZ, AD, SF, AE, JG, HY, DB, AS, DSG, MM, AW, JK, MNR, BAW, AN, MC, SM, BS, TG, ATP, AT; supervision or mentorship: MH, AT, JCC. Each author contributed important intellectual content during manuscript drafting or revision and agrees to be personally accountable for the individual's own contributions and to ensure that questions pertaining to the accuracy or integrity of any portion of the work, even one in which the author was not directly involved, are appropriately investigated and resolved, including with documentation in the literature if appropriate. Ms Logeman and Dr Hanson are supported by the National Health and Medical Research Council (NHMRC) Program grant (ID1092957). Dr Tong is supported by an NHMRC Fellowship (ID1106716). The study was funded by The University of Sydney Research Accelerator Grant and the NHMRC Program Grant Better Evidence and Translation in Chronic Kidney Disease (BEAT-CKD; ID1092957). The funders had no role in the study design, data collection, analysis, reporting, or decision to submit for publication. The authors declare that they have no relevant financial interests. We thank all the patients, caregivers, and health care professionals who gave their time to participate in the study. Received November 21, 2019. Evaluated by 2 external peer reviewers, with direct editorial input from an Associate Editor, who served as Acting Editor-in-Chief. Accepted in revised form March 8, 2020. The involvement of an Acting Editor-in-Chief was to comply with AJKD's procedures for potential conflicts of interest for editors, described in the Information for Authors & Journal Policies.
Publisher Copyright:
© 2020 National Kidney Foundation, Inc.
Keywords
- 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
