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

https://doi.org/10.1053/j.ajkd.2020.03.014Get rights and content

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.

Section snippets

Study Design

The Delphi survey has been used to generate consensus on core outcomes to be used in trials for different patient populations, including children with other medical conditions.17, 18, 19, 20, 21, 22 The SONG-Kids Delphi survey was administered online with 2 rounds. Participants prioritized outcomes for trials in children across all stages of CKD. The survey was conducted in 3 languages (English, French, and Hindi). The English survey was translated into French and Hindi by a bilingual HCP and

Participant Characteristics

In round 1, 557 participants across 48 countries completed the survey, of whom 72 (13%) were patients, 132 (24%) were family members/caregivers, and 353 (63%) were HCPs. By language, 533 (96%; 71 patients, 123 caregivers, and 339 HCPs) completed the English survey, 23 (4%; 1 patient, 8 caregivers, and 14 HCPs) completed the French survey, and 1 (0.1%; 1 caregiver) completed the survey in Hindi. In round 2, a total of 312 (56% overall retention rate) participants from 48 countries completed the

Discussion

Mortality, kidney function, life participation, blood pressure, cardiovascular disease, and infection were the outcomes of highest priority to children with CKD, their caregivers, and HCPs. The top 4 outcomes (mortality, kidney function, life participation, and blood pressure) were consistently within the top 5 across all 3 stakeholder groups. For patients, infection was in the top 5, and self-esteem and depression were in the top 5 for caregivers and HCPs, respectively. Collectively, these

Article Information

Authors’ Full Names and Academic Degrees

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,

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