Range and Heterogeneity of Outcomes in Randomized Trials of Pediatric Chronic Kidney Disease

Lauren Chong, Benedicte Sautenet, Allison Tong, Camilla Hanson, Susan Samuel, Michael Zappitelli, Allison Dart, Susan Furth, Allison Eddy, Jaap Groothoff, Nicholas Webb, Hui-Kim Yap, Detlef Bockenhauer, Aditi Sinha, Stephen Alexander, Stuart Goldstein, Debbie Gipson, Gayathri Raman, Jonathan Craig

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15 Citations (Scopus)

Abstract

Objective To determine the range and heterogeneity of outcomes reported in randomized controlled trials of interventions for children with chronic kidney disease (CKD). Study design The Cochrane Kidney and Transplant Specialized Register was searched to March 2016. Randomized trials involving children across all stages of CKD were selected. All outcome domains and measurements were extracted from included trials. The frequency and characteristics of the outcome domains and measures were evaluated. Results From 205 trials included, 6158 different measurements of 100 different outcome domains were reported, with a median of 22 domains per trial (IQR 13-41). Overall, 52 domains (52%) were surrogate, 38 (38%) were clinical, and 10 (10%) were patient-reported. The 5 most commonly reported domains were blood pressure (76 [37%] trials), relapse/remission (70 [34%]), kidney function (66 [32%]), infection (61 [30%]), and height/pubertal development (51 [25%]). Mortality (14%), cardiovascular disease (4%), and quality of life (1%) were reported infrequently. The 2 most frequently reported outcomes, blood pressure and relapse/remission, had 56 and 81 different outcome measures, respectively. Conclusions The outcomes reported in clinical trials involving children with CKD are extremely heterogeneous and are most often surrogate outcomes, rather than clinical and patient-centered outcomes such as cardiovascular disease and quality of life. Efforts to ensure consistent reporting of outcomes that are important to patients and clinicians will improve the value of trials to guide clinical decision-making. In our study, non-English articles were excluded.

Original languageEnglish
Pages (from-to)110-117.e11
Number of pages19
JournalJournal of Pediatrics
Volume186
DOIs
Publication statusPublished - 2017

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    Chong, L., Sautenet, B., Tong, A., Hanson, C., Samuel, S., Zappitelli, M., Dart, A., Furth, S., Eddy, A., Groothoff, J., Webb, N., Yap, H-K., Bockenhauer, D., Sinha, A., Alexander, S., Goldstein, S., Gipson, D., Raman, G., & Craig, J. (2017). Range and Heterogeneity of Outcomes in Randomized Trials of Pediatric Chronic Kidney Disease. Journal of Pediatrics, 186, 110-117.e11. https://doi.org/10.1016/j.jpeds.2017.03.034