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Assessment of Vascular Function in Patients With Chronic Kidney Disease
Published on: June 16, 2014
Range and Heterogeneity of Outcomes in Randomized Trials of Pediatric Chronic Kidney Disease
Lauren S H Chong1, Benedicte Sautenet2, Allison Tong1
1Sydney School of Public Health, The University of Sydney, Sydney, Australia; Centre for Kidney Research, The Children's Hospital at Westmead, Sydney, Australia.
Insights
Outcomes in pediatric chronic kidney disease (CKD) trials are highly varied, with a focus on surrogate measures over patient-centered results like quality of life. Standardizing outcome reporting is crucial for better clinical decision-making in pediatric CKD research.
Area of Science:
- Nephrology
- Pediatric Nephrology
- Clinical Trials Methodology
Background:
- Randomized controlled trials (RCTs) are essential for evaluating interventions in pediatric chronic kidney disease (CKD).
- The heterogeneity of reported outcomes in these trials can complicate the interpretation and application of findings.
- Standardizing outcome reporting is needed to enhance the clinical utility of research in pediatric CKD.
Purpose of the Study:
- To systematically assess the range and diversity of outcomes reported in RCTs for children with CKD.
- To identify the most frequently reported outcome domains and measures in this population.
- To evaluate the proportion of surrogate versus clinical and patient-reported outcomes.
Main Methods:
- A comprehensive search of the Cochrane Kidney and Transplant Specialized Register up to March 2016 was conducted.
- RCTs involving pediatric participants across all stages of CKD were included.
- Outcome domains and measurements were extracted, and their frequency and characteristics were analyzed.
Main Results:
- 205 trials reported 6,158 different measurements across 100 outcome domains.
- A median of 22 outcome domains were reported per trial.
- Surrogate outcomes (52%) predominated over clinical (38%) and patient-reported (10%) outcomes. Blood pressure and relapse/remission were most common, while mortality, cardiovascular disease, and quality of life were infrequent.
Conclusions:
- Clinical trials for pediatric CKD exhibit significant outcome heterogeneity, with a strong emphasis on surrogate endpoints.
- Patient-centered outcomes such as quality of life and cardiovascular disease were infrequently reported.
- Harmonizing outcome reporting is vital to improve the value of evidence for guiding clinical practice and decision-making in pediatric CKD.
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.
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