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Updated: Mar 16, 2026
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Published on: July 19, 2021
How randomised trials have improved the care of children with kidney disease
Elisabeth M Hodson1,2, Jonathan C Craig3,4
1Centre for Kidney Research, The Children's Hospital at Westmead, Locked Bag 4001, Westmead, Sydney, NSW, 2145, Australia. elisabeth.hodson@health.nsw.gov.au.
Insights
Randomised controlled trials (RCTs) are crucial for evaluating interventions in pediatric kidney disease. Research in urinary tract infections and nephrotic syndrome has significantly advanced care for children with kidney conditions.
Area of Science:
- Pediatric Nephrology
- Clinical Trial Methodology
- Evidence-Based Medicine
Background:
- Randomized controlled trials (RCTs) are the gold standard for assessing intervention efficacy and safety.
- Participant randomization balances known and unknown characteristics between groups, attributing outcome differences to interventions.
- A comprehensive registry of kidney disease trials was searched to identify relevant pediatric studies.
Purpose of the Study:
- To identify and analyze randomized controlled trials (RCTs) involving pediatric kidney disease patients.
- To highlight key advances in pediatric nephrology driven by clinical trial evidence.
- To assess the landscape of pediatric kidney disease research, focusing on common conditions.
Main Methods:
- Systematic search of the Cochrane Kidney and Transplant's Specialised Register.
- Identification and categorization of 482 RCTs involving children with kidney diseases.
- Analysis of trial characteristics, including participant numbers and specific conditions studied.
Main Results:
- A total of 482 pediatric trials were identified, with the majority focusing on urinary tract infection (UTI) and nephrotic syndrome.
- Most trials were small, with a median of 46 participants.
- Only 26 trials enrolled 200 or more participants, with 18 of these focusing on UTI.
Conclusions:
- RCTs are fundamental to advancing pediatric kidney disease care.
- Significant progress in managing UTI, nephrotic syndrome, chronic kidney disease (CKD), and kidney transplantation in children is attributed to trial findings.
- Future research should consider trial size and scope to maximize impact on pediatric nephrology outcomes.
Abstract:
Randomised controlled trials (RCTs) provide the most reliable way to evaluate the benefits and harms of interventions. Participants are divided into groups using methods that balance the characteristics (both known and unknown) of the participants between treatment groups; thus, differences in outcomes are due to the interventions administered. From Cochrane Kidney and Transplant's Specialised Register, a comprehensive registry of trials in kidney disease, we identified 482 trials involving children. The vast majority concerned urinary tract infection (UTI; 134) and nephrotic syndrome (136). Most were small, with a median enrolment of 46 children, with only 26 trials enrolling 200 or more participants, and of these, 18 involved children with UTI. We discuss a number of important advances in the care of children with UTI with or without vesico-ureteric reflux, nephrotic syndrome, chronic kidney disease (CKD) and kidney transplantation that have been driven largely by trials in these conditions.
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