L SH CHONG1,2, B SAUTENET1,2, C S HANSON1,2, A TONG1,2, J C CRAIG1,2, ON BEHALF OF SONG-KIDS STEERING GROUP
1Sydney School of Public Health, The University of Sydney, Sydney, New South Wales; 2Centre for Kidney Research, The Children’s Hospital at Westmead, Westmead, Sydney, New South Wales
Aim: To assess the scope and consistency of outcomes reported in randomised controlled trials (RCTs) of interventions for children in any stage of chronic kidney disease (CKD).
Background: RCTs have been conducted to improve outcomes in children with CKD, but the outcomes reported may not be relevant to children, their families or clinicians. The variability in outcome domains and measures also makes it impossible to compare the effectiveness of interventions.
Methods: The Cochrane Renal Register of Controlled Trials was searched for all RCTs involving children across all stages of CKD published before March 2016. The frequency of reporting of each outcome domain, and the measurement characteristics were evaluated.
Results: From the 205 trials, 99 different outcome domains were reported. Across all outcome domains, 50 (51%) were surrogate, 40 (40%) were clinical, and 9 (9%) were patient-reported. The three most commonly reported domains were: blood pressure (75 [37%] trials), medication use/duration (73 [36%] trials) and relapse/remission (72 [35%]). Mortality was reported in 28 (14%) of trials. Cardiovascular disease and quality of life were reported in 8 (4%) and 2 (1%) trials, respectively. There was inconsistency of measures and time points used across all trials. Across the 99 outcome domains, 1671 different measurements were reported.
Conclusions: RCTs involving children with CKD across all disease stages and treatment modalities primarily report surrogate outcomes, rather than focusing on clinical and patient-centred outcomes such as mortality, cardiovascular disease, quality of life and cognition. Furthermore, the multiplicity and heterogeneity of outcomes at all levels – domain, measurement, threshold, and time points – deters efforts to evaluate the comparative effectiveness of interventions and the ability to use trial evidence in decision-making.