92 - Conduct and Reporting of Pilot Randomized Controlled Trials in Pediatrics: A Systematic Review

Publication Number: 2088.92

Anjali Kukan, The Hospital for Sick Children, Toronto, ON, Canada; Anmol Samra, The Hospital for Sick Children, Toronto, ON, Canada; Abby Tafler, University of Toronto Temerty Faculty of Medicine, Toronto, ON, Canada; Amber Lee, University of Toronto Temerty Faculty of Medicine, Oakville, ON, Canada; Kevin Thorpe, University of Toronto, Toronto, ON, Canada; Jessie Cunningham, The Hospital for Sick Children, Toronto, ON, Canada; Vincent Albert Flores, The Hospital for Sick Children, London, ON, Canada; Sanjay Mahant, The Hospital for Sick Children, Toronto, ON, Canada; Peter J. Gill, The Hospital For Sick Children, Toronto, ON, Canada

Background: Pilot randomized controlled trials (RCTs) are smaller-scale studies conducted to assess the feasibility of definitive trials by first testing key metrics such as recruitment, retention, and intervention acceptability. The Consolidated Standards of Reporting Trials (CONSORT) statement, first published in 1997, outlines essential items for reporting clinical trials. In 2016, CONSORT released an extension for randomized pilot and feasibility trials to address persistent gaps in reporting. Several reviews have investigated the reporting quality across disciplines using the CONSORT extension, yet none have provided a comprehensive overview specific to pediatrics.

Objective: This systematic review sought to evaluate adherence to the CONSORT extension and examined methodological characteristics of pediatric pilot RCTs. Findings aim to identify strengths and opportunities for improvement in pediatric trial design.

Design/Methods: A random sample of 100 individual-level pediatric (≤18 years) pilot and feasibility RCTs published between 2020-2024 were included, regardless of setting or outcome. Studies were identified through Ovid MEDLINE, Ovid EMBASE, Cochrane CENTRAL, Ebsco CINAHL, and Clarivate Web of Science, and screened in duplicate using Covidence. Journal endorsement of CONSORT, multisite design, industry funding, sample size, a published trial protocol, and registration in a clinical trial database were evaluated as predictors of better CONSORT adherence using an ordinal regression model.

Results: The mean adherence to the CONSORT extension was 56%, with only 9% exhibiting high adherence (≥75% items). Feasibility criteria were evaluated in 56% of studies, with the most evaluated metrics being acceptability (54% [30/56]), protocol adherence (48% [27/56]), and safety (45% [25/56]). Studies that provided a registration number (odds ratio [OR]: 9.9; 95% confidence interval [CI] 3.4-29.3) and published the protocol (OR: 7.4; 95% CI 2.0-27.2) showed higher adherence to CONSORT extension guidelines, indicating more complete reporting of trial items.

Conclusion(s): Pediatric pilot trial reporting quality remains suboptimal, with considerable variation in adherence to CONSORT guidelines. Editors, reviewers, and sponsors can promote CONSORT adherence during manuscript submission to enhance reporting quality, and further efforts to support guideline implementation are warranted.

Table 1. Characteristics of Included Pediatric Pilot Studies
Characteristics of the included studies. Most were done in North America, led by universities, funded by governments, and often focused on neurology, mental health, and pediatric medical sub-specialties.

Figure 1. Forest plot for the ordinal regression analysis.
Studies that published a study protocol or listed a trial registration number had higher odds of better CONSORT adherence