• Andrew G Miller, Martha Aq Curley, Claire Destrampe, Heidi Flori, Robinder Khemani, Amy Ohmer, Neal J Thomas, Nadir Yehya, Shan Ward, Leanne West, Kanecia O Zimmerman, Saranya Venkatachalam, Sonya Sutton, and Christoph P Hornik.
• Division of Pediatric Critical Care Medicine, Duke University Medical Center, Durham, North Carolina; and Respiratory Care Services, Duke University Medical Center, Durham, North Carolina. Andrew.g.miller@duke.edu.
• Respir Care. 2024 Sep 26; 69 (10): 128412931284-1293.

BackgroundPediatric ARDS is associated with significant morbidity and mortality. High-quality data from clinical trials in children are limited due to numerous barriers to their design and execution. Here we describe the collaborative development of a master protocol as a tool to address some of these barriers and support the conduct of pediatric ARDS studies.MethodsUsing PubMed, we performed a literature search of randomized controlled trials (RCTs) in pediatric ARDS to characterize the current state and evaluate potential benefit of harmonized master protocols. We used a multi-stakeholder, collaborative, and team science-oriented process to develop a master protocol template with links to common data elements (CDEs) for pediatric ARDS trials.ResultsWe identified 11 RCTs that enrolled between 14-200 total subjects per trial. Interventions included mechanical ventilation, prone positioning, corticosteroids, and surfactant. Studies displayed significant heterogeneity in ARDS definition, design, inclusion and exclusion criteria, and reported outcomes. Mortality was reported in 91% of trials and ventilator-free days in 73%. The trial heterogeneity made pooled analysis unfeasible. These findings underscore the need for a method to facilitate combined analysis of future trials through standardization of trial elements. As a potential solution, we developed a master protocol, iteratively revised with input from a multidisciplinary panel of experts and organized into 3 categories: instructions and general information, templated language, and a series of text options of common pediatric ARDS trial scenarios. Finally, we linked master protocol sections to relevant CDEs previously defined for pediatric ARDS and captured in a series of electronic case report forms.ConclusionsThe majority of pediatric ARDS trials identified were small and heterogeneous in study design and outcome reporting. Using a master protocol template for pediatric ARDS trials with CDEs would support combining and comparing pediatric ARDS trial findings and increase the knowledge base.Copyright © 2024 by Daedalus Enterprises.

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