Prioritization of Randomized Clinical Trial Questions for Children Hospitalized With Common Conditions
Authors: Coon Eric R. , McDaniel Corrie E. , Paciorkowski Natalia , Grimshaw Meg , Frakes Elizabeth , Ambroggio Lilliam , Auger Katherine A. , Cohen Eyal , Garber Matthew , Gill Peter J. , Jennings Rebecca , Joshi Neha Shirish , Leyenaar JoAnna K. , McCulloh Russell , Pantell Matthew S. , Sauers-Ford Hadley S. , Schroeder Alan R. , Srivastava Raj , Wang Marie E. , Wilson Karen M. , Kaiser Sunitha V. ,   RCT conference series group, Kemper Alex R , Heath Anna , Fromme H. Barrett , Jennings Brittany N , Wainscott Caitlin E , Russell Christopher J , McCulloch Charles E. , Snow Clota H , Alvarez Francisco J , Percelay Jack M , Nicholson Karee S , Morton Kayce M , Dias Magna , Marek Rachel L , Wilcox Roger A , Hyde Sadie A , Mahant Sanjay , Edwards Yeelen R 
Published: May 2024

Importance

There is a lack of randomized clinical trial (RCT) data to guide many routine decisions in the care of children hospitalized for common conditions. A first step in addressing the shortage of RCTs for this population is to identify the most pressing RCT questions for children hospitalized with common conditions.

Objective

To identify the most important and feasible RCT questions for children hospitalized with common conditions.

Design, Setting, and Participants

For this consensus statement, a 3-stage modified Delphi process was used in a virtual conference series spanning January 1 to September 29, 2022. Forty-six individuals from 30 different institutions participated in the process. Stage 1 involved construction of RCT questions for the 10 most common pediatric conditions leading to hospitalization. Participants used condition-specific guidelines and reviews from a structured literature search to inform their development of RCT questions. During stage 2, RCT questions were refined and scored according to importance. Stage 3 incorporated public comment and feasibility with the prioritization of RCT questions.

Main Outcomes and Measures

The main outcome was RCT questions framed in a PICO (population, intervention, control, and outcome) format and ranked according to importance and feasibility; score choices ranged from 1 to 9, with higher scores indicating greater importance and feasibility.

Results

Forty-six individuals (38 who shared demographic data; 24 women [63%]) from 30 different institutions participated in our modified Delphi process. Participants included children’s hospital (n = 14) and community hospital (n = 13) pediatricians, parents of hospitalized children (n = 4), other clinicians (n = 2), biostatisticians (n = 2), and other researchers (n = 11). The process yielded 62 unique RCT questions, most of which are pragmatic, comparing interventions in widespread use for which definitive effectiveness data are lacking. Overall scores for importance and feasibility of the RCT questions ranged from 1 to 9, with a median of 5 (IQR, 4-7). Six of the top 10 selected questions focused on determining optimal antibiotic regimens for 3 common infections (pneumonia, urinary tract infection, and cellulitis).

Conclusions and Relevance

This consensus statementhas identified the most important and feasible RCT questions for children hospitalized with common conditions. This list of RCT questions can guide investigators and funders in conducting impactful trials to improve care and outcomes for hospitalized children.