ToolCAP: Novel Tools to improve management of paediatric Community-Acquired Pneumonia - a randomized controlled trial, Statistical Analysis Plan

Abstract

Abstract The ToolCAP cohort study is a prospective, observational, multi-site platform study designed to collect harmonized, high-quality clinical, imaging, and biological data on children with IMCI-defined pneumonia in low- and middle-income countries (LMICs). The primary objective is to inform the development and validation of diagnostic and prognostic tools, including lung ultrasound (LUS), point-of-care biomarkers, and AI-based models, to improve pneumonia diagnosis, management, and antimicrobial stewardship. This statistical analysis plan (SAP) outlines the analytic strategy for describing the study population, assessing the performance of candidate diagnostic tools, and enabling data sharing in support of secondary research questions and AI model development. Children under 12 years presenting with suspected pneumonia are enrolled within 24 hours of presentation and undergo clinical assessment, digital auscultation, LUS, and optional biological sampling. Follow-up occurs on Day 8 and Day 29 to assess outcomes including recovery, treatment response, and complications. The SAP details variable definitions, data management strategies, and pre-specified analyses, including descriptive summaries, sensitivity and specificity of diagnostic tools against clinical reference standards, and exploratory subgroup analyses.