Project Details

Preventing bronchiectasis in children: a multicenter RCT and cohort study

Anne Chang, MBBS, MPHTM, PhD, FRACP, FASPR

Summary

BACKGROUND: Bronchiectasis is a serious neglected disease and the burden/importance of chronic cough (the most common bronchiectasis symptom) often goes unrecognized, and has large unmet patient needs. Addressing prevention factors through prospective studies are clinician and consumer priorities. GAP: There are currently no child bronchiectasis prevention nor disease characterization studies that can identifying pheno-endotypes. HYPOTHESIS: Our hypotheses are: In children with chronic wet cough (CWC), 9-months of azithromycin (vs controls) reduces (i) future bronchiectasis or recurrent-protracted bacterial bronchitis (PBB) and, (ii) improves clinical outcomes and will be cost saving, in those with ‘high-risk traits;’ and (iii) gene expression signature and/or airway shotgun metagenomic biomarkers can identify those who will develop airway diseases of recurrent-PBB, bronchiectasis +/- asthma. METHODS: International multicenter cohort study with an embedded parallel, superiority RCT. For the RCT, we plan to recruit 368 children (9 sites, 4 countries) where children will be randomized to receive 9-months of azithromycin or no treatment (controls). Our primary outcome is the proportion of children with recurrent-PBB (>3 episodes PBB/yr) by 12-months or bronchiectasis and, secondary outcomes include quality-of-life score and asthma. RESULTS: Pending. IMPACT: Our study will likely lead to the first ever intervention for preventing bronchiectasis, and if successful, it will improve children’s quality-of-life and future lung health. It may provide novel discovery data that focus on improving clinical management by adopting precision medicine approaches through studying whether blood gene expression signature and/or airway shotgun metagenomic biomarkers can identify those who will develop airway diseases of recurrent-PBB, bronchiectasis +/- asthma.