Improving outcomes for children with bronchiectasis: a multicenter RCT using a novel mucolytic
BACKGROUND: Bronchiectasis, an under-researched and under-serviced, chronic lung disease, has good outcomes in children if diagnosed early and with optimal treatment of the underlying infection, inflammation and impaired airway clearance. GAP: There are currently no licensed therapies specifically for bronchiectasis, deemed as one of the most neglected chronic respiratory illness by the European Respiratory Society. Erdosteine, a novel mucolytic with 4 potential actions specific for bronchiectasis offers an opportunity to reduce acute respiratory exacerbations and sputum volumes in people with bronchiectasis, thus improving clinical outcomes. HYPOTHESIS: Our primary hypothesis is that 12-months of twice-daily erdosteine (compared to placebo) reduces exacerbations in children with bronchiectasis. Our secondary hypotheses are: In children with bronchiectasis, (i) those receiving 12-months of twice-daily erdosteine (compared to placebo) will have better clinical outcomes, and (ii) inflammatory and/or microbial biomarkers could be used to identify pathways and/or predict those at greater risk of recurrent exacerbations. METHODS: We are undertaking a parallel, multicenter, double-blind, randomized controlled trial (RCT), concealed 1:1 allocation. We plan to recruit 174 children (from 5 sites, 3 countries [Australia, Malaysia and Philippines). We will include children (aged 2-19 years) with bronchiectasis who have had ≥2 exacerbations in the last 18-months. Blood and sputum will be collected at baseline and during an exacerbation in some participants. RESULTS: Pending. IMPACT: If our primary hypothesis is correct, its results are immediately applicable and can be incorporated into clinical practice guidelines (Team members are involved in various national/international guidelines eg. the ERS clinical practice guidelines). Results from our secondary aim will provide novel important data but require confirmation in a different (validation) cohort prior to their introduction into clinical practice.