The BETTER Study: Using erdosteine to improve outcomes of children and adults with bronchiectasis.

Synopsis

Why the research project is important

Bronchiectasis is under-researched and under-serviced. At the Queensland Children’s Hospital, children with bronchiectasis are being increasingly diagnosed (~1/week). There are few randomized controlled trials (RCT) or evidence-based interventions addressing the unmet needs of paediatric bronchiectasis. In contrast to cystic fibrosis, there are no licensed therapies specific for this condition.

Establishing a management plan to reduce the number of exacerbations for children with bronchiectasis is of paramount importance to improve clinical outcomes. Respiratory exacerbations are important as they are associated with future lung function decline and are the major cause of impaired quality of life. Thus, we are investigating the bronchiectasis-specific treatment erdosteine which is a promising novel mucolytic.

What the research seeks to do

Erdosteine is licensed in 40 countries, but not Australia. Historically, this medication has been shown to improve clinical outcomes in other airway conditions.

The BronchiEctasis Trial Testing ERdosteine study is a multi-center, double blinded RCT. We plan to recruit 194 children and adults (aged 2-49 years) with bronchiectasis who have had ≥2 exacerbation in the last 18 months, from 8 national and international sites (3 countries: Australia, Malaysia, and the Philippines). Blood, sputum, and nasal swab samples will be collected at baseline and during exacerbations.

What are the research outcomes/ impact

The primary aim is that 12 months of twice-daily erdosteine (compared to the placebo) results in a reduction of exacerbations in children and adults with bronchiectasis. The secondary aims seek to show that those on erdosteine (compared to the placebo) have better clinical outcomes, including such factors as quality of life, lung function improvement, duration of exacerbations, and we will determine the cost-effectiveness of the treatment.

If our hypothesis is correct, the trial results would aid Therapeutic Goods Administration approval of erdosteine in Australia and incorporation of the novel therapy into bronchiectasis clinical practice guidelines.