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.

Funding Body

Children’s Hospital Foundation Translator Grant

NHMRC Medical Research Future Fund (MRFF)

Thrasher Research Fund

Further Details

Read our blog on BETTER outcomes for children and adults with bronchiectasis.

Learn about the Cough and Airways Research Group (CAARG).

For further information, please contact investigator A/Prof Julie Marchant at  jm.marchant@qut.edu.au.

QUT

Prof Anne Chang

Prof Stephanie Yerkovich

A/Prof Julie Marchant

A/Prof Brent Masters

Prof Steven McPhail

Dr Hannah O’Farrell

Dr Vikas Goyal

Dr Margaret McElrea

Partners

Prof Keith Grimwood (Griffith University)

Dr Nic West (Griffith University)

Prof Peter Morris (Menzies School of Health Research)

Dr Gabrielle McCallum (Menzies School of Health Research)

A/Prof Robyn Marsh (Menzies School of Health Research)

Mrs Lesley Versteegh (Menzies School of Health Research)

A/Prof Lucy Morgan (Concord Repatriation General Hospital)

A/Prof Lucy Burr (Department of Respiratory and Sleep Medicine)

Dr Katherine Baines (University of Newcastle)

Prof Paul Torizillo (Department of Respiratory Medicine)

Assoc Prof Andre Schultz (University of Western Australia)