Azithromycin for Poorly Controlled Asthma in Children: A Randomized Controlled Trial

To assess whether adding oral azithromycin to standard asthma therapy in children with poorly controlled asthma improves asthma control.

This trial included 120 children, 5 to 15 years of age, with poorly controlled asthma despite inhaled corticosteroid and/or long-acting β-agonist in combination therapy. Poorly controlled asthma was defined as either a CACT (patients 5 to 11 years old) or an ACT (patients ≥12 years of age) score of ≤19.

The study was an open label randomized controlled trial. Patients who met inclusion criteria were randomized to three times weekly azithromycin (10 mg/kg) for 3 months in addition to standard treatment versus standard treatment alone. All patients were stepped-up in standard therapy at the time of enrollment unless step-up was recently completed within the last 1 month. Patients were assessed at 4 weeks and 12 weeks.

Primary outcomes were CACT and ACT scores at 3 months. Secondary outcomes included asthma control (in accordance with 2018 Global Initiative for Asthma guidelines), number of exacerbations requiring oral steroids or hospitalization, spirometry, positive throat swab results, change in fractional exhaled nitric oxide level, change in percentage of neutrophils in sputum, and adverse effects.

Patients in the azithromycin group versus control group had higher mean CACT and ACT scores at 3 months of 21.7 ± 2.17 vs 18.33 ±2.19 respectively (P < .001).

Children in the azithromycin group demonstrated better asthma control and had fewer exacerbations than those on standard therapy alone. Observed beneficial effects were similar with eosinophilic vs noneosinophilic asthma. There were no significant differences in spirometry, adverse events, or throat swabs between the groups.

The addition of oral azithromycin in pediatric patients with poorly controlled asthma improved asthma control and reduced exacerbations.

The outcomes of this study support adding oral azithromycin to standard therapies in pediatric patients with poorly controlled asthma. Azithromycin appears to be safe and shows clinically relevant improvements in asthma control, based on validated asthma control questionnaires and 2018 Global Initiative for Asthma guidelines. Further investigation with a larger study population in a placebo-controlled study would be valuable, as well as further investigation into which asthma phenotype would benefit the most from this therapy.