Azithromycin did not reduce extent of structural lung disease in infants with cystic fibrosis


disclosures: Paste reports receiving salary funding from the National Health and Medical Research Counsel. See the study for the relevant financial disclosures from all other authors.

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Treatment with azithromycin from the time of childhood cystic fibrosis diagnosis did not reduce the extent of structural lung disease at 36 months of age, but reduced airway inflammation and morbidity, according to the COMBAT CF study.

In addition, azithromycin treatment was safe and improved some clinical outcomes, researchers reported in: The Lancet Respiratory Medicine.

Stephen M. Stick, PhD, quote
Data are derived from Stick SM, et al. Lancet Respir Med† 2022;doi:10.1016/S2213-2600(22)00165-5.

“Until recently, there were no proven interventions in newborns with cystic fibrosis that address the fundamental pathological triggers for lung disease – dehydration and inflammation of the airways. There are now two options: hypertonic saline, which moisturizes the airways and stops the progression of structural damage.” , and azithromycin, which reduces inflammation.” Stephen M. Stick, PhD, from the Department of Respiratory and Sleep Medicine at Perth Children’s Hospital, the University of Western Australia and the Wal-yan Respiratory Research Center at the Telethon Kids Institute, to Healio. “Azithromycin should be considered in combination with hypertonic saline for any young child who does not have access to the new class of modulator therapy that has been shown to be transformational for much older individuals with cystic fibrosis.”

Stick and colleagues conducted the randomized, double-blind, placebo-controlled Phase 3 COMBAT CF trial from June 2012 to July 2017, which enrolled 130 patients from eight pediatric cystic fibrosis centers in Australia and New Zealand. Infants were 3 to 6 months old. and was diagnosed with cystic fibrosis. Infants were randomly assigned to oral azithromycin 10 mg/kg body weight (n = 68) three times per week or placebo (n = 62) until 36 months of age.

The primary outcomes were the percentage of children with radiologically defined bronchiectasis and the percentage of total lung volume affected.

At 36 months of age, 88% of infants assigned to azithromycin and 94% to placebo had bronchiectasis (OR = 0.49; 95% CI 0.12-2).

The investigators reported no difference between groups in total respiratory disease (median difference = -0.02%; 95% CI, -0.59 to 0.56).

Infants assigned azithromycin had fewer hospital days for pulmonary exacerbations (mean difference = -6.3; 95% CI, -10.5 to –2.1) and fewer courses of inhaled or oral antibiotics (incidence ratio = 0 .88, 95% CI 0.81 -0.97).

Airway inflammatory concentrations were also lower in infants assigned azithromycin, including interkeukin-8 (median difference = –1.2; 95% CI, –1.9 to –0.5) and neutrophil elastase activity (median difference = –0.6; 95% CI, –1.1 to –0.5) –0.2) at 36 months in the pre-planned exploratory analysis.

Investigators observed no azithromycin effect on BMI at 36 months (mean difference = 0.4; 95% CI, -0.1 to 0.9).

In addition, according to the researchers, there was no evidence of pathogen emergence with azithromycin treatment, and there were few adverse outcomes with no differences between those who received the study drug or placebo.

†[Cystic fibrosis transmembrane conductance regulator] modulator therapy changes the lives of people with cystic fibrosis,” Stick told Healio. “However, because of the cost of these therapies, very few young children outside the US have access to them. … Therefore, clinical trials in young children are needed comparing the efficacy of modulator therapies to azithromycin plus hypertonic saline.”

For more information:

Stephen M. Stick, PhD, can be reached at

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