Aztreonam for inhalation solution (AZLI) in patients with cystic fibrosis, mild lung impairment, and P. aeruginosa

Abstract

Background Previous aztreonam for inhalation solution (AZLI) studies included patients with cystic fibrosis, Pseudomonas aeruginosa ( PA) airway infection, and forced expiratory volume in 1 s (FEV 1) 25% to 75% predicted. This double-blind, multicenter, randomized, placebo-controlled trial enrolled patients (≥ 6 years) with FEV 1 > 75% predicted. Methods AZLI 75 mg (n = 76) or placebo (n = 81) was administered 3-times daily for 28 days with a 14-day follow-up. Results Day 28 treatment effects were 1.8 points for CFQ-R-Respiratory Symptoms Scale (95% CI: −2.8, 6.4; p = 0.443; primary endpoint); −1.2 for log 10 sputum PA colony-forming units (p = 0.016; favoring AZLI), and 2.7% for relative FEV 1% predicted (p = 0.021; favoring AZLI). Treatment effects favoring AZLI were larger for patients with baseline FEV 1 < 90% predicted compared to ≥ 90% predicted. AZLI was well-tolerated. Conclusions Effects on respiratory symptoms were modest; however, FEV 1 improvements and bacterial density reductions support a possible role for AZLI in these relatively healthy patients. ClinicalTrials.gov identifier: NCT00712166.