An 18-Month Study of the Safety and Efficacy of Repeated Courses of Inhaled Aztreonam Lysine in Cystic Fibrosis

Chronic airway infection with Pseudomonas aeruginosa (PA) causes morbidity and mortality in patients with cystic fibrosis (CF) Additional anti PA therapies are needed to improve health status and health related quality of life AIR CF3 was an international 18 month open label study to evaluate the safety and efficacy of repeated courses of aztreonam for inhalation solution (AZLI now marketed as Cayston (R)) in patients aged >= 6 years with CF and PA infection who previously participated in one of two Phase 3 studies AIR CF1 or AIR CF2 Patients received up to nine courses (28 days on/28 days off) of 75 mg AZLI two (BID) or three times daily (TID) based on randomization in the previous trials 274 patients mean age 28 5 years (range 8-74 years) participated Mean treatment adherence was high (92 0% BID group 88 0% TID group) Hospitalization rates were low and adverse events were consistent with CF With each course of AZLI FEV1 and scores on the Cystic Fibrosis Questionnaire Revised Respiratory Symptom scale improved and bacterial density in sputum was reduced Benefits waned in the 28 days off therapy but weight gain was sustained over the 18 months There were no sustained decreases in PA susceptibility A dose response was observed AZLI TID treated patients demonstrated greater improvements in lung function and respiratory symptoms over 18 months Repeated intermittent 28 day courses of AZLI treatment were well tolerated Clinical benefits in pulmonary function health related quality of life and weight were observed with each course of therapy AZLI is a safe and effective new therapy in patients with CF and PA airway infection Pediatr Pulmonol 2010, 45 1121-1134 (C) 2010 Wiley Liss Inc