Late Breaking Abstract - Efficacy and safety of azithromycin maintenance therapy in primary ciliary dyskinesia – The BESTCILIA randomized placebo-controlled trial

Abstract

Introduction: Use of maintenance therapy with the macrolide antibiotic azithromycin (AZI) is increasing in different chronic respiratory disorders incl. primary ciliary dyskinesia (PCD), although no clear evidence exists in PCD. Aims and Objectives: The primary objective was to determine efficacy of AZI maintenance therapy on respiratory exacerbations in PCD. The secondary objectives were to determine efficacy on lung function, health-related quality of life, sputum microbiology, and hearing impairment, and assess safety. Methods: Patients with confirmed diagnosis of PCD, age 7-50 years, were enrolled in this European multi-centre, parallel group, double-blind, randomized, placebo-controlled trial. Participants were randomly assigned to AZI 250/500 mg according to body weight ( Results: A total of 90 participants were randomized - 49 assigned to AZI and 41 to placebo. The AZI group had significant lower rate of exacerbations (rate ratio 0.46, 95%CI: 0.27; 0.80, p=0.006) and pathogenic bacterial species in sputum (rate ratio 0.39, 95%CI: 0.24; 0.64, p=0.0002), compared to the placebo group. There was no significant difference between the treatment groups in lung function, hearing level, or Respiratory symptoms, Sinus symptoms and Ear & hearing symptoms on the quality of life measure. Gastrointestinal complaints were more common in the AZI group. Conclusions: This first multi-national randomized controlled trial on pharmacotherapy in PCD showed that maintenance AZI for 6 months was safe and reduced rate of respiratory exacerbations and rate of pathogenic bacterial species in sputum to less than half.