Prophylactic azithromycin in patients with primary ciliary dyskinesia

Introduction: Primary ciliary dyskinesia (PCD) is a rare genetic disease characterized by abnormalities in ciliary function, responsible for abnormal mucus clearance and repeated respiratory tract infections. The potential benefit of macrolide antibiotics has been evaluated in variety of chronic respiratory diseases. The efficacy of long-term azithromycine (AZT) has never been studied in PCD patients. Aim: The aim of this retrospective study was to investigate the effect of long-term AZT on the decrease in annual number of pulmonary exacerbations in children with PCD. Materials and Methods: All of our patients followed in our Pediatric center and who received low dose of AZT for more than 6 months were included. The clinical data (rate of pulmonary exacerbations, lung function analyses, ENT symptoms and changes in the airways bacterial colonizations) were collected from 1 year before and up to 5 years after the initiation of the AZT treatment. Results: A total of 31 children were included (11 years +/- 4 years). After 1 year, we observed a significant reduction in the annual number of pulmonary exacerbations (on average 1.7 +/- 2.7 exacerbations per year before treatment versus 0.5 +/- 1 exacerbation per year after 1 year of treatment) and a significant improvement in ENT symptoms. However, the lung function decline was not modified. No difference in the airways colonizations by pathogens, nor emerging antibiotic resistance were observed during the treatment. Conclusion: Our study showed a clinical improvement of PCD children treated by long term AZT. Those results must be confirmed by a larger multicenter study, such as the one currently conducted by the BEAT PCD group.