RT Journal Article
SR Electronic
T1 Late Breaking Abstract - Efficacy and safety of azithromycin maintenance therapy in primary ciliary dyskinesia – The BESTCILIA randomized placebo-controlled trial
JF European Respiratory Journal
JO Eur Respir J
FD European Respiratory Society
SP RCT5102
DO 10.1183/13993003.congress-2019.RCT5102
VO 54
IS suppl 63
A1 Kobbernagel, Helene
A1 Buchvald, Frederik F.
A1 Haarman, Eric G.
A1 Casaulta, Carmen
A1 Collins, Samuel A.
A1 Hogg, Claire
A1 Kuehni, Claudia E.
A1 Lucas, Jane S.
A1 Omran, Heymut
A1 Quittner, Alexandra L.
A1 Werner, Claudius
A1 Rosthøj, Susanne
A1 Sørensen, Anne L.
A1 Nielsen, Kim G.
YR 2019
UL http://erj.ersjournals.com/content/54/suppl_63/RCT5102.abstract
AB 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 (</≥ 40 kg) or placebo, 3 times a week for 6 months.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.FootnotesCite this article as: European Respiratory Journal 2019; 54: Suppl. 63, RCT5102.This is an ERS International Congress abstract. No full-text version is available. Further material to accompany this abstract may be available at www.ers-education.org (ERS member access only).