Efficacy and safety of azithromycin maintenance therapy in primary ciliary dyskinesia (BESTCILIA): A multicentre, double-blind, randomised, placebo-controlled phase 3 trial
The Lancet Respiratory Medicine May 11, 2020
Kobbernagel HE, Buchvald FF, Haarman EG, et al. - Researchers undertook a multicentre, double-blind, parallel group, randomised, placebo-controlled phase 3 trial, named BESTCILIA (Better Experimental Screening and Treatment for Primary Ciliary Dyskinesia), to assess the efficacy as well as the safety of azithromycin maintenance therapy for 6 months in patients having primary ciliary dyskinesia (PCD). This inquiry was carried out at 6 European PCD clinics (tertiary paediatric care centres and university hospitals in Denmark, Germany, Netherlands, Switzerland, and UK). This study included only cases with a verified diagnosis of PCD, ages between 7 to 50 years, and predicted FEV1 greater than 40%. Patients were randomized (1:1) to azithromycin 250 mg or 500 mg as tablets based on bodyweight (≥ 40 kg) or identical placebo, three times a week for 6 months. The number of respiratory exacerbations over 6 months was assessed as the primary endpoint. Findings revealed good tolerability of azithromycin maintenance therapy for 6 months, which halved the rate of respiratory exacerbations in patients in this first multinational randomised controlled trial on pharmacotherapy in PCD. For patients with PCD who experience frequent exacerbations, azithromycin maintenance therapy affords a choice potentially resulting in decreased requirement for additional antibiotic treatments and preventing irreversible lung damage.
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