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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 (

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