Suchergebnisse

Background: Use of maintenance antibiotic therapy with the macrolide azithromycin is increasing in different chronic respiratory disorders including primary ciliary dyskinesia (PCD). However, evidence for its efficacy in PCD is lacking. We aimed to determine efficacy and safety of azithromycin maintenance therapy for 6 months in patients with PCD. Methods: The BESTCILIA (Better Experimental Screening and Treatment for Primary Ciliary Dyskinesia) trial was a multi-centre, double-blind, parallel group, randomised, placebo-controlled phase 3 trial at 6 European PCD clinics at tertiary paediatric level and university hospitals (Denmark, Germany, Netherlands, Switzerland, and United Kingdom). Patients with a confirmed diagnosis of PCD, aged 7-50 years, and FEV1 >40% predicted, were recruited. Participants were randomly assigned (1:1), stratified by age and study site, via a web-based randomisation system to azithromycin 250 or 500 mg as tablets according to body weight (

Data on primary ciliary dyskinesia (PCD) epidemiology is scarce and published studies are characterised by low numbers. In the framework of the European Union project BESTCILIA we aimed to combine all available datasets in a retrospective international PCD cohort (iPCD Cohort). We identified eligible datasets by performing a systematic review of published studies containing clinical information on PCD, and by contacting members of past and current European Respiratory Society Task Forces on PCD. We compared the contents of the datasets, clarified definitions and pooled them in a standardised format. As of April 2016 the iPCD Cohort includes data on 3013 patients from 18 countries. It includes data on diagnostic evaluations, symptoms, lung function, growth and treatments. Longitudinal data are currently available for 542 patients. The extent of clinical details per patient varies between centres. More than 50% of patients have a definite PCD diagnosis based on recent guidelines. Children aged 10–19 years are the largest age group, followed by younger children (≤9 years) and young adults (20–29 years). This is the largest observational PCD dataset available to date. It will allow us to answer pertinent questions on clinical phenotype, disease severity, prognosis and effect of treatments, and to investigate genotype–phenotype correlations.