Clinical trials in primary ciliary dyskinesia (PCD) patients that used a definition for pulmonary exacerbations

First author [ref.]#Study aimsStudy populationMethod used to develop the definitionDefinition of exacerbation
Kobbernagel [16]Protocol for randomised controlled trial to determine the efficacy and safety of azithromycin maintenance therapyPCD children aged >7 years and adultsFace-to-face discussion at BESTCILIA study meetingEither
Respiratory symptoms (not listed) leading to start of systemic antibiotic treatment, irrespective of results of bacterial culture
Decline in FEV1 % pred ≥10% relative to the FEV1 % pred at randomisation, irrespective of whether antibiotics are prescribed
Ratjen [17]Changes in airway inflammation during pulmonary exacerbationsCystic fibrosis and PCD children aged >6 yearsResearcher definedIncrease in respiratory symptoms (not listed) treated with oral antibiotics
Sunther [15]Recovery of baseline lung function after pulmonary exacerbationPCD childrenResearcher definedA change in respiratory status for which intravenous antibiotics were prescribed

FEV1: forced expiratory volume in 1 s. #: all three trials were published in 2016.