No evidence of germline PTEN mutations in familial prostate cancer

Airway inflammation is a key driver of cystic fibrosis (CF) lung disease. The advent of cystic fibrosis transmembrane conductance regulator (CFTR) modulators has the potential to transform the care of CF, however the direct effect of these therapies on lung inflammation is unknown. Here, we profile airway inflammation in bronchoalveolar lavage (BAL) of preschool aged children with CF on CFTR modulator therapy (ivacaftor or ivacaftor/lumacaftor), untreated children with CF, and age-matched healthy controls. We show that children treated with ivacaftor have lower pulmonary concentrations of inflammatory mediators CCL3, CXCL9, CCL2, IL-8, IL-1β, and IL-6 relative to untreated children with CF. Conversely, there was no significant effect of lumacaftor/ivacaftor treatment on airway inflammation. This is the first work to illustrate a difference in early life airway inflammation with CFTR treatment, highlights the effectiveness of ivacaftor in early life, and suggests that BAL inflammatory profile may represent a biomarker of therapeutic response to treatment.