Archivos de bronconeumología
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Arch. Bronconeumol. · Dec 2018
Multicenter Study Observational StudyCompassionate Use of Lumacaftor/Ivacaftor in Cystic Fibrosis: Spanish Experience.
The most common cystic fibrosis (CF)-causing mutation is deltaF508 (F508del), which is present in 28% of CF Spanish patients. While the literature based on real-life studies on CF patients homozygous F508del treated with lumacaftor/ivacaftor is limited, it demonstrates the need for better strategies to prevent related adverse events (AEs) as well as the development of newer drugs. ⋯ While treatment with lumacaftor/ivacaftor resulted in an improvement in the number of pulmonary severe exacerbations, no improvement in ppFEV1 or BMI was found.