1. Real‐life efficacy and safety of elexacaftor/tezacaftor/ivacaftor on severe cystic fibrosis lung disease patients
- Author
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Renate Kos, Anne H. Neerincx, Dominic W. Fenn, Paul Brinkman, Rianne Lub, Steffie E. M. Vonk, Jolt Roukema, Monique H. Reijers, Suzanne W. J. Terheggen‐Lagro, Josje Altenburg, Christof J. Majoor, Lieuwe D. Bos, Eric G. Haarman, Anke H. Maitland‐van der Zee, and the Amsterdam Mucociliary Clearance Disease (AMCD) Research Group
- Subjects
cystic fibrosis ,elexacaftor/tezacaftor/ivacaftor ,modulators ,sputum ,Therapeutics. Pharmacology ,RM1-950 - Abstract
Abstract Elexacaftor/tezacaftor/ivacaftor (ETI) is a cystic fibrosis (CF) transmembrane conductance regulator modulator, which has shown efficacy in CF patients (≥6 years) with ≥1 Phe508del mutation and a minimal function mutation. In October 2019, ETI became available on compassionate use basis for Dutch CF patients with severe lung disease. Our objective was to investigate safety and efficacy of ETI in this patient group in a real‐life setting. A multicenter longitudinal observational study was conducted to examine changes in FEV1, BMI, and adverse events at initiation and 1, 3, 6, and 12 months after starting ETI. The number of exacerbations was recorded in the 12 months before and the 12 months after ETI treatment. Patients eligible for compassionate use had a FEV1
- Published
- 2022
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