Peripheral lung effect of elexacaftor/tezacaftor/ivacaftor in adult cystic fibrosis

Research output: Contribution to journalArticle

Abstract

Despite being an important patient group, adult cystic fibrosis patients with an FEV1 below 40%predicted have been excluded from clinical trials with elexacaftor/tezacaftor/ivacaftor. We conducted a real-life 3 months follow-up study in 14 adult CF patients (median FEV1 34%predicted) demonstrating significant treatment effects in terms of FEV1 (an increase of 12%predicted at 4 weeks, remaining stable thereafter). Corresponding decreases in lung clearance index LCI (by 31%predicted, down from baseline 247%predicted) and ventilation heterogeneity in the acinar compartment (Sacin) (by 411%predicted, down from baseline 798%predicted) suggest a distinct peripheral lung effect. One patient had intermittent treatment interruptions because of drug-induced liver injury. Our real-life data confirm that treatment with elexacaftor/tezacaftor/ivacaftor is effective in severely obstructive patients, and this is the first study to show time evolution of ventilation distribution improvement, pointing to the peripheral lung as the main site of treatment effect.

Original languageEnglish
JournalJournal of Cystic Fibrosis
DOIs
Publication statusE-pub ahead of print - 6 Apr 2021

Bibliographical note

Copyright © 2021 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.

Keywords

  • adult cystic fibrosis patients
  • Peripheral lung effect

Fingerprint Dive into the research topics of 'Peripheral lung effect of elexacaftor/tezacaftor/ivacaftor in adult cystic fibrosis'. Together they form a unique fingerprint.

Cite this