Early and sustained improvements of lung clearance index from two to sixteen weeks of elexacaftor/tezacaftor/ivacaftor therapy in patients with cystic fibrosis-a real world study.
ELX/TEZ/IVA
LUM/IVA
cystic fibrosis
lung clearance index (LCI)
modulator therapy
ppFEV1
real-life
ventilation inhomogeneity
Journal
Frontiers in pharmacology
ISSN: 1663-9812
Titre abrégé: Front Pharmacol
Pays: Switzerland
ID NLM: 101548923
Informations de publication
Date de publication:
2023
2023
Historique:
received:
16
12
2022
accepted:
21
02
2023
entrez:
27
3
2023
pubmed:
28
3
2023
medline:
28
3
2023
Statut:
epublish
Résumé
Since the introduction of CFTR modulator therapies, longitudinal real-life data of lung clearance index (LCI) during treatment is scarce. In this single-centre, post-approval setting, we report data of 51 patients with different stages of lung disease, age 2-52 years with repeated measurements of forced expiratory volume as a percentage of the predicted value (ppFEV₁) and LCI after 2, 4, and 16 weeks of CFTR modulator treatment and at baseline. In 25 patients during elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) treatment, significant improvements of LCI (median -1.4) and ppFEV₁ (median +8.3%) were observed after only 2 weeks, and were maintained after 4 and 16 weeks of treatment (LCI: -2.0, -2.2; ppFEV₁: +7.2%, +11.8%). We observed a significant correlation between LCI improvement at week 16 and lower baseline LCI. In 26 younger and healthier patients receiving lumacaftor/ivacaftor (LUM/IVA) treatment, no significant changes of LCI and ppFEV₁ occured. With ELX/TEZ/IVA, our data shows rapid, significant improvements of LCI and ppFEV₁ already after 2 weeks. Early LCI measurements can help to assess the patient's response to this high-cost therapy.
Identifiants
pubmed: 36969845
doi: 10.3389/fphar.2023.1125853
pii: 1125853
pmc: PMC10030732
doi:
Types de publication
Journal Article
Langues
eng
Pagination
1125853Informations de copyright
Copyright © 2023 Appelt, Steinkamp, Sieber and Ellemunter.
Déclaration de conflit d'intérêts
DA is a subinvestigator in clinical studies and has received sponsored travel to participate in conferences from Teva Pharmaceutical industries. GS reports fees for scientific work from CF TEAM Forschung, Innsbruck during the conduct of the study. HE is a site principal investigator in clinical studies and received honoraria for lectures from Vertex Pharmaceuticals. SS is employed by STAT-UP Statistical Consulting & Data Science GmbH, Munich, Germany. HE is a site principal investigator in clinical studies and received honoraria for lectures from Vertex Pharmaceuticals.
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