Clinical response to lumacaftor-ivacaftor in patients with cystic fibrosis according to baseline lung function.
Adolescent
Adult
Aminophenols
/ therapeutic use
Aminopyridines
/ therapeutic use
Benzodioxoles
/ therapeutic use
Chloride Channel Agonists
/ therapeutic use
Cystic Fibrosis
/ drug therapy
Disease Progression
Drug Combinations
Female
France
Humans
Male
Quinolones
/ therapeutic use
Respiratory Function Tests
Journal
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society
ISSN: 1873-5010
Titre abrégé: J Cyst Fibros
Pays: Netherlands
ID NLM: 101128966
Informations de publication
Date de publication:
03 2021
03 2021
Historique:
received:
31
05
2020
revised:
04
06
2020
accepted:
04
06
2020
pubmed:
28
6
2020
medline:
22
1
2022
entrez:
28
6
2020
Statut:
ppublish
Résumé
Phase 3 trials have demonstrated the safety and efficacy of lumacaftor-ivacaftor (LUMA-IVA) in patients with cystic fibrosis (CF) homozygous for the Phe508del CFTR mutation and percent predicted forced expiratory volume in 1 s (ppFEV To evaluate the safety and effectiveness of LUMA-IVA over the first year of treatment in patients with ppFEV 827 patients were classified into 3 subgroups according to ppFEV Phe508del homozygous CF patients benefit from LUMA-IVA at all levels of baseline lung function
Sections du résumé
BACKGROUND
Phase 3 trials have demonstrated the safety and efficacy of lumacaftor-ivacaftor (LUMA-IVA) in patients with cystic fibrosis (CF) homozygous for the Phe508del CFTR mutation and percent predicted forced expiratory volume in 1 s (ppFEV
METHODS
To evaluate the safety and effectiveness of LUMA-IVA over the first year of treatment in patients with ppFEV
RESULTS
827 patients were classified into 3 subgroups according to ppFEV
CONCLUSION
Phe508del homozygous CF patients benefit from LUMA-IVA at all levels of baseline lung function
Identifiants
pubmed: 32591294
pii: S1569-1993(20)30752-9
doi: 10.1016/j.jcf.2020.06.012
pii:
doi:
Substances chimiques
Aminophenols
0
Aminopyridines
0
Benzodioxoles
0
Chloride Channel Agonists
0
Drug Combinations
0
Quinolones
0
lumacaftor, ivacaftor drug combination
0
Types de publication
Journal Article
Multicenter Study
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
220-227Informations de copyright
Copyright © 2020. Published by Elsevier B.V.
Déclaration de conflit d'intérêts
Declaration of Competing Interest All authors declare no conflicts of interest related to the work submitted for publication.