Effect of lumacaftor-ivacaftor on mucociliary clearance and clinical outcomes in cystic fibrosis: Results from the PROSPECT MCC sub-study.
Adolescent
Adult
Aminophenols
/ therapeutic use
Aminopyridines
/ therapeutic use
Benzodioxoles
/ therapeutic use
Child
Chloride Channel Agonists
/ therapeutic use
Cohort Studies
Cystic Fibrosis
/ drug therapy
Cystic Fibrosis Transmembrane Conductance Regulator
/ therapeutic use
Drug Combinations
Female
Humans
Longitudinal Studies
Male
Middle Aged
Mucociliary Clearance
/ drug effects
Prospective Studies
Quinolones
/ therapeutic use
Young Adult
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:
01 2022
01 2022
Historique:
received:
05
03
2021
revised:
03
05
2021
accepted:
04
05
2021
pubmed:
5
6
2021
medline:
3
3
2022
entrez:
4
6
2021
Statut:
ppublish
Résumé
CFTR function is required for normal mucociliary clearance (MCC) and cough-assisted clearance (CC). Lumacaftor-ivacaftor is approved for use in people with cystic fibrosis (CF) carrying two copies of F508del-CFTR. In this observational study performed at four study sites, we characterized the effect of lumacaftor-ivacaftor on mucociliary and cough clearance and related this to other clinical and research endpoints after one month of treatment. Twenty-five adolescents and adults were enrolled. No effect on whole lung MCC was observed, but CC was significantly increased. Sweat chloride improved by 18 mEq/L in this group, indicating a modest restoration of CFTR activity, but no demonstrable change in FEV
Identifiants
pubmed: 34083123
pii: S1569-1993(21)00131-4
doi: 10.1016/j.jcf.2021.05.004
pmc: PMC8630086
mid: NIHMS1706192
pii:
doi:
Substances chimiques
Aminophenols
0
Aminopyridines
0
Benzodioxoles
0
Chloride Channel Agonists
0
Drug Combinations
0
Quinolones
0
lumacaftor, ivacaftor drug combination
0
Cystic Fibrosis Transmembrane Conductance Regulator
126880-72-6
Types de publication
Journal Article
Multicenter Study
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
143-145Subventions
Organisme : NIDDK NIH HHS
ID : P30 DK065988
Pays : United States
Organisme : NIDDK NIH HHS
ID : P30 DK072482
Pays : United States
Informations de copyright
Copyright © 2021 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.
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