Effect of lumacaftor-ivacaftor on mucociliary clearance and clinical outcomes in cystic fibrosis: Results from the PROSPECT MCC sub-study

Autor: John P. Clancy, Agathe Ceppe, Jihong Wu, Timothy E. Corcoran, Pradeep Bhambhvani, Peter J. Mogayzel, Beth L. Laube, Scott D. Sagel, Felix Ratjen, Joseph M. Pilewski, Steven M. Rowe, William D. Bennett, Scott H. Donaldson
Jazyk: angličtina
Rok vydání: 2021
Předmět:
0301 basic medicine
Pulmonary and Respiratory Medicine
Adult
Male
medicine.medical_specialty
Adolescent
Cystic Fibrosis
Mucociliary clearance
Sweat chloride
Aminopyridines
Cystic Fibrosis Transmembrane Conductance Regulator
Lung Clearance Index
Quinolones
Aminophenols
Cystic fibrosis
Gastroenterology
Article
Ivacaftor
Cohort Studies
03 medical and health sciences
chemistry.chemical_compound
Young Adult
0302 clinical medicine
Internal medicine
medicine
Humans
Benzodioxoles
Longitudinal Studies
Prospective Studies
Child
Chloride Channel Agonists
Lung
business.industry
Lumacaftor
respiratory system
Middle Aged
medicine.disease
respiratory tract diseases
Drug Combinations
030104 developmental biology
medicine.anatomical_structure
030228 respiratory system
chemistry
Mucociliary Clearance
Pediatrics
Perinatology and Child Health
Female
business
medicine.drug
Zdroj: J Cyst Fibros
Popis: 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 FEV1 or lung clearance index was observed. We speculate that the modest effect of lumacaftor-ivacaftor on CFTR function was insufficient to yield an improvement in MCC.
Databáze: OpenAIRE
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