Characterizing CFTR modulated sweat chloride response across the cf population: Initial results from the CHEC-SC study

•Modulator-induced sweat chloride (SC) responses across the CF population were consistent with those reported from prior clinical studies.•Variation in SC response to a single highly effective modulator, ivacaftor, was evident across individuals with differing mutation profiles; in vivo SC responses...

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Bibliographic Details
Published in:Journal of cystic fibrosis 2023-01, Vol.22 (1), p.79-88
Main Authors: Mayer-Hamblett, N, Zemanick, ET, Odem-Davis, K, VanDevanter, D, Warden, M, Rowe, SM, Young, J, Konstan, MW, for-the-CHEC-SC-Study-Group
Format: Article
Language:English
Subjects:
Aminophenols - therapeutic use
Benzodioxoles - therapeutic use
CFTR function
CFTR modulators
Chloride Channel Agonists - therapeutic use
Chlorides
Cystic Fibrosis
Cystic Fibrosis Transmembrane Conductance Regulator - genetics
Drug Combinations
Epidemiology
Humans
Mutation
Sweat
Sweat chloride
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Summary:•Modulator-induced sweat chloride (SC) responses across the CF population were consistent with those reported from prior clinical studies.•Variation in SC response to a single highly effective modulator, ivacaftor, was evident across individuals with differing mutation profiles; in vivo SC responses correlated with in vitro ivacaftor modulated CFTR functional responses.•With the exception of the cohort of F508del homozygous participants established on tezacaftor/ivacaftor, there were notable shifts in the proportion of participants with SC levels
ISSN:1569-1993
1873-5010
DOI:10.1016/j.jcf.2022.07.008