Journal
JOURNAL OF CYSTIC FIBROSIS
Volume 13, Issue 2, Pages 139-147Publisher
ELSEVIER SCIENCE BV
DOI: 10.1016/j.jcf.2013.09.007
Keywords
Cystic fibrosis; Nasal potential difference; Variance; Sweat test
Categories
Funding
- Vertex Pharmaceuticals
- Vertex Pharmaceuticals Incorporated
- Cystic Fibrosis Foundation Therapeutics, Inc.
- FDA Office of Orphan Products Development [FD-R-003432-01]
- NCRR/NIEI [CTSA UL 1RR014780]
- CFF [HOCH13B0]
- NHLBI/NIH
- NIDDK/NIH [P30-DK089507]
- ITHS NIH/NCATS [2UL1TR000423]
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Background: We examined data from a Phase 2 trial {NCT00457821} of ivacaftor, a CFTR potentiator, in cystic fibrosis (CF) patients with a G551D mutation to evaluate standardized approaches to sweat chloride measurement and to explore the use of sweat chloride and nasal potential difference (NPD) to estimate CFTR activity. Methods: Sweat chloride and NPD were secondary endpoints in this placebo-controlled, Multicenter trial. Standardization of sweat collection, processing, and analysis was employed for the first time. Sweat chloride and chloride ion transport (NPD) were integrated into a model of CFTR activity. Results: Within-patient sweat chloride determinations showed sufficient precision to detect differences between dose-groups and assess ivacaftor treatment effects. Analysis of changes in sweat chloride and NPD demonstrated that patients treated with ivacaftor achieved CFTR activity equivalent to approximately 35%-40% of normal. Conclusions: Sweat chloride is useful in multicenter trials as a biomarker of CFTR activity and to test the effect of CFTR potentiators. (C) 2013 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.
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