Chest
CommentaryChange in Sweat Chloride as a Clinical End Point in Cystic Fibrosis Clinical Trials: The Ivacaftor Experience
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Acknowledgments
Financial/nonfinancial disclosures: The authors have reported to CHEST that no potential conflicts of interest exist with any companies/organizations whose products or services may be discussed in this article.
Other contributions: The views expressed in this commentary are those of the authors and do not necessarily reflect the views or policies of the US Food and Drug Administration.
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2023, Journal of Cystic FibrosisCitation Excerpt :The relationship between modulator-induced SC response and clinical outcomes has proven elusive. There have been clear genotype-level [26] but limited patient-level, associations observed between SC concentration and lung function changes, in particular within the setting of recent clinical studies of ivacaftor ([27,28]) and lumacaftor/ivacaftor [29]. More recently, a modest correlation between SC response and short term lung function change was observed among a cohort initiating ETI, suggesting that large effect sizes combined with heterogeneity as defined by factors including CFTR genotype use may prove useful in elucidating such an association [13].
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