Association between biomarkers of tobacco smoke exposure and clinical efficacy of ivacaftor in the G551D observational trial (GOAL)

•Urinary acrolein is increased in people with CF who are exposed to tobacco smoke.•Urinary acrolein is associated with at least 12 mmol/L less improvement in sweat chloride after 6 months of ivacaftor treatment, a deficit equivalent to the treatment effect of tezacaftor/ivacaftor in F508del homozygo...

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Bibliographic Details
Published in:Journal of cystic fibrosis 2024-09, Vol.23 (5), p.959-966
Main Authors: Baker, Elizabeth, Harris, William T., Guimbellot, Jennifer S., Bliton, Kyle, Rowe, Steven M., Raju, S. Vamsee, Oates, Gabriela R.
Format: Article
Language:English
Subjects:
CFTR modulators
Cystic fibrosis
Ivacaftor
Smoke exposure
Tobacco
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Summary:•Urinary acrolein is increased in people with CF who are exposed to tobacco smoke.•Urinary acrolein is associated with at least 12 mmol/L less improvement in sweat chloride after 6 months of ivacaftor treatment, a deficit equivalent to the treatment effect of tezacaftor/ivacaftor in F508del homozygous patients or those with residual CFTR function.•People with CF with low urinary acrolein have 2.5 times higher odds of a robust response to ivacaftor (sweat chloride
ISSN:1569-1993
1873-5010
1873-5010
DOI:10.1016/j.jcf.2024.07.010