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Article

Real-life impact of genotype and severity of lung disease on efficacy of elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis.

Pulmonary pharmacology & therapeutics - 1 Mar 2025

Sheikh Shahid, Holtzlander Melissa, Eisner Mariah, Gushue Courtney, Palacios Sabrina, Kotha Kavitha, Imran Sehyr, McCoy Karen S

Abstract excerpt

BACKGROUND: Elexacaftor-tezacaftor-ivacaftor (ETI) therapy has shown improvement in lung function, BMI and reduction in pulmonary exacerbations but the impact of genotype and severity of lung disease on heterogeneity of ETI efficacy in real life is not known. METHODS: This is a prospective observational study. Clinical data at baseline and at one-year of therapy were compared for the total cohort and for two...

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Identifiers

PMID: 39978537
DOI: 10.1016/j.pupt.2025.102345
PMCID: PMC13309060

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