Real-World Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis: A 24-Month Italian National Registry Study

In this nationwide real-world cohort, ETI was associated with sustained improvements in lung function, nutritional status, and hospitalization burden. The attenuation of lung function gains after the first year, particularly in less severe disease, supports the need for individualized monitoring and cautious treatment de-escalation in the ETI era.

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Publication Details

Journal
Journal of Clinical Medicine
Published
2026-04-02
DOI
https://doi.org/10.3390/jcm15072699
Primary Topic
Cystic Fibrosis Research Advances
Type
article
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article

Real-World Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis: A 24-Month Italian National Registry Study

Annalisa Amato, Donatello Salvatore, Giuseppe Campagna, Rita Padoan et al.
Journal of Clinical Medicine
Cystic Fibrosis Research Advances
article

Real-World Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis: A 24-Month Italian National Registry Study

Annalisa Amato, Donatello Salvatore, Giuseppe Campagna, Rita Padoan, Angela Pepe, M. Salvatore
article en

Abstract

In this nationwide real-world cohort, ETI was associated with sustained improvements in lung function, nutritional status, and hospitalization burden. The attenuation of lung function gains after the first year, particularly in less severe disease, supports the need for individualized monitoring and cautious treatment de-escalation in the ETI era.

Journal of Clinical MedicineVol. 15(7)
Ospedale San Carlo (IT), Cystic Fibrosis Research Foundation (IT), Ludwig Boltzmann Institute for Rare and Undiagnosed Diseases (AT), Sapienza University of Rome (IT)
Zero hunger
Openalex Percentile: Top 31%
Cystic Fibrosis Research Advances
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