Decreased Pulmonary Exacerbations and Lower IV Antibiotic Usage Following Vanzacaftor/Tezacaftor/Deutivacaftor Treatment in People with Cystic Fibrosis with <i>F508del</i> -Minimal Function Genotypes

<title>Extract</title> Cystic fibrosis (CF) is multisystemic genetic disease caused by mutations in the CF transmembrane conductance regulator (<italic>CFTR</italic>) gene that result in reduced or no CFTR protein function [1–3]. Clinically, people with CF can have respiratory, pancreatic, hepatic, sweat gland, and gastrointestinal dysfunction [3, 4]. A key diagnostic test for CF is measurement of chloride concentration in sweat, which measures CFTR function. Importantly, reductions in sweat chloride concentration (which indicate improvements in CFTR function) have been demonstrated to correlate with improvements in clinical outcomes, including reductions in pulmonary exacerbation (PEx) rate and improvements in percent predicted FEV<sub>1</sub> (ppFEV<sub>1</sub>) rate of change over time, in clinical studies of CFTR modulators [5].

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Publication Details

Journal
European Respiratory Journal
Published
2026-04-23
DOI
https://doi.org/10.1183/13993003.02538-2025
Primary Topic
Cystic Fibrosis Research Advances
Type
article
Field-Weighted Citation Impact
0.00

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article

Decreased Pulmonary Exacerbations and Lower IV Antibiotic Usage Following Vanzacaftor/Tezacaftor/Deutivacaftor Treatment in People with Cystic Fibrosis with F508del -Minimal Function Genotypes

Claire L. Keating, Teja Thorat, Marcus Alexander Mall, Nitin Nair et al.
European Respiratory Journal
Cystic Fibrosis Research Advances
article

Decreased Pulmonary Exacerbations and Lower IV Antibiotic Usage Following Vanzacaftor/Tezacaftor/Deutivacaftor Treatment in People with Cystic Fibrosis with F508del -Minimal Function Genotypes

Claire L. Keating, Teja Thorat, Marcus Alexander Mall, Nitin Nair, Emilio Arteaga‐Solis, Alexander Horsley, Bonnie Ramsey, Patrick Sosnay
article en

Abstract

<title>Extract</title> Cystic fibrosis (CF) is multisystemic genetic disease caused by mutations in the CF transmembrane conductance regulator (<italic>CFTR</italic>) gene that result in reduced or no CFTR protein function [1–3]. Clinically, people with CF can have respiratory, pancreatic, hepatic, sweat gland, and gastrointestinal dysfunction [3, 4]. A key diagnostic test for CF is measurement of chloride concentration in sweat, which measures CFTR function. Importantly, reductions in sweat chloride concentration (which indicate improvements in CFTR function) have been demonstrated to correlate with improvements in clinical outcomes, including reductions in pulmonary exacerbation (PEx) rate and improvements in percent predicted FEV<sub>1</sub> (ppFEV<sub>1</sub>) rate of change over time, in clinical studies of CFTR modulators [5].

European Respiratory Journal
Seattle Children's Hospital (US), Columbia University Irving Medical Center (US), University of Manchester (GB), German Center for Lung Research (DE), Vertex Pharmaceuticals (United States) (US), Charité - Universitätsmedizin Berlin (DE)
Vertex Pharmaceuticals
Good health and well-being
Openalex Percentile: Top 15%
Cystic Fibrosis Research Advances
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Decreased Pulmonary Exacerbations and Lower IV Antibiotic Usage Following Vanzacaftor/Tezacaftor/Deutivacaftor Treatment in People with Cystic Fibrosis with <i>F508del</i> -Minimal Function Genotypes — Claire L. Keating, Teja Thorat, et al. · European Respiratory Journal (2026) | TGRS Research Map | TGRS