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

Extract Cystic fibrosis (CF) is multisystemic genetic disease caused by mutations in the CF transmembrane conductance regulator (CFTR) 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 FEV₁ (ppFEV₁) rate of change over time, in clinical studies of CFTR modulators [5].