Evaluating elexacaftor/tezacaftor/ivacaftor (ETI; Trikafta™) for treatment of patients with non-cystic fibrosis bronchiectasis (NCFBE): A clinical study protocol

• Published in: PloS one Vol. 20; no. 2; p. e0316721
• Main Authors: Swenson, Colin E, Hunt, William R, Manfredi, Candela, Beltran, Diana J, Hong, Jeong S, Davis, Brian R, Suzuki, Shingo, Barillá, Cristina, Rab, Andras, Chico, Cynthia, Dangerfield, Joy, Streby, Ashleigh, Barton, Erin, Cox, Elizabeth M, Stecenko, Arlene A, Westbrook, Adrianna, Kapolka, Rebecca, Sorscher, Eric J
• Format: Journal Article
• Language: English
• Published: United States Public Library of Science 01.02.2025; Public Library of Science (PLoS)
• Subjects: Adult; Air flow; Aminophenols - administration & dosage; Aminophenols - therapeutic use; Benzodioxoles - therapeutic use; Biopsy; Bronchiectasis; Bronchiectasis - drug therapy; Bronchiectasis - genetics; Cell differentiation; Chloride; Chloride Channel Agonists - therapeutic use; Chloride conductance; Chlorides; Consent; Cough; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator - genetics; Cystic Fibrosis Transmembrane Conductance Regulator - metabolism; Diagnosis; Differentiation (biology); Drug Combinations; Female; Health services; Humans; In vitro methods and tests; Indoles - administration & dosage; Indoles - therapeutic use; Infections; Inflammation; Ion transport; Laboratories; Lung diseases; Male; Middle Aged; Monolayers; Mutation; Pathogenesis; Patients; Pyrazoles - therapeutic use; Pyridines - therapeutic use; Pyrrolidines - therapeutic use; Quality of life; Quinolines; Quinolones - therapeutic use; Respiratory tract infection; Sputum; Sweat; Toxins; Treatment Outcome; United States > US

Non-cystic fibrosis bronchiectasis (NCFBE) is a disease that exhibits dilatation of airways, airflow obstruction, persistent cough, excessive sputum production, and refractory respiratory infections. NCFBE exhibits clinical and pathological manifestations similar to key features of cystic fibrosis (...