Outcome of elexacaftor/tezacaftor/ivacaftor therapy in patients with cystic fibrosis and solid organ transplantation

• Published in: Pediatric pulmonology Vol. 58; no. 2; pp. 602 - 605
• Main Authors: Ørum, Matilde B., Rönsholt, Frederikke F., Jeppesen, Majbritt, Bendstrup, Elisabeth, Katzenstein, Terese L., Ott, Peter, Perch, Michael, Pressler, Tacjana, Qvist, Tavs, Jensen‐Fangel, Søren
• Format: Journal Article
• Language: English
• Published: United States Wiley Subscription Services, Inc 01-02-2023
• Subjects: Aminophenols; Benzodioxoles - adverse effects; Case reports; CFTR therapy; Chloride Channel Agonists; Cystic fibrosis; Cystic Fibrosis - drug therapy; Cystic Fibrosis - genetics; Cystic Fibrosis Transmembrane Conductance Regulator - genetics; Cystic Fibrosis Transmembrane Conductance Regulator - therapeutic use; cystic fibrosis transmembrane conductance regulator therapy; Double-Blind Method; Drug Combinations; Drug therapy; ETI; Humans; Mutation; Organ Transplantation; Retrospective Studies; solid organ transplantation; Transplants & implants; solid organ transplantation; CFTR therapy; ETI; cystic fibrosis transmembrane conductance regulator therapy; cystic fibrosis
• ISSN: 8755-6863; 1099-0496
• DOI: 10.1002/ppul.26217

We report a case series of four patients with cystic fibrosis (CF) and previous solid organ transplantation (SOT) receiving elexacaftor/tezacaftor/ivacaftor therapy for 6 months or more. Data was collected retrospectively. The treatment was well tolerated and all patients reported subjective improvements.