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- Title
Multisystemic Effects of Elexacaftor–Tezacaftor–Ivacaftor in Adults with Cystic Fibrosis and Advanced Lung Disease.
- Authors
Burgel, Pierre-Régis; Paillasseur, Jean-Louis; Durieu, Isabelle; Reynaud-Gaubert, Martine; Hamidfar, Rebecca; Murris-Espin, Marlène; Danner-Boucher, Isabelle; Chiron, Raphaël; Leroy, Sylvie; Douvry, Benoit; Grenet, Dominique; Mely, Laurent; Ramel, Sophie; Montcouquiol, Sylvie; Burnet, Espérie; Ouaalaya, El Hassane; Sogni, Philippe; Da Silva, Jennifer; Martin, Clémence
- Abstract
Rationale: Limited data exist on the safety and effectiveness of elexacaftor-tezacaftor-ivacaftor (ETI) in people with cystic fibrosis (pwCF) and advanced lung disease. Objectives: To evaluate the effects of ETI in an unselected population of pwCF and advanced lung disease. Methods: A prospective observational study, including all adults aged 18 years and older with percentage predicted forced expiratory volume in 1 second (ppFEV1) ⩽ 40 who initiated ETI from December 2019 to June 2021 in France, was conducted. PwCF were followed until August 8, 2022. Results: ETI was initiated in 434 pwCF with a median ppFEV1 of 30 (interquartile range, 25–35), including 27 with severe cystic fibrosis liver disease and 183 with diabetes. PwCF were followed for a median of 587 (interquartile range, 396–728) days after ETI initiation. Discontinuation of ETI occurred in 12 (2.8%) pwCF and was due mostly to lung transplantation (n = 5) or death (n = 4). Absolute increase in ppFEV1 by a mean of +14.2% (95% confidence interval, 13.1–15.4%) occurred at 1 month and persisted throughout the study. Increase in ppFEV1 in the youngest age quartile was almost twice that of the oldest quartile (P < 0.001); body mass index < 18.5 kg/m2 was found in 38.6% at initiation versus 11.3% at 12 months (P = 0.0001). Increases in serum concentrations of vitamins A and E, but not 25-hydroxy vitamin D3, were observed. Significant reductions in the percentages of pwCF using oxygen therapy, noninvasive ventilation, nutritional support, and inhaled and systemic therapies (including antibiotics) were observed; insulin was discontinued in 12% of patients with diabetes. Conclusions: ETI is safe in pwCF and advanced lung disease, with multisystem pulmonary and extrapulmonary benefits.
- Subjects
FRANCE; LUNGS; CYSTIC fibrosis; LUNG diseases; PULMONARY fibrosis; HEPATIC fibrosis; FORCED expiratory volume
- Publication
Annals of the American Thoracic Society, 2024, Vol 21, Issue 7, p1053
- ISSN
2329-6933
- Publication type
Article
- DOI
10.1513/AnnalsATS.202312-1065OC