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Elexacaftor/Tezacaftor/Ivacaftor in Patients with Cystic Fibrosis Homozygous for the F508del Mutation and Advanced Lung Disease: A 48-Week Observational Study
| Content Provider | MDPI |
|---|---|
| Author | D’andria, Michele Carnovale, Vincenzo Iacotucci, Paola Terlizzi, Vito Colangelo, Carmela Ferrillo, Lorenza Pepe, Angela Francalanci, Michela Taccetti, Giovanni Buonaurio, Serena Celardo, Assunta Salvadori, Laura Marsicovetere, Giovanni Ferrara, Nicola Salvatore, Donatello |
| Copyright Year | 2022 |
| Description | Background: Elexacaftor/tezacaftor/ivacaftor (ETI) is the newest cystic fibrosis transmembrane conductance regulator (CFTR) modulator drug approved for the treatment of patients with cystic fibrosis (pwCF) aged ≥6 years with at least one copy of the F508del mutation (F) in the CFTR gene or another mutation that is responsive to treatment with ETI. This study determined the effectiveness and safety of ETI in a cohort of severely affected pwCF with an F/F genotype. Methods: Retrospective observational study in F/F pwCF treated for 48 weeks, enrolled in an ETI managed access program available to subjects with advanced lung disease $(ppFEV_{1}$ < 40). Twenty-six patients from three centres were included. The main outcomes included lung function, sweat chloride concentration (SCC), nutrition, frequency of pulmonary exacerbations (PEx), CFQ-R, and safety. Results: $ppFEV_{1}$ improved by 12.06 (95%CI 8.54, 15.57) from baseline after 4 weeks of treatment with ETI, 15.32 (11.3, 19.34) after 24 weeks, and 14.48 (10.64, 18.32) after 48 weeks. The increase in $FEV_{1}$ was accompanied by a decrease in SCC, improvement of BMI, and noticeable reduction in PEx. An overall good safety profile was observed. Conclusions: In F/F pwCF with advanced lung disease with an F/F genotype, ETI was safe and associated with clinical improvement. |
| Starting Page | 1021 |
| e-ISSN | 20770383 |
| DOI | 10.3390/jcm11041021 |
| Journal | Journal of clinical medicine |
| Issue Number | 4 |
| Volume Number | 11 |
| Language | English |
| Publisher | MDPI |
| Publisher Date | 2022-02-16 |
| Access Restriction | Open |
| Subject Keyword | Journal of clinical medicine Journal of Clinical Medicine Respiratory System Elexacaftor/tezacaftor/ivacaftor Advanced Lung Disease Cystic Fibrosis |
| Content Type | Text |
| Resource Type | Article |
