Elexacaftor-Tezacaftor-Ivacaftor as a Final Frontier in the Treatment of Cystic Fibrosis: Definition of the Clinical and Microbiological Implications in a Case-Control Study
The use of modulator drugs that target the Cystic Fibrosis transmembrane conductance regulator (CFTR) is the final frontier in the treatment of Cystic Fibrosis (CF), a genetic multiorgan disease. F508del is the most common mutation causing defective formation and function of CFTR. Elexacaftor-tezaca...
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| Format: | Article |
| Language: | English |
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MDPI AG
2022-05-01
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| Series: | Pharmaceuticals |
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| Online Access: | https://www.mdpi.com/1424-8247/15/5/606 |
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| author | Giuseppe Migliorisi Mirella Collura Francesca Ficili Tiziana Pensabene Dafne Bongiorno Antonina Collura Francesca Di Bernardo Stefania Stefani |
| author_facet | Giuseppe Migliorisi Mirella Collura Francesca Ficili Tiziana Pensabene Dafne Bongiorno Antonina Collura Francesca Di Bernardo Stefania Stefani |
| author_sort | Giuseppe Migliorisi |
| collection | DOAJ |
| description | The use of modulator drugs that target the Cystic Fibrosis transmembrane conductance regulator (CFTR) is the final frontier in the treatment of Cystic Fibrosis (CF), a genetic multiorgan disease. F508del is the most common mutation causing defective formation and function of CFTR. Elexacaftor-tezacaftor-ivacaftor is the first triple combination of CFTR modulators. Herein, we report on a one-year case-control study that involved 26 patients with at least one F508del mutation. Patients were assigned to two similar groups, and patients with the worse clinical condition received treatment with the triple combination therapy. The study aimed to define the clinical and especially microbiological implications of treatment administration. The treatment provided significant clinical benefits in terms of respiratory, pancreatic, and sweat function. After one year of therapy, airway infection rates decreased and pulmonary exacerbations were dramatically reduced. Finally, treated patients reported a surprising improvement in their quality of life. The use of triple combination therapy has become essential in most CF people carrying the F508del mutation. Although the clinical and instrumental benefits of treatment are thoroughly known, further investigations are needed to properly define its microbiological respiratory implications and establish the real advantage of life-long treatment with elexacaftor-tezacaftor-ivacaftor. |
| first_indexed | 2024-03-10T03:09:26Z |
| format | Article |
| id | doaj.art-6738a46a09504dd3a5c4018f581d6541 |
| institution | Directory Open Access Journal |
| issn | 1424-8247 |
| language | English |
| last_indexed | 2024-03-10T03:09:26Z |
| publishDate | 2022-05-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Pharmaceuticals |
| spelling | doaj.art-6738a46a09504dd3a5c4018f581d65412023-11-23T12:35:17ZengMDPI AGPharmaceuticals1424-82472022-05-0115560610.3390/ph15050606Elexacaftor-Tezacaftor-Ivacaftor as a Final Frontier in the Treatment of Cystic Fibrosis: Definition of the Clinical and Microbiological Implications in a Case-Control StudyGiuseppe Migliorisi0Mirella Collura1Francesca Ficili2Tiziana Pensabene3Dafne Bongiorno4Antonina Collura5Francesca Di Bernardo6Stefania Stefani7Unit of Clinical Microbiology, ARNAS Civico-Di Cristina-Benfratelli, 90127 Palermo, ItalyCystic Fibrosis and Respiratory Pediatric Center, Children’s Hospital G. Di Cristina, ARNAS Civico-Di Cristina-Benfratelli, 90127 Palermo, ItalyCystic Fibrosis and Respiratory Pediatric Center, Children’s Hospital G. Di Cristina, ARNAS Civico-Di Cristina-Benfratelli, 90127 Palermo, ItalyUnit of Clinical Microbiology, ARNAS Civico-Di Cristina-Benfratelli, 90127 Palermo, ItalyDepartment of Biomedical and Biotechnological Sciences, University of Catania, 95124 Catania, ItalyUnit of Clinical Microbiology, ARNAS Civico-Di Cristina-Benfratelli, 90127 Palermo, ItalyUnit of Clinical Microbiology, ARNAS Civico-Di Cristina-Benfratelli, 90127 Palermo, ItalyDepartment of Biomedical and Biotechnological Sciences, University of Catania, 95124 Catania, ItalyThe use of modulator drugs that target the Cystic Fibrosis transmembrane conductance regulator (CFTR) is the final frontier in the treatment of Cystic Fibrosis (CF), a genetic multiorgan disease. F508del is the most common mutation causing defective formation and function of CFTR. Elexacaftor-tezacaftor-ivacaftor is the first triple combination of CFTR modulators. Herein, we report on a one-year case-control study that involved 26 patients with at least one F508del mutation. Patients were assigned to two similar groups, and patients with the worse clinical condition received treatment with the triple combination therapy. The study aimed to define the clinical and especially microbiological implications of treatment administration. The treatment provided significant clinical benefits in terms of respiratory, pancreatic, and sweat function. After one year of therapy, airway infection rates decreased and pulmonary exacerbations were dramatically reduced. Finally, treated patients reported a surprising improvement in their quality of life. The use of triple combination therapy has become essential in most CF people carrying the F508del mutation. Although the clinical and instrumental benefits of treatment are thoroughly known, further investigations are needed to properly define its microbiological respiratory implications and establish the real advantage of life-long treatment with elexacaftor-tezacaftor-ivacaftor.https://www.mdpi.com/1424-8247/15/5/606Cystic Fibrosiselexacaftor-tezacaftor-ivacaftormicrobiologyairway colonization |
| spellingShingle | Giuseppe Migliorisi Mirella Collura Francesca Ficili Tiziana Pensabene Dafne Bongiorno Antonina Collura Francesca Di Bernardo Stefania Stefani Elexacaftor-Tezacaftor-Ivacaftor as a Final Frontier in the Treatment of Cystic Fibrosis: Definition of the Clinical and Microbiological Implications in a Case-Control Study Pharmaceuticals Cystic Fibrosis elexacaftor-tezacaftor-ivacaftor microbiology airway colonization |
| title | Elexacaftor-Tezacaftor-Ivacaftor as a Final Frontier in the Treatment of Cystic Fibrosis: Definition of the Clinical and Microbiological Implications in a Case-Control Study |
| title_full | Elexacaftor-Tezacaftor-Ivacaftor as a Final Frontier in the Treatment of Cystic Fibrosis: Definition of the Clinical and Microbiological Implications in a Case-Control Study |
| title_fullStr | Elexacaftor-Tezacaftor-Ivacaftor as a Final Frontier in the Treatment of Cystic Fibrosis: Definition of the Clinical and Microbiological Implications in a Case-Control Study |
| title_full_unstemmed | Elexacaftor-Tezacaftor-Ivacaftor as a Final Frontier in the Treatment of Cystic Fibrosis: Definition of the Clinical and Microbiological Implications in a Case-Control Study |
| title_short | Elexacaftor-Tezacaftor-Ivacaftor as a Final Frontier in the Treatment of Cystic Fibrosis: Definition of the Clinical and Microbiological Implications in a Case-Control Study |
| title_sort | elexacaftor tezacaftor ivacaftor as a final frontier in the treatment of cystic fibrosis definition of the clinical and microbiological implications in a case control study |
| topic | Cystic Fibrosis elexacaftor-tezacaftor-ivacaftor microbiology airway colonization |
| url | https://www.mdpi.com/1424-8247/15/5/606 |
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