A Review of Trikafta: Triple Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulator Therapy
| Autor: | Abdulsabar Sanni, Anas Zaher, Jude ElSaygh, Hassan ElSaygh, Dalal H Elsori |
|---|---|
| Rok vydání: | 2021 |
| Předmět: |
elexacaftor
Pulmonology trikafta Disease Bioinformatics Pediatrics orkambi Cystic fibrosis cystic fibrosis Ivacaftor chemistry.chemical_compound Internal Medicine medicine Adverse effect cystic fibrosis therapy Lung biology lumacaftor business.industry Lumacaftor General Engineering cystic fibrosis triple therapy medicine.disease Cystic fibrosis transmembrane conductance regulator Clinical trial medicine.anatomical_structure chemistry tezacaftor biology.protein ivacaftor business medicine.drug |
| Zdroj: | Cureus |
| ISSN: | 2168-8184 |
| DOI: | 10.7759/cureus.16144 |
| Popis: | Cystic fibrosis (CF) is a potentially fatal genetic disease that causes serious lung damage. With time, researchers have a more complete understanding of the molecular-biological defects that underlie CF. This knowledge is leading to alternative approaches regarding the treatment of this condition. Trikafta is the third FDA-approved drug that targets the F508del mutation of the CFTR gene. The drug is a combination of three individual drugs which are elexacaftor (ELX), tezacaftor (TEZ), and ivacaftor (IVA). This trio increases the activity of the cystic fibrosis transmembrane conductance regulator (CFTR) protein and reduces the mortality and morbidity rates in CF patients. The effectiveness of Trikafta, seen in clinical trials, outperforms currently available therapies in terms of lung function, quality of life, sweat chloride reduction, and pulmonary exacerbation reduction. The safety and efficacy of CFTR modulators in children with CF have also been studied. Continued evaluation of patient data is needed to confirm its long-term safety and efficacy. In this study, we will focus on reviewing data from clinical trials regarding the benefits of CFTR modulator therapy. We address the impact of Trikafta on lung function, pulmonary exacerbations, and quality of life. Adverse events of the different CFTR modulators are discussed. |
| Databáze: | OpenAIRE |
| Externí odkaz: |
|