Abstract
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.
Highlights
BackgroundCystic fibrosis (CF) is a chronic, progressive, autosomal recessive disease that affects approximately 35,000 people in the United States [1,2]
This study evaluated the efficacy of VX-445 (ELX) in triple combination (TC) with TEZ and IVA in subjects with CF who are homozygous for the F508del mutation (F/F)
This study evaluated the efficacy of VX-445 in triple combination with TEZ and IVA in subjects with CF who are heterozygous for F508del and a minimal function mutation (F/MF subjects)
Summary
BackgroundCystic fibrosis (CF) is a chronic, progressive, autosomal recessive disease that affects approximately 35,000 people in the United States [1,2]. The primary defect is a mutant CFTR gene causing a decrease or absence of cystic fibrosis transmembrane conductance regulator (CFTR) activity. CFTR protein is an epithelial anion transporter of chloride and bicarbonate. It regulates salt and water balance on the surface of cells and is encoded by the CFTR gene. A defect in the CFTR protein will cause pulmonary, gastrointestinal, pancreatic, and reproductive system diseases [3]. In CF, the CFTR protein is often expressed in epithelial cells' apical membranes. CFTR proteins are expressed in the airway epithelia, paranasal sinuses, pancreas, gut epithelia, biliary tree epithelia, vas deferens epithelia, and sweat duct epithelia [5]. The airway epithelia carry the highest levels of CFTR expression
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