Pharmaceuticals · 6 authors, 5 centres
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This is a narrative review summarizing clinical studies, observational data, and case reports on the triple combination therapy elexacaftor-tezacaftor-ivacaftor (ETI) for cystic fibrosis. The central result is that ETI is a life-changing therapy for the majority of people with CF, demonstrating safety and efficacy in reducing multiple disease manifestations.
This narrative review discusses the clinical outcomes of ETI therapy for cystic fibrosis based on published clinical studies, case reports, and real-world observational data. It describes ETI as a combination of the correctors elexacaftor and tezacaftor with the potentiator ivacaftor, designed to target the underlying CFTR protein defect. The review states that ETI has shown significant efficacy in reducing pulmonary and gastrointestinal manifestations, sweat chloride concentration, and exocrine pancreatic dysfunction, with benefits reported in both short- and long-term follow-up up to two years. It also notes the reporting of therapy-related adverse effects and emphasizes the need for close monitoring by a multidisciplinary team. A limitation acknowledged is that not all people with CF are responsive to ETI due to differences in CFTR mutations, and access is limited by cost and regulatory issues.
