Elexacaftor/tezacaftor/ivacaftor as rescue therapy in a patient with the cystic fibrosis genotype <i>F508DEL</i>/<i>G1244E</i>

Salvatore, Donatello; Colangelo, Carmela; D’Andria, Michele; Marsicovetere, Giovanni; Passarella, Domenica

doi:10.1002/ccr3.4713

Cited by 1 publication

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References 16 publications

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“…ETI therapy was also associated with a significant reduction in the incidence of pulmonary exacerbations, the need for oral and intravenous antibiotic therapy, and an improvement in the 6-min walking test distance. Similar findings were also reported in a case report of a 50-year-old woman with CF (F508del/G1244E genotype) [ 48 ]. Another case reported the ability of ETI in combating persistent infection in a 24-year-old woman with CF (F508del/R1066C genotype) and advanced lung disease [ 49 ].…”

Section: Clinical Outcomes Of Eti Therapy In Case Reports Observation...supporting

confidence: 90%

Elexacaftor-Tezacaftor-Ivacaftor: A Life-Changing Triple Combination of CFTR Modulator Drugs for Cystic Fibrosis

et al. 2023

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Cystic fibrosis (CF) is a potentially fatal monogenic disease that causes a progressive multisystemic pathology. Over the last decade, the introduction of CF transmembrane conductance regulator (CFTR) modulator drugs into clinical practice has profoundly modified the lives of many people with CF (PwCF) by targeting the fundamental cause of the disease. These drugs consist of the potentiator ivacaftor (VX-770) and the correctors lumacaftor (VX-809), tezacaftor (VX-661), and elexacaftor (VX-445). In particular, the triple combination of CFTR modulators composed of elexacaftor, tezacaftor, and ivacaftor (ETI) represents a life-changing therapy for the majority of PwCF worldwide. A growing number of clinical studies have demonstrated the safety and efficacy of ETI therapy in both short- and long-term (up to two years of follow-up to date) and its ability to significantly reduce pulmonary and gastrointestinal manifestations, sweat chloride concentration, exocrine pancreatic dysfunction, and infertility/subfertility, among other disease signs and symptoms. Nevertheless, ETI therapy-related adverse effects have also been reported, and close monitoring by a multidisciplinary healthcare team remains vital. This review aims to address and discuss the major therapeutic benefits and adverse effects reported by the clinical use of ETI therapy for PwCF.

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Section: Clinical Outcomes Of Eti Therapy In Case Reports Observation...supporting

confidence: 90%

Elexacaftor-Tezacaftor-Ivacaftor: A Life-Changing Triple Combination of CFTR Modulator Drugs for Cystic Fibrosis

et al. 2023

View full text Add to dashboard Cite

show abstract

Elexacaftor/tezacaftor/ivacaftor as rescue therapy in a patient with the cystic fibrosis genotype F508DEL/G1244E

Cited by 1 publication

References 16 publications

Elexacaftor-Tezacaftor-Ivacaftor: A Life-Changing Triple Combination of CFTR Modulator Drugs for Cystic Fibrosis

Elexacaftor-Tezacaftor-Ivacaftor: A Life-Changing Triple Combination of CFTR Modulator Drugs for Cystic Fibrosis

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