• Medicina clinica · Oct 2023

    Safety and effectiveness of treatment with elexacaftor, tezacaftor and ivacaftor in adults with cystic fibrosis.

    • Nieto RoyoRosaRServicio de Neumología, Hospital Universitario Ramón y Cajal, Madrid, España; Departamento de Medicina, Universidad de Alcalá, Madrid, España. Electronic address: rosanr23@hotmail.com., Diego Durán Barata, Deisy Barrios Barreto, Winnifer Briceño Franquiz, and Luis Máiz Carro.
    • Servicio de Neumología, Hospital Universitario Ramón y Cajal, Madrid, España; Departamento de Medicina, Universidad de Alcalá, Madrid, España. Electronic address: rosanr23@hotmail.com.
    • Med Clin (Barc). 2023 Oct 27; 161 (8): 338341338-341.

    IntroductionCystic fibrosis (CF) is a disease caused by mutations in the gene located on chromosome 7 that encodes the CF transmembrane conductance regulator protein. Several trials have demonstrated the efficacy and safety of the ELE/TEZ/IVA combination in patients who have at least one F508del mutation. The main objective of the study was to evaluate the safety at 3 and 6 months of treatment with ELE/TEZ/IVA in adult patients with CF.MethodsThis is a real-life, prospective, single-center, cross-sectional study that included adult patients from the CF multidisciplinary unit. The demographic and clinical characteristics of all patients were recorded. During the time of the study, 3 visits were carried out (baseline, at 3 and at 6 months). Side effects were recorded during the follow-up time.Results3 months after the start of treatment, a statistically significant improvement was observed. of lung function, BMI, pulmonary exacerbations and energy level, as well as in all the categories of the CFQ-R questionnaire except in the digestive domain. This improvement was maintained, but not increased at 6 months in all variables, except BMI, where differences were observed between 3 and 6 months of treatment.ConclusionsIn the cohort studied, treatment with ELE/TEZ/IVA has a good safety profile. and produces an early improvement in lung function, BMI, quality of life and the "energy level" of adult patients with CF, which is maintained at 6 months of treatment.Copyright © 2023 Elsevier España, S.L.U. All rights reserved.

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