Seguridad y efectividad del tratamiento con elexacaftor, tezacaftor e ivacaftor en adultos con fibrosis quística / Safety and effectiveness of treatment with elexacaftor, tezacaftor and ivacaftor in adults with cystic fibrosis

Introducción La fibrosis quística (FQ) es una enfermedad causada por mutaciones en el gen localizado en el cromosoma 7 que codifica la proteína reguladora de la conductancia transmembrana de la FQ. Varios ensayos han demostrado la eficacia y seguridad de la combinación ELE/TEZ/IVA en los pacientes que tienen al menos una mutación F508del. El objetivo principal del estudio fue evaluar la seguridad a los 3 y 6 meses del tratamiento con ELE/TEZ/IVA en pacientes adultos con FQ. Métodos Se trata de un estudio transversal, prospectivo y unicéntrico de vida real en el que se incluyeron pacientes adultos de la unidad multidisciplinar de FQ del Hospital Universitario Ramón y Cajal que cumplían criterios para recibir tratamiento con ELE/TEZ/IVA. Se registraron las características demográficas y clínicas de todos los pacientes. Durante el tiempo del estudio, se llevaron a cabo 3 visitas (basal, a los 3 y a los 6 meses). Se registraron los efectos secundarios y la evolución de la función hepática durante el tiempo de seguimiento. Resultados A los 3 meses del inicio del tratamiento se observó una mejoría estadísticamente significativa de la función pulmonar, el IMC, las exacerbaciones pulmonares y el nivel de energía, así como en todas las categorías del cuestionario CFQ-R excepto en el dominio digestivo. Esta mejoría se mantuvo, pero no se incrementó, a los 6 meses en todas las variables, excepto en el IMC, donde sí se observaron diferencias entre los 3 y 6 meses de tratamiento. Conclusiones En la cohorte estudiada, el tratamiento con ELE/TEZ/IVA tiene un buen perfil de seguridad y produce un mejoría precoz en la función pulmonar, el IMC, la calidad de vida y el «nivel de energía» de los pacientes adultos con FQ, que se mantiene a los 6 meses de tratamiento (AU)

Introduction Cystic 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. Methods This 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. Results 3 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. Conclusions In 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 (AU)

[Safety and effectiveness of treatment with elexacaftor, tezacaftor and ivacaftor in adults with cystic fibrosis]. / Seguridad y efectividad del tratamiento con elexacaftor, tezacaftor e ivacaftor en adultos con fibrosis quística.

INTRODUCTION: Cystic 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. METHODS: This 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. RESULTS: 3 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. CONCLUSIONS: In 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.