Experience With Elexacaftor/Tezacaftor/Ivacaftor in Patients With Cystic Fibrosis and Advanced Disease.

Autor:	Carrasco Hernández L; Unidad Médico-Quirúrgica de Enfermedades Respiratorias, Hospital Universitario Virgen del Rocío, Sevilla, Spain; CIBER de Enfermedades Respiratorias (CIBERES), Instituto de Salud Carlos III, Madrid, Spain., Girón Moreno RM; Hospital Universitario de La Princesa, Madrid, Spain., Balaguer Cartagena MN; Hospital Universitario La Fe, Valencia, Spain., Peláez A; CIBER de Enfermedades Respiratorias (CIBERES), Instituto de Salud Carlos III, Madrid, Spain; Hospital Universitario de La Princesa, Madrid, Spain; Facultad de Medicina, Universidad Autónoma de Madrid, Madrid, Spain. Electronic address: apl00028@gmail.com., Sole A; Hospital Universitario La Fe, Valencia, Spain., Álvarez Fernández A; CIBER de Enfermedades Respiratorias (CIBERES), Instituto de Salud Carlos III, Madrid, Spain; Hospital Vall d'Hebron. Vall d'Hebron Institut de Recerca (VHIR), Barcelona, Spain., Felipe Montiel A; Hospital Vall d'Hebron. Vall d'Hebron Institut de Recerca (VHIR), Barcelona, Spain., Olveira C; Unidad de Fibrosis Quística y Bronquiectasias, Servicio de Neumología, Hospital Regional Universitario de Málaga, Málaga, Spain; Instituto de Investigación Biomédica de Málaga-Plataforma BIONAND, Departamento de Medicina y Dermatología, Universidad de Málaga, Málaga, Spain., Olveira G; Instituto de Investigación Biomédica de Málaga-Plataforma BIONAND, Departamento de Medicina y Dermatología, Universidad de Málaga, Málaga, Spain; Servicio de Endocrinología y Nutrición, Hospital Regional Universitario de Málaga, CIBER de Diabetes y Enfermedades Metabólicas Asociadas, Instituto de Salud Carlos III, Málaga, Spain., Gómez Bonilla A; Hospital Universitario de Cruces, Barakaldo, Spain., Gómez Crespo B; Hospital Universitario de Cruces, Barakaldo, Spain., García Clemente M; Hospital Universitario Central de Asturias, Instituto de Investigación del Principado de Asturias (ISPA), Asturias, Spain., Solís García M; Hospital Universitario Central de Asturias, Instituto de Investigación del Principado de Asturias (ISPA), Asturias, Spain., Quaresma Vázquez J; Hospital Universitario Ramón y Cajal, Madrid, Spain., Blitz Castro E; Hospital Universitario Ramón y Cajal, Madrid, Spain., Rodríguez González J; Hospital Universitario Nuestra Señora de la Candelaria, Tenerife, Spain., Expósito Marrero A; Hospital Universitario Nuestra Señora de la Candelaria, Tenerife, Spain., Diab-Cáceres L; Hospital Universitario 12 de Octubre. Madrid, Spain., Ramos Hernández C; Hospital Álvaro Cunqueiro, Vigo, Spain., Zamarrón de Lucas E; Hospital Universitario La Paz. Instituto de Investigación Idipaz, Madrid, Spain., Prados Sanchez C; Hospital Universitario La Paz. Instituto de Investigación Idipaz, Madrid, Spain., Blanco Aparicio M; Hospital Universitario A Coruña, A Coruña, Spain., López Neyra A; Unidad de Fibrosis Quística, Hospital Universitario Infantil Niño Jesús de Madrid, Madrid, Spain., Sanz Santiago V; Unidad de Fibrosis Quística, Hospital Universitario Infantil Niño Jesús de Madrid, Madrid, Spain., Luna Paredes C; Hospital Universitario 12 de Octubre. Madrid, Spain., Delgado Pecellín I; CIBER de Enfermedades Respiratorias (CIBERES), Instituto de Salud Carlos III, Madrid, Spain; Unidad de Neumología y Alergología Pediátrica, Hospital Universitario Virgen del Rocío, Universidad de Sevilla, Sevilla, Spain., Asensio de la Cruz Ó; Unidad de Fibrosis Quística, Unidad de Neumología y Alergología Pediátrica, Hospital Universitario Parc Taulí, Sabadell, Barcelona, Spain., Quintana Gallego E; Unidad Médico-Quirúrgica de Enfermedades Respiratorias, Hospital Universitario Virgen del Rocío, Sevilla, Spain; CIBER de Enfermedades Respiratorias (CIBERES), Instituto de Salud Carlos III, Madrid, Spain.
Jazyk:	English; Spanish; Castilian
Zdroj:	Archivos de bronconeumologia [Arch Bronconeumol] 2023 Sep; Vol. 59 (9), pp. 556-565. Date of Electronic Publication: 2023 Jun 09.
DOI:	10.1016/j.arbres.2023.05.017
Abstrakt:	Introduction: Elexacaftor/tezacaftor/ivacaftor (ETI) was used through the early access programme in Spain from December 2019 in cystic fibrosis (CF) patients with homozygous or heterozygous F508del mutation with advanced lung disease. Methodology: Multicentre, ambispective, observational, study in which 114 patients in follow-up in 16 national CF units were recruited. Clinical data, functional tests, nutritional parameters, quality of life questionnaires, microbiological isolates, number of exacerbations, antibiotic treatments and side effects were collected. The study also compared patients with homozygous and heterozygous F508del mutations. Results: Of the 114 patients, 85 (74.6%) were heterozygous for F508del mutation, and the mean age was 32.2±9.96 years. After 30 months of treatment, lung function measured by FEV 1 % showed improvement from 37.5 to 48.6 (p<0.001), BMI increased from 20.5 to 22.3 (p<0.001), and all isolated microorganisms decreased significantly. The total number of exacerbations was also significantly reduced from 3.9 (±2.9) to 0.9 (±1.1) (p<0.001). All items in the CFQ-R questionnaire showed improvement, except for the digestive domain. Oxygen therapy use decreased by 40%, and only 20% of patients referred for lung transplantation remained on the active transplant list. ETI was well-tolerated, with only 4 patients discontinuing treatment due to hypertransaminemia. Conclusions: ETI decreases the number of exacerbations, increases lung function and nutritional parameters, decrease in all isolated microorganisms, for 30 months of treatment. There is an improvement in the CFQ-R questionnaire score except for the digestive item. It is a safe and well-tolerated drug. (Copyright © 2023 SEPAR. Published by Elsevier España, S.L.U. All rights reserved.)
Databáze:	MEDLINE
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