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

Enrique Blitz Castro, Esther Quintana Gallego, Óscar Asensio de la Cruz, Isabel Delgado Pecellín, Carmen Luna Paredes, Verónica Sanz Santiago, Alejandro López Neyra, Marina Blanco Aparicio, Concha Prados Sanchez, Ester Zamarrón de Lucas, Cristina Ramos Hernández, Laila Diab-Cáceres, Andrea Expósito Marrero, Jesús Rodríguez González, Laura Carrasco Hernández, Joana Quaresma Vázquez, Marta Solís García, Marta García Clemente, Beatriz Gómez Crespo, Ainhoa Gómez Bonilla, Gabriel Olveira, Casilda Olveira, Almudena Felipe Montiel, Antonio Álvarez Fernández, Amparo Sole, Adrián Peláez, Mari Nieves Balaguer Cartagena, Rosa Mª Girón Moreno

Journal: Archivos de bronconeumologia 2023;59(9):556-565

PMID: 37400317

Full Text on Ediciones Doyma, S.L. View on PubMed

Abstract

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% 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.

Address: 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.; Hospital Universitario de La Princesa, Madrid, Spain.; Hospital Universitario La Fe, Valencia, Spain.; 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: [email protected].; 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.; Hospital Vall d'Hebron. Vall d'Hebron Institut de Recerca (VHIR), Barcelona, Spain.; 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.; 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.; Hospital Universitario de Cruces, Barakaldo, Spain.; Hospital Universitario Central de Asturias, Instituto de Investigación del Principado de Asturias (ISPA), Asturias, Spain.; Hospital Universitario Ramón y Cajal, Madrid, Spain.; Hospital Universitario Nuestra Señora de la Candelaria, Tenerife, Spain.; Hospital Universitario 12 de Octubre. Madrid, Spain.; Hospital Álvaro Cunqueiro, Vigo, Spain.; Hospital Universitario La Paz. Instituto de Investigación Idipaz, Madrid, Spain.; Hospital Universitario A Coruña, A Coruña, Spain.; Unidad de Fibrosis Quística, Hospital Universitario Infantil Niño Jesús de Madrid, Madrid, Spain.; 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.; Unidad de Fibrosis Quística, Unidad de Neumología y Alergología Pediátrica, Hospital Universitario Parc Taulí, Sabadell, Barcelona, Spain.

DOI: 10.1016/j.arbres.2023.05.017

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MeSH Terms: Adult, Humans, Young Adult, Aminophenols, Cystic Fibrosis, Cystic Fibrosis Transmembrane Conductance Regulator, Mutation, Quality of Life