Resumen de Experience With Elexacaftor/Tezacaftor/Ivacaftor in Patients With Cystic Fibrosis and Advanced Disease
Laura Carrasco Hernández, Rosa María Girón Moreno, Mari Nieves Balaguer Cartagena, Adrián Peláez, Amparo Solé Jover, Antonio Álvarez Fernández, Almudena Felipe Montiel, Casilda Olveira Fuster, Gabriel Olveira Fuster, Ainhoa Gómez Bonilla, Beatriz Gómez Crespo, Marta M. García Clemente, Marta Solís García, Joana Quaresma Vázquez, Enrique Blitz Castro, Jesús Rodríguez González, Andrea Expósito Marrero, Layla Diab Cáceres, Cristina Ramos Hernández, Ester Zamarrón de Lucas, María Concepción Prados Sánchez, Marina Blanco Aparicio, Alejandro López Neyra, V. Sanz Santiago, C. Luna Paredes, Isabel Delgado Pecellín, Óscar Asensio de la Cruz, Esther Quintana Gallego
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 FEV1% 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.
