Experience With Elexacaftor/Tezacaftor/Ivacaftor in Patients With Cystic Fibrosis and Advanced Disease.
Обработка статьи · этапы, ошибки и управление
Ошибки и ограничения
История · было → стало
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Carrasco Hernández Laura, Girón Moreno Rosa Mª, Balaguer Cartagena Mari Nieves, Peláez Adrián, Sole Amparo, Álvarez Fernández Antonio, Felipe Montiel Almudena, Olveira Casilda, Olveira Gabriel, Gómez Bonilla Ainhoa, Gómez Crespo Beatriz, García Clemente Marta, Solís García Marta, Quaresma Vázquez Joana, Blitz Castro Enrique, Rodríguez González Jesús, Expósito Marrero Andrea, Diab-Cáceres Laila, Ramos Hernández Cristina, Zamarrón de Lucas Ester, Prados Sanchez Concha, Blanco Aparicio Marina, López Neyra Alejandro, Sanz Santiago Verónica, Luna Paredes Carmen, Delgado Pecellín Isabel, Asensio de la Cruz Óscar, Quintana Gallego Esther
Аннотация
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.