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

• Laura Carrasco Hernández ^[1] ; Rosa Mª Girón Moreno ^[2] ; Mari Nieves Balaguer Cartagena ^[3] ; Adrián Peláez ^[4] ; Amparo Sole ^[3] ; Antonio Álvarez Fernández ^[4] ; Almudena Felipe Montiel ^[12] ; Casilda Olveira ^[5] ; Gabriel Olveira ^[5] ; Ainhoa Gómez Bonilla ^[13] ; Beatriz Gómez Crespo ^[13] ; Marta García Clemente ^[6] ; Marta Solís García ^[6] ; Joana Quaresma Vázquez ^[7] ; Enrique Blitz Castro ^[7] ; Jesús Rodríguez González ^[14] ; Andrea Expósito Marrero ^[14] ; Laila Diab-Cáceres ^[8] ; Cristina Ramos Hernández ^[9] ; Ester Zamarrón de Lucas ^[10] ; Concha Prados Sanchez ^[11] ; Marina Blanco Aparicio ^[11] ; Alejandro López Neyra ^[15] ; Verónica Sanz Santiago ^[15] ; Carmen Luna Paredes ^[8] ; Isabel Delgado Pecellín ^[4] ; Óscar Asensio de la Cruz ^[16] ; Esther Quintana Gallego ^[1]
  1. [1] Hospital Universitario Virgen del Rocío

     Hospital Universitario Virgen del Rocío

     Sevilla, España

     
  2. [2] Hospital Universitario de la Princesa

     Hospital Universitario de la Princesa

     Madrid, España

     
  3. [3] Hospital Universitario La Fe

     Hospital Universitario La Fe

     Valencia, España

     
  4. [4] Instituto de Salud Carlos III

     Instituto de Salud Carlos III

     Madrid, España

     
  5. [5] Hospital Regional Universitario de Málaga

     Hospital Regional Universitario de Málaga

     Málaga, España

     
  6. [6] Hospital Universitario Central de Asturias

     Hospital Universitario Central de Asturias

     Oviedo, España

     
  7. [7] Hospital Ramón y Cajal

     Hospital Ramón y Cajal

     Madrid, España

     
  8. [8] Hospital Universitario 12 de Octubre

     Hospital Universitario 12 de Octubre

     Madrid, España

     
  9. [9] Hospital Álvaro Cunqueiro

     Hospital Álvaro Cunqueiro

     Vigo, España

     
  10. [10] Hospital Universitario La Paz

      Hospital Universitario La Paz

      Madrid, España

      
  11. [11] Complexo Hospitalario Universitario da Coruña

      Complexo Hospitalario Universitario da Coruña

      A Coruña, España

      
  12. [12] Hospital Vall d’Hebron. Vall d’Hebron Institut de Recerca (VHIR), Barcelona, Spain
  13. [13] Hospital Universitario de Cruces, Barakaldo, Spain
  14. [14] Hospital Universitario Nuestra Señora de la Candelaria, Tenerife, Spain
  15. [15] Unidad de Fibrosis Quística, Hospital Universitario Infantil Niño Jesús de Madrid, Madrid, Spain
  16. [16] Unidad de Fibrosis Quística, Unidad de Neumología y Alergología Pediátrica, Hospital Universitario Parc Taulí, Sabadell, Barcelona, Spain

  Mostrar afiliaciones +
• Localización: Archivos de bronconeumología: Organo oficial de la Sociedad Española de Neumología y Cirugía Torácica SEPAR y la Asociación Latinoamericana de Tórax ( ALAT ), ISSN 0300-2896, Vol. 59, Nº. 9, 2023, págs. 556-565
• Idioma: inglés
• Texto completo no disponible (Saber más ...)
• Resumen

  • 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.