Real Life With Tezacaftor and Ivacaftor in Adult Patients With Cystic Fibrosis: Spanish

Abstract

Cystic fibrosis (CF) is an autosomal recessive, multisystem genetic disease that mainly affects the exocrine glands due to the absence or alteration of a protein, called cystic fibrosis transmembrane conductance regulator (CFTR).1,2 Until a decade ago, the only treatments available tried to control or prevent the symptoms that were occurring. However, in recent years a line of treatments that improve the functionality of the altered protein has been developed, called CFTR modulators. Tezacaftor–ivacaftor (TEZ/IVA) is modulator of CFTR, indicated in a combined administration regimen for the treatment of CF patients ≥ 6 years, homozygous or heterozygous for the F508del mutation with residual function mutations.3 This drug has been available in Spain since October 1, 2019.