Introduction. Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) is a combination of three cystic fibrosis transmembrane conductance regulator (CFTR) modulating drugs that improves CFTR function in cystic fibrosis (CF) patients homozygous or heterozygous for Phe508del mutation. The aim of the study was to evaluate the response to ELX/TEZ/IVA treatment both from clinical-functional and morpho-structural point of view.
Methods. We retrospectively collected data from CF patients followed at Parma CF Centre (Italy) treated with ELX/TEZ/IVA between March and November 2021. We evaluated post-treatment changes in biometric parameters, respiratory function, quality of life, pulmonary exacerbations and lung structure by chest magnetic resonance imaging (MRI).
Results. 19 patients (32.5±10.2 years) were included in the study. After 6 months of treatment with ELX/TEZ/IVA, MRI showed significant improvements in morphological score (p <0.001), with reduction in bronchial wall thickening (p <0.001) and mucus plugging (p 0.007) (Fig. 1 and Fig. 2). Lung function showed significant improvement in predicted FEV1% (p <0.001), FVC%, FEV1/FVC and LCI2.5% (p <0.005). Moreover, a significant improvement was found in body mass index (BMI) (p <0.001), pulmonary exacerbations (p 0.018) and sweat chloride concentration (p <0.001).
Conclusions. Our study confirms the efficacy of ELX/TEZ/IVA in CF patients not only from a clinical point of view but also in terms of morphological changes of the lungs.