Follow-up of a cohort of patients with cystic fibrosis in therapy with Elexacaftor-Tezacaftor-Ivacaftor

<bold>Background:</bold> Elexacaftor-Tezacaftor-Ivacaftor (ELX/TEZ/IVA) has been tested in clinical trials and it is highly effective for patients with cystic fibrosis (CF) with at least one F508del allele, with improvements in sweat chloride, pulmonary function and nutrition. <bold>Objective:</bold> The aim of the study is to establish the efficacy of ELX/TEZ/IVA in a real-life setting. <bold>Methods:</bold> A prospective, observational study was conducted in the regional center of Friuli-Venezia-Giulia (Italy). 61 patients were enrolled. They were assessed 6 and 12 months after starting the therapy. 14 patients had a period of follow-up of 24 months. Outcomes included changes in percent predicted FEV1 (ppFEV1), sweat chloride concentration [Cl-], body mass index (BMI), quality of life, glycated hemoglobin (HbA1c), Oral Glucose Tolerance Test (OGTT) and Erythrocyte sedimentation rate (ESR) and C-reactive protein (CRP). Comparations were made in patients >12 years with 6-11 years and in patients previously treated with Lumacaftor-Ivacaftor with patients who were not. <bold>Results:</bold> Average age was 18 years. At 6 months ppFEV1 improved by 21 points and it was stable at 12 and 24 months. Significative improvements were observed in [Cl-], BMI, ESR, CRP, quality of life and number of pulmonary exacerbations. The changes were greater in patients not previously treated with modulators and in patients older than 12 years but they were substantial in all groups. No significant changes were observed in OGTT and HbA1c. <bold>Conclusions:</bold> Treatment with ELX/TEZ/IVA effectively improved lung function, BMI, sweat chloride concentration, markers of systemic inflammation and number of pulmonary exacerbations in real-life clinical practice.