Real-life experience with a generic formulation of Elexacaftor/Tezacaftor/ Ivacaftor in patients with Cystic Fibrosis with responsive CFTR variants without previous modulators therapy

<bold>Introduction:</bold> Pivotal studies have shown that Elexacaftor/Tezacaftor/Ivacaftor (ETI) improves lung function, nutritional status and Quality of Life and reduces sweat chloride concentration and pulmonary exacerbations in patients with Cystic Fibrosis (pwCF) with responsive CFTR variants. <bold>Objective:</bold> To report real-life effects of a generic formulation of ETI (Trixacar^R, Gador Argentina) use in pwCF with responsive CFTR variants without previous modulator therapy. <bold>Patients and Methods:</bold> Prospective, cohort study conducted in pwCF 6 years or older receiving a generic formulation of ETI at the Respiratory Center of Hospital de Niños Ricardo Gutiérrez. Clinical variables: BMI, pulmonary exacerbations and CFQR (respiratory domain), pulmonary function (FEV1 and LCI_2.5) sweat chloride, and fecal elastase were analyzed before and 9 months after starting ETI therapy. <bold>Results:</bold> Twenty-four patients (12 male) with a median (IQR) age of 15 (12.5 – 16.9) years. <table-wrap><object-id>erj;64/suppl_68/PA556/TB1</object-id><object-id>T1</object-id><object-id>TB1</object-id><table><colgroup><col></col><col></col><col></col><col></col></colgroup><tbody><tr><td></td><td><bold>Baseline</bold></td><td><bold>Post Treatment</bold></td><td><bold>P</bold></td></tr><tr><td>Sweat Chloride* (mEq/L) (n= 22)</td><td>88 (75 - 98)</td><td>54 (41 - 63) (3° month)</td><td><0.001</td></tr><tr><td>BMI Z Score* (n= 24)</td><td>-0.19 (−0.66 - 0.29)</td><td>0.16 (−0.04 - 1)</td><td>0.011</td></tr><tr><td>ppFEV₁* (n=22)</td><td>62.5 (44 - 80)</td><td>78 (60 - 91)</td><td><0.001</td></tr><tr><td>LCI_2.5* (n= 8)</td><td>10.7 (9.7 - 12.7)</td><td>8.3 (6.6 - 9.9)</td><td><0.01</td></tr><tr><td>Severe exacerbations (n= 24)</td><td>0.42</td><td>0</td><td><0.01</td></tr><tr><td>CFQR* (respiratory)</td><td>56 (39 - 83)</td><td>83 (74 - 90)</td><td><0.01</td></tr></tbody></table></table-wrap> *median (IQR) <bold>Conclusions:</bold> the use of a generic formulation of ETI in pwCF with responsive CFTR variants showed a significant improvement in lung function and quality of life with a significant reduction in sweat chloride and severe pulmonary exacerbations after 9 months of treatment.