The following is a summary of “Long-term clinical outcomes of elexacaftor/tezacaftor/ivacaftor therapy in adults with cystic fibrosis and advanced pulmonary disease,” published in the November 2023 issue of Pulmonology by Savi, et al.

The efficacy of the combination therapy of elexacaftor, tezacaftor, and ivacaftor (ELX/TEZ/IVA) in treating cystic fibrosis (CF) patients with Phe508del mutations has been established. For a study, researchers sought to assess the enduring effects of ELX/TEZ/IVA on clinical outcomes in severe CF patients.

A prospective evaluation was conducted on lung function, pulmonary exacerbation (PEx), sweat chloride concentration, body mass index (BMI), and the respiratory domain of the cystic fibrosis questionnaire-revised (CFQ-R RD) in a cohort of CF patients considered for inclusion in an ELX/TEZ/IVA compassionate program. Assessments were performed at baseline, 12, and 24 months post-initiation of modulator therapy. The number of PExs in the year before enrollment was recorded.

Thirty-six adult CF patients (median age 36.7 years; BMI 19.8 kg/m²; FEV¹ 36.5% predicted) were included. At 12 and 24 months, there was a significant increase in the percent predicted forced expiratory volume in 1 s (ppFEV¹) from baseline (+12.5%, P < 0.0001, and +13%, P < 0.0001, respectively). The median number of PExs reduced from 4.0 in the year before enrollment to 0.0 and 1.0 at 12 and 24 months, respectively (both p < 0.0001). CFQ-R RD score improved by 22.4 points (P < 0.0001) and 16.7 points (P < 0.0001) at 12 and 24 months, respectively. Sweat chloride levels decreased, and BMI significantly increased.

Long-term ELX/TEZ/IVA combination therapy significantly improves lung function, reduces PEx rates, enhances CFQ-R RD scores, decreases sweat chloride levels, and increases BMI in severe CF patients.

Source: resmedjournal.com/article/S0954-6111(23)00294-9/fulltext