Age-specific efficacy and tolerability of the CFTR modulator elexacaftor/tezacaftor/ivacaftor in children with cystic fibrosis

Cystic fibrosis (CF) is an autosomal recessive disease resulting from mutations in the CF transmembrane conduction regulator (CFTR) gene. Elexacaftor/tezacaftor/ivacaftor is a highly effective combination drug designed to eliminate the underlying cause of CF in patients with at least one variant of F508del in the genotype. Comparative studies of efficacy and safety in different age groups make it possible to determine the optimal timing of initiation and benefits of targeted therapy for certain age groups. The purpose of the study: to study the efficacy and safety of elexacaftor/tezacaftor/ivacaftor therapy in two age groups. Materials and methods. The data was studied from the Russian Registry of patients with CF aged 6–18 years. The patients were divided into 2 groups: 6–12 years old and 12–18 years old, comparable in genotypes. The dynamics of nutritional status, spirometry, sweat test (ST), blood biochemical parameters, blood pressure and adverse reactions were evaluated 12 months after the start of treatment. Results. In both groups, statistically significant improvements in body mass index, spirometry, and ST were obtained (the average indicators shifted to the zone of borderline values). In the group of 6–12 years, more children achieved borderline and normal ST results (83% vs. 62%). Adverse reactions was isolated and mostly temporary in both groups, with some predominance in the older age group after a year.