Efficacy and safety of pathogenetic therapy for cystic fibrosis in children in the Chechen Republic: Use of a generic CFTR modulator and experience of switching within a same international non-proprietary name
TL;DR
The use of both the original drug and generic Trilexa in children with CF is associated with clinical improvements in key efficacy indicators and a favorable safety profile.
Abstract
Introduction . Cystic fibrosis is a hereditary disease associated with progressive damage to the bronchopulmonary system and the risk of disability. The introduction of triple CFTR modulators (ivacaftor/tezacaftor/elexaftor and ivacaftor) has significantly improved the prognosis. However, the high cost of the original drug Trikafta® and limited healthcare resources have driven interest in the bioequivalent generic drug Trilexa®. Aim . To evaluate the efficacy and safety of the pathogenetic therapy drugs Trikafta and Trilexa in children with cystic fibrosis in the Chechen Republic, as well as the transition from the original to the generic version within the same INN (ivacaftor + tezacaftor + elexacaftor and ivacaftor), in routine clinical practice. Materials and methods . A retrospective, single-center observational study was conducted in the Chechen Republic. The analysis included 22 patients under 18 years of age with a genotype corresponding to the indications for triple CFTR modulator therapy. Three groups were formed: Group 1 (n = 5) – treatment with Trikafta only; Group 2 (n = 2) – treatment with Trilexa only; Group 3 (n = 15) – patients switched from Trikafta to Trilexa. Changes in sweat chloride concentrations, body mass index (BMI), pulmonary function parameters (FEV₁, FVC), the frequency of pulmonary exacerbations, and the safety profile were assessed. Results . A decrease in sweat chloride concentrations was noted in all groups, indicating restoration of CFTR function. In Group 3, sweat chloride levels decreased from 91.0 to 41.0 mmol/L while on Trikafta and remained stable after switching to Trilexa. BMI significantly increased from 14.9 to 15.7 kg/m² during the Trikafta phase (p = 0.002) and continued to increase after switching to Trilexa. FEV₁ and FVC remained consistently high after switching (p > 0.05). The incidence of pulmonary exacerbations decreased and remained minimal while on Trilexa. Conclusion. The use of both the original drug and generic Trilexa in children with CF is associated with clinical improvements in key efficacy indicators and a favorable safety profile.