Study of the efficacy and safety of the generic drug ivacaftor + tezacaftor + elexacaftor/ivacaftor (trade name Trilexa®)

作者：Е. И. Кондратьева, Anna Y. Voronkova, I. R. Fatkhullina, А. В. Орлов, O. G. Pyaterkina, T. Protasova, O. S. Psyurnikova · 发表于：PULMONOLOGIYA · 年份：2025 · DOI：10.18093/0869-0189-2025-35-6-832-843 · 被引用次数：6 · 研究领域：Cystic Fibrosis Research Advances、Chemical and Physical Studies、Pediatric health and respiratory diseases

Cystic fibrosis (CF) is a universal exocrine disorder that affects virtually all organs and systems containing exocrine glands. A genetic defect in the cystic fibrosis transmembrane conductance regulator gene leads to increased viscosity of exocrine gland secretions, primarily in the respiratory tract. CFTR modulators improve the synthesis, processing, and/or function of the defective CFTR protein, which is a chloride channel. Objective. To evaluate the efficacy of the generic drug ivacaftor + tezacaftor + elexacaftor/ivacaftor (trade name Trilexa®), in a group of patients not previously receiving triple targeted therapy after 3 months of treatment in patients who have not previously received the first threecomponent CFTR modulator. Methods. Data from the Russian CF Patient Registry, entered into the “Targeted Therapy” block, were analyzed for the period from January to August 2025 (main group) and for the period from January 2022 to August 2022 (comparison group). Two groups of patients were identified: Group 1 included 28 pediatric patients (12 boys/16 girls) with a confirmed diagnosis of cystic fibrosis, who started receiving triple targeted therapy generic drug ivacaftor + tezacaftor + elexacaftor/ivacaftor. Group 2, the comparison group, consisted of 34 pediatric patients (14 boys/20 girls) who started receiving the original triple targeted drug ivacaftor + tezacaftor + elexacaftor/ivacaftor (trade name Trikafta®). Results. When assessing the nutritional status, a signif...