THE FIRST RESULTS OF TARGETED THERAPY IN CHILDREN WITH CYSTIC FIBROSIS IN KAZAKHSTAN

Abstract

Introduction. Cystic fibrosis (CF) is an inherited, autosomal recessive disease caused by mutations in the CFTR gene. In the last decade, the effectiveness of CF treatment has increased significantly due to the development of targeted drugs aimed at restoring the function of the CFTR protein. The highest efficacy is observed in the triple combination elexacaftor/tezacaftor/ivacaftor (Trikafta® (Vertex Pharmaceuticals, USA). In Kazakhstan, it has been used since 2023 (generic elexacaftor/tezacaftor/ivacaftor Trilexa®, Tuteur S.A.S.I.F.I.A., Buenos Aires, Argentina). There have been no previous reports on the results of using targeted therapy in patients with CF in Kazakhstan. There is very little information about the effectiveness and safety of generic forms of CFTR modulators. Aim: to evaluate the effectiveness and safety of the generic targeted drug elexacaftor/tezacaftor/ivacaftor Trilexa® (Tuteur S.A.S.I.F.I.A., Buenos Aires, Argentina) in children and adolescents with CF in real clinical practice Materials and methods. An observational study included five children with CF aged 11 to 17 years who were treated with the three-component targeted drug elexacaftor/tezacaftor/ivacaftor. The safety of the drug was monitored by recording adverse events and the dynamics of the levels of biochemical blood parameters. The effectiveness of elexacaftor/tezacaftor/ivacaftor was assessed after 12 months based on the results of a sweat test, anthropometric characteristics, respiratory function (FEV1), number of exacerbations and courses of antibacterial therapy. Conclusion. Within 12 months of therapy with the combination of elexacaftor/tezacaftor/ivacaftor, positive dynamics of clinical and functional parameters and the safety of three-component targeted therapy were shown. It is necessary to expand the circle of children with CF receiving three-component targeted therapy and continue research to assess its effectiveness in Kazakhstan.