Regional experience in monitoring children with cystic fibrosis on targeted therapy in the Republic of Tatarstan within one international nonproprietary name

Background. Due to the widespread use of targeted therapy drugs with various trade names in the treatment of cystic fibrosis, there is a need for a clinical evaluation of the results of treatment with these drugs and their tolerability and safety within the framework of routine clinical practice.

Objective. The aim of the study was to present the experience of the Republic of Tatarstan in the etiopathogenetic therapy of ivacaftor + tezacaftor + elexacaftor and ivacaftor with a change of trade name within the same international nonproprietary name in pediatric patients diagnosed with cystic fibrosis, to demonstrate the clinical efficacy of this drug and its safety.

Conclusion. During 2025, 80 patients received etiopathogenetic therapy with ivacaftor + tezacaftor + elexacaftor and ivacaftor, including 12 children under 6 years of age, 19 aged 6 to 12 years, and 46 patients aged 12 to 18 years. The average age of patients at the start of therapy was 7.5 years. Patients with the pulmonary-intestinal form of the disease (E 84.8) prevailed – 78.3%, with a predominantly pulmonary form (E 84.0) – 21.7%. Prior to the initiation of targeted therapy and when changing the drug to a triple fixed dose combination, a comprehensive assessment of the functional capabilities of the child's body was carried out within the framework of one international nonproprietary name. Monitoring of the safety and effectiveness of treatment was carried out 14 days after the start of therapy, 1 month after, 3 months after, 6 months after, and 12 months after. Against the background of the three-component targeted therapy, patients showed an improvement in respiratory function (FEV1 and FVC), positive weight and height dynamics. When changing the drug within the same international nonproprietary name , the indicators of the function of external respiration remained at the achieved level. During the 6 months of follow-up, there were no exacerbations of cystic fibrosis that required hospitalization.

1. Clinical guidelines Cystic fibrosis in children and adults, approved on December 5, 2025, https://cr.minzdrav.gov.ru/. (In Russ.)