Regional experience in monitoring children with cystic fibrosis on targeted therapy in the Republic of Tatarstan

Abstract

In connection with the introduction of targeted therapy for cystic fibrosis (CF) into clinical practice, there is a need to study the clinical effectiveness of these drugs and to record their possible side effects in children.

The aim was to present the regional experience of the Republic of Tatarstan in the use of etiopathogenetic therapy (ivacaftor + tezacaftor + elexacaftor) in 41 children diagnosed with CF.

Methods. Before initiation of triple therapy, a comprehensive assessment of body functions was carried out. Monitoring of safety and effectiveness was carried out at 14 days, 1 month, 3 months, 6 months, and 12 months after from the start of therapy.

Results. In the first 2 weeks from the start of therapy, 10 (27%) patients reported mild side effects in the form of increased cough, sputum, and rhinorrhea, 2 (5,4%) patients had skin rashes, and 3 (8,1%) patients had elevated blood aminotransferases. One child developed encephalopathy due to arterial hypertension several months after starting therapy.

Conclusion. Monitoring of the safety of the therapy over 12 months in children with CF showed a satisfactory profile. The therapy was associated with an improvement in the body functions, nutritional status, and quality of life.