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Experience of using targetted drugs in children with cystic fibriosis in the Astrakhan region
[Pediatrics]
Olga Bashkina; Diana Fikretovna Sergienko;
The article presents the clinical experience of using the lumacaftor/ivacaftor in 7 children with F508del/F508del genotype for 24 weeks and using the elexacaftor/tezacaftor/ivacaftor+ivacaftor in 3 patients with F508del genotypes in combination with one of 178 mutations, including F508del for 12 weeks. During the study, four from the seven patients discontinued the lumacaftor/ivacaftor due to lack of efficacy and serious adverse events. In 3 patients, there was a significant increase in the parameters of the function of external respiration against the background of a decrease in the values of sweat chlorides. In the course of the work, a positive assessment was given of the action of the modulator Trikafta®, its high efficiency, safety and good tolerability, followed up for 12 weeks in 3 patients.
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Keywords: cystic fibrosis, lumacaftor/ivacaftor, children, elexacaftor/tezacaftor/ivacaftor+ivacaftor, Astrakhan region
Founders:
Stavropol State Medical Academy
Pyatigorsk State Research Institute of Balneotherapeutics
Pyatigorsk State Pharmaceutical Academy