Klin Farmakol Farm. 2024;38(4):137-144 | DOI: 10.36290/far.2024.021

Benefit of innovative therapy in patients with cystic fibrosis

Mária Kolesárová¹, Michaela Križová¹, Dana Marcinčáková¹, Zuzana Mačeková²

¹ Katedra farmakológie a toxikológie, Univerzita veterinárskeho lekárstva a farmácie v Košiciach, Slovenská republika

² Katedra lekárenstva a sociálnej farmácie, Univerzita veterinárskeho lekárstva a farmácie v Košiciach, Slovenská republika

Cystic fibrosis (CF) is an autosomal recessive genetic disease caused by a genetic mutation of the gene for CFTR (Cystic Fibrosis Transmembrane Conductance Regulator), which encodes a protein - CF transmembrane conductance regulator ensuring, among other things, the movement of chloride ions through the cell membrane. The clinical picture of the disease is characterized by chronic inflammation of the bronchopulmonary system, pancreatic insufficiency and increased salt content in sweat. The aim of the work was to determine the benefit of innovative combined therapy with CFTR protein modulators ivacaftor/tezacaftor/elexafactor (Kaftrio) in combination with ivacaftor (Kalydeco) and ivacaftor/lumakaftor (Orkambi). The results were obtained through a questionnaire filled out by 26 respondents with CF taking these CFTR protein modulators. 20 women and 6 men took part in the survey, and the average age of the respondents was 27.5 years. The majority of patients (92 %) were assigned to the combination of ivacaftor/tezacaftor/elexafactor in combination with ivacaftor for 24 months and 36 months. Two patients (8 %) took ivacaftor/lumacaftor for 24 months and 36 months. The effectiveness of the innovative CF therapy was evaluated mainly by monitoring the values of FEV₁ (expiratory volume of air in one second), sweat chloride and weight of the patients, whose values were improved in almost all patients compared to the period before the indicated innovative therapy. This indicates a change and improvement in airway function. From the results, it can be confirmed that the innovative therapy with CFTR protein modulators for CF patients significantly improved the patients' quality of life.

Keywords: CFTR, cystic fibrosis, elexacaftor, ivacaftor, correctors, lumacaftor, potentiators, tezacaftor.

Accepted: January 23, 2025; Published: February 4, 2025  Show citation