Introduction: In our country, treatment with Elexacaftor/Tezacaftor/Ivacaftor+Ivacaftor first started in patients with the worst lung function, with the lowest FEV1 (forced expiratory volume in the first second).
Aims: To demonstrate the effect of modulatory therapy in CF patients with moderate and severe clinical picture.
Methods: The data were taken from the medical history, before starting the therapy and six months after its application (Mar-Aug 2023).We used the sweat test, spirometry (FEV1), enzyme status:AST(Aspartate aminotransferase,ALT(Alanine aminotransferase), BMI-nutritional status, number of exacerbations. Need for oxygen therapy.
Results: Sweat test, before therapy mean value is 94 (75-115) mmol/l, six months after therapy 40 mmol/l (22-96). FEV1-before therapy, mean value 30.5% (16-40) and after therapy mean value 47% (22-59%). BMI at the beginning of therapy, mean value 21.8 (16.2-28.9) and after 6 months of modulatory drug, mean value 23 (17.4-28.3). Hepatic enzymes:ALT pre-mean value 22.6 U/L, after 43 U/L. AST pre-mean value 19.7U/L, after 32 U/L. Тhree patients were on oxygen support before therapy, after 6 months of modulators, only one patient is still on oxygen. Оne patient had 2 hospitalizations, one had 1 hospital stay, and another 2 patients had an exacerbation, which were treated at home with per os antibiotics.
Conclusions: Modulatory therapy in CF patients with moderate and severe clinical picture has a positive effect on nutritional status, improvement of lung function, correction of chloride channel function, slight elevation of hepatic enzymes. There is a decrease in the number of exacerbations and the need for oxygen therapy at home.