Modulatory therapy experience in patients with cystic fibrosis in Turkey: A multi-center study

Abstract

Background: Modulatory therapies are not reimbursed in Turkey and approach to the treatment is currently available for three months only by court decision. In between the court decisions, most of the patients could not receive the treatment. The aim of the current study is to report the results in patients. Methods: In this ongoing study, patients who have used modulator therapy for at least 3 months from 12 centers, were included in the study. Demographical data of the patients, sweat test values at baseline and during treatment, FEV1% and lung clearence index (LCI) values, body mass indexes (BMI), side effects of the treatment, age-appropriate quality of life questionnaires (CFQR), PHQ-9 (depression) and GAD-7 (anxiety) questionnaires were recorded. Results: The median age of 111 patients was 18 years. 86 patients were receiving ivacaftor/ tezacaftor/elexacaftor, whereas 13 patients on ivacaftor, six on lumacaftor/ivacaftor, and six subjects on tezacaftor/ivacaftor. The FEV1% of 86 patients who completed the three month treatment, increased by 15.4%. The BMI value of the 91 patients who completed the three month treatment, increased by 4.8%. The increase in FEV1% value of 68 patients receiving elexacaftor/tezacaftor/ivacaftor was 26.7% (p<0.001). The increase in FEV1% of 29 patients who completed the 6 month treatment, was 18.5%. Statistical significant improvement was observed in the depression and anxiety levels and quality of life parameters of the patients at 6th month (p<0.05). Conclusion: Significant improvements of our patients demonstrate the major benefits of CFTR modulator treatments and the necessity of continuous access to this treatment for all eligible patients.