Affiliation:
1. National Medical Research Center for Otorhinolaryngology;
Pirogov Russian National Research Medical University
2. Research Institute of Childhood, Cystic Fibrosis CentreResearch Centre for Medical Genetics
3. Pirogov Russian National Research Medical University
Abstract
Introduction. Chronic rhinosinusitis (CRS) with underlying cystic fibrosis (CF) is one of the most challenging inflammatory diseases of the paranasal sinuses (PNS). The treatment of CF in general and CF-associated CRS in particular keeps developing exponentially: special drug delivery systems have been invented, drugs purpose-developed for the treatment of patients with CF (dornase alfa) are used, and radicality of surgical intervention is increasing. In recent years, CF innovative targeted therapy has been introduced. It aims to improve the course of the bronchopulmonary infectious and inflammatory process, nutritional status and pancreatic function, but its efficacy in relation to the PNS condition is still insufficiently studied.Aim. To evaluate the efficacy of ivacaftor/elexacaftor/tezacaftor therapy for CF sinonasal manifestations in children and to determine possible parallels of the CRS course vs other characteristics of the CF course.Materials and methods. A total of 15 children with CF receiving triple targeted therapy were included in the first series of observations. Computed tomography (CT) scans were assessed using the Lund-Mackay scores (LMS) before and 7.6 ± 3.0 months after starting the therapy. In addition, the changes in sweat chloride levels and pulmonary function test results were taken into account. The control group included 11 patients with comparable initial radiographic data who did not receive such therapy.Results and discussion. The treatment group showed a significant improvement in the PNS condition in the form of a decrease in the LMS scores from 15.6 ± 4.5 to 2.1 ± 3.0 (p = 0.001) with inhomogeneous changes vs different PNS groups. A certain parallelism between changes in the CT view and the level of sweat chlorides and pulmonary function test results was reported. In the control group, a nonsignificant deterioration in the course of CRS was observed (from 12.3 ± 6.3 to 14.6 ± 4.9 scores according to LMS; p = 0.108).Conclusions. The study demonstrated high efficacy of CF triple targeted therapy with ivacaftor/elexacaftor/tezacaftor in relation to the severe CRS with a rapid onset of effect in the form of PNS pneumatization restoration according to CT findings.
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