ASSOCIATION OF NUTRITIONAL STATUS AND MUCOACTIVE THERAPY REGIMEN WITH LUNG FUNCTION PARAMETERS IN CHILDREN WITH CYSTIC FIBROSIS AGED 6–17 YEARS RECEIVING ELEXACAFTOR/TEZACAFTOR/IVACAFTOR: A CROSS-SECTIONAL STUDY

Abstract

Introduction: Cystic fibrosis is an autosomal recessive disease caused by mutations in the CFTR gene, leading to impaired ion transport, thickening of secretions, and damage to the bronchopulmonary system. Progressive mucoobstruction remains the leading cause of mortality. To improve sputum rheological properties, mucolytics (dornase alfa) and rehydrating agents (hypertonic saline, mannitol) are used; however, their role in the era of highly effective targeted therapy remains debatable. Aim: To evaluate the relationship between nutritional status, the regimen of mucoactive therapy, and pulmonary ventilation function parameters in children with cystic fibrosis receiving targeted therapy. Materials and methods: A single-center cross-sectional study was conducted involving 50 patients (aged 6–17 years) with cystic fibrosis receiving elexacaftor/tezacaftor/ivacaftor. Spirometry parameters (FEV1, FVC), nutritional status (BAZ), and the inhalation therapy regimen (dornase alfa, hypertonic saline) were analyzed. Results: The study included 24 (48%) boys and 26 (52%) girls, with a mean patient age of 12.5±3.1 years. A statistically significant positive correlation was found between BAZ and FEV1 (Rs=0.432; p=0.002) and FVC (Rs=0.450; p=0.001). Patients receiving combined mucoactive therapy (dornase alfa + hypertonic saline) had significantly higher FEV1 values compared to the dornase alfa monotherapy group (p=0.027). The frequency of administration of each drug individually did not correlate significantly with functional parameters (p > 0.05). Conclusion: Nutritional status is closely associated with lung function in children with cystic fibrosis. Combined mucoactive therapy is associated with a clinically significant improvement in bronchial patency even in the context of targeted treatment, which justifies maintaining mucolytic therapy in the comprehensive management of patients. Prospective randomized studies are required to definitively confirm the benefit of the combination.