Results of Comparative Effectiveness of Pathogenetic Therapy with CFTR Modulators in Children with Cystic Fibrosis

Background. Cystic fibrosis (CF) pharmacotherapy aims to restore CFTR protein function. Comparing CFTR modulators’ effectiveness can inform personalized treatment approaches.

The aim of the study is to evaluate the effectiveness of CFTR modulator therapy in children with cystic fibrosis.

Materials and methods. An open prospective comparative non-randomized study (December 2022 — November 2023) included 81 CF patients aged 2–17 years: 35 received triple therapy (elecsacaftor/tezacaftor/ivacaftor), 23 received double therapy (ivacaftor/lumacaftor), and 23 were controls without CFTR modulators. Evaluations occurred at baseline and after 12 months.

Results. Triple therapy improved nutritional status, lung function (FEV1, FVC, MEF25–75), sweat chloride levels, pancreatic elastase, and liver enzymes (alkaline phosphatase, GGTP); double therapy improved cholestasis markers (OB, alkaline phosphatase, GGTP). Controls showed declining lung function, worsening pancreatic insufficiency, and persistently elevated alkaline phosphatase. Targeted therapy reduced hospitalizations due to bronchopulmonary exacerbations.

Conclusion. CFTR modulator therapy decreased hospitalization risks (HR = 2.09–11.00). Triple therapy had greater benefits than double therapy in improving nutrition, respiratory function, and lowering sweat chlorides (normalizing in 35.5 % of patients). Double therapy effectively reduced cholestasis.

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