Results of Targeted Therapy of Cystic Fibrosis in Children in the Orenburg Region in 2022

Abstract

Aim. To evaluate tolerability, safety and efficacy of target products used in the treatment of cystic fibrosis (CF) in children.

Design. Retrospective and prospective comparative study.

Materials and methods. The study included 12 patients aged 3 to 18 years old with complete genetic analysis of CF, which can be treated with target therapy (TT), and a chronic bronchopulmonary process. Seven children were treated with a two-component drug and five children — with a three-component drug. A control group included 12 patients with CF, but not treated with TT; the groups were similar in age and sex and had mutations of respective classes. All subjects underwent clinical, laboratory and instrumental tests and assessments. Condition was monitored at baseline, in 2 weeks, 1, 3, 6 months.

Results. At baseline, the majority of patients had minor complaints which resolved without any intervention. Adverse effects, which required drug discontinuation, were recorded in one patient treated with the two-component drug. Patients, who received the three-component therapy, had significant improvement in nutritional status, marked and stable reduction in sweat test values.

Conclusion. Modern TT for CF is well-tolerated, quickly improves patients' state, especially when a three-component drug is used. Subjective perception of improved breathing function, reported by patients, was not confirmed with pulmonary function test results and requires monitoring in a larger population.