Targeted therapy in adult patients with cystic fibrosis: impact on the course of lower respiratory tract infections and the effectiveness of antibacterial treatment according to the registry of the Russian Federation

   CFTR modulators significantly improve clinical parameters and quality of life in people with cystic fibrosis (CF), but the effect of this treatment on the microbial profile of the respiratory tract has not been sufficiently studied.

   The aim. To assess changes in the microbial profile and clinical characteristics in adult CF patients during targeted therapy, to determine the correlation of these parameters with the duration of CFTR modulators treatment.

   Methods. The study included data on patients over 18 years of age from the national registry of patients with cystic fibrosis for 2021 – 2023. Data on microbiological, functional and anthropometric parameters of patients, as well as information on the use of CFTR modulators and antibiotics were analyzed.

   Results. The study included 1 141 adult patients, the average age was 25.32 years, the proportion of men was 50.74 %. CFTR modulators were taken by 450 patients. Only in this group was a significant improvement in forced expiratory volume in the first second (p < 0.001) and an increase in body mass index observed in 2021 – 2023. Pseudomonas aeruginosa is the dominant pathogen, most often in the form of chronic infection. The groups by the dominant pathogen remained stable; the change in groups in 283 of 1,141 patients was not associated with the use of CFTR modulators or its duration. At the beginning of therapy with CFTR modulators (less than one year), an increase in the number of patients receiving oral antibacterial therapy was noted. With a longer use of targeted therapies, the number of courses of antibacterial therapy decreases.

   Conclusion. According to the national registry of patients with cystic fibrosis for 2021 – 2023, the number of adults receiving CFTR modulators is increasing. Targeted therapy significantly improves respiratory function and nutritional status, but the microbiological profile of the respiratory tract of patients over 18 years of age did not change in the first years of treatment.

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