CFTR Modulator Therapy For Cystic Fibrosis

Abstract

Introduction
Cystic fibrosis (CF) is the most common life-limiting autosomal recessive condition in Caucasians, affecting the respiratory system, digestive tract and all exocrine glands. It is caused by a mutation in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The genetic analysis, in addition to diagnosis, serves to create a modern individual CFTR modulator therapy.
Aim: The aim of this article is to do a presentation of products for CFTR modulator therapy, which are available worldwide; introduction to their pharmacokinetic and pharmacodynamic properties; evaluation of the effect of their application.
Materials: We have conducted a study of medical literature related to the products for CFTR modulator therapy and review of information on the topic.
Results: The use of approved CFTR modulator therapy products in patients suitable for their use (specific genotype) results in an improvement in FEV1 values and body mass index and a reduction in the incidence of exacerbations in these patients.
Conclusion: CFTR modulator therapy significantly improves the prognosis and quality of life of cystic fibrosis patients as a result of certain mutations in the CFTR gene. Quality care for other patients with other mutations is especially important so that they can benefit from personalized treatment in the future.