Open AccessCYSTIC FIBROSIS: A BREAKTHROUGH IN 21ST-CENTURY THERAPY
Author(s) -
O. Simonova,
Y. Gorinova,
V.P. Chernevich
Publication year - 2020
Publication title -
rossijskij pediatričeskij žurnal
Language(s) - English
Resource type - Journals
eISSN - 2413-2918
pISSN - 1560-9561
DOI - 10.18821/1560-9561-2020-23-1-35-41
Subject(s) - ivacaftor , cystic fibrosis , medicine , azithromycin , disease , intensive care medicine , web of science , bioinformatics , cystic fibrosis transmembrane conductance regulator , meta analysis , microbiology and biotechnology , biology , antibiotics
The review presents new data on the latest advances in the treatment of cystic fibrosis, a rare genetic disease. The methods used were literature search in the Scopus, Web of Science, and EMBASE databases. The importance of a number of drugs prescribed for anti-inflammatory purposes (ibuprofen, azithromycin) is discussed, data from multicenter studies of new drugs are presented. The role of mucolytic agents and the need to develop new antibacterial compounds are shown. Particular attention in the review is given to the development of new targeted therapies for cystic fibrosis. The data of studies of ivacaftor, lumacactor, tezacactor in this category of patients are presented, as well as a spectrum of mutations of the cystic fibrosis gene in which these molecules can be prescribed. The experience of the use of correctors in adult patients with cystic fibrosis in the Russian Federation is described, the effectiveness and safety of the long-term use of these drugs are described.