Léčba Erdheimovy-Chesterovy choroby.

Erdheim-Chester disease (ECD) is a rare form of histiocytosis, in present time ECD is classified as Langerhans-cell histiocytosis. It encompasses a spectrum of disorders ranging from asymptomatic bone lesions to multisystemic, life-threatening variants with CNS involvement. ECD prognosis is very variable. In this article, ECD treatment possibilities are widely discussed. The first line treatment is usually cladribine and the second line treatment is anakinra. ECD therapy is only partially effective with 50% treatment responses in various modalities at the maximum. ECD pathogenesis understanding has been widely improved with the discovery of MAPK pathway mutations, that have led to the use of targeted therapy (MEK and BRAF inhibitors) trials, with major efficacy. In the year 2020 BRAF and MEK inhibitors are recomended as first line treatment for severe CNS impairment, in other ECD cases the BRAF and MEK inhibitors are used as the second-line therapy. [ABSTRACT FROM AUTHOR]