Abstract
Despite of the constant intense research, myelodysplastic syndrome (MDS) remains one of the most difficultly treatable haematological malignancies. Recently, after some time, new promising therapeutic approaches began to appear in this indication (currently there are about 55 new drugs evaluated).
Significant progress in the MDS treatment was obtained by introduction of demethylating agents, primarily azacitide in the therapy of high-risk patients or lenalidomide in the therapy of patients with anaemia, low-risk MDS and partial deletion of chromosome 5 (5q). Identification of prognostically significant mutations and their incorporation into the prognostic systems remains an important challenge.
Based on the presence of certain mutations, specific inhibitors, for example IDH1/2 inhibitors, have already been developed.