New therapeutic options in myelodysplastic syndrome: Literature review and single-center treatment results

Abstract

Myelodysplastic syndromes (MDS) comprise a group (continuum) of clonal hematopoietic diseases associated with high risk of transformation into acute myeloid leukemia (AML) and unfavorable prognosis. Compared to the other hematologic malignant diseases, there was only a modest improvement in survival of MDS patients over the last years. Allogeneic stem cell transplantation remains the only curative option for these patients, how-ever, most of them are not candidates for transplanta-tion. This review focuses on the long-term outcomes of existing therapies and novel agents that are currently tested at different stages of clinical trials. These include inhibitors of TGFβ, various kinase inhibitors, and immune checkpoint inhibitors. Administration of new therapies in the patients with different pathogenetic MDS variants is discussed.