Abstract

Myelodysplastic syndromes (MDS) are a heterogeneous group of hematopoietic stem cell disorders. Although the currently used classification schemes and prognostic algorithms, which are based predominantly on morphologic assessment of blood and marrow smears, have been shown to be valid for defining disease subgroups, they do not take into consideration the significant biological diversity of MDS. As the numerous pathophysiologic pathways that are involved in MDS are being unraveled, new molecular targets are being identified. Novel and targeted therapeutic agents, including inhibitors of farnesyltransferases and receptor tyrosine kinases, more potent thalidomide analogs and epigenetic therapies, have produced encouraging results and might offer durable benefits to patients with MDS. This review intends to provide a concise report on some of the most up-to-date therapies being investigated in MDS.