Drug insight: emerging new drugs in the treatment of myelodysplastic syndromes.
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.
Faderl
Stefan
S
Department of Leukemia, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Blvd., Houston, TX 77030, USA. sfaderl@mdanderson.org
Kantarjian
Hagop M
HM
D000970:Antineoplastic Agents / Q000494:pharmacology / Q000627:therapeutic use*; D004791:Enzyme Inhibitors / Q000627:therapeutic use; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D009190:Myelodysplastic Syndromes / Q000188:drug therapy* / Q000503:physiopathology; D013792:Thalidomide / Q000031:analogs & derivatives / Q000627:therapeutic use
D000970:Antineoplastic Agents; D004791:Enzyme Inhibitors; D007166:Immunosuppressive Agents; D013792:Thalidomide