Targeted therapies in myelodysplastic syndrome.

Therapeutic alternatives for patients with myelodysplastic syndrome (MDS) have expanded in recent years but remain limited. While agents approved by the US Food and Drug Administration (FDA), including azacitidine, decitabine, and lenalidomide, have yielded hematologic and cytogenetic responses in a substantial portion of patients, these therapies are not curative. Active investigation of novel targets with biological relevance in myelopoiesis has stimulated the pharmacologic development of a multitude of agents that show promise in the treatment of MDS. Many of these drugs have entered or completed early phase clinical testing in MDS and include immunomodulatory agents, immunosuppressive therapies, survival signal inhibitors, thrombopoiesis-stimulating agents, pharmacologic differentiators, and anti-angiogenic and apoptotic agents. As we continue to collect clinical experience with these agents, the repertoire of available therapeutics for the treatment of MDS will expand and provide a foundation for novel therapeutic combinations.