Biological Basis of Novel Therapies for Myelodysplastic Syndrome

Myelodysplastic syndrome (MDS) represents a group of malignant clonal disorders characterized by ineffective hematopoiesis and an accumulation of hematopoietic precursors in the bone marrow, frequently resulting in transformation to acute leukemia and death. However, recent understanding of MDS pathophysiology has provided novel treatment approaches with pharmacologic agents capable of manipulating pathways intrinsic and extrinsic to the malignant hematopoietic clone. These advances provide new therapeutic options for patients and provide a basis for further understanding and treatment of this disease.