Treatment of Acute Myeloid Leukemia and Myelodysplastic Syndromes

Abstract

Recent advances in understanding the mechanisms and pathways leading to the pathogenesis of myeloid neoplasms, acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS), have led to the identification of numerous molecular abnormalities that may be responsible for leukemogenesis and are resulting in progress in the clinical management of these disorders. In AML, large prospective trials conducted over the last 20–30 years have established standard regimens combining cytotoxic agents, in particular cytarabine and anthracyclines. Current research in AML is attempting to better stratify patients by identifying the risk factors responsible for chemotherapy resistance, and ways to incorporate newer agents with specific and targeted activity into the standard regimens. Treatment of MDS has been problematic and until recently no effective drugs were available for managing patients with these disorders. However, the identification of epigenetic modifiers and immunomodulatory agents has now improved outcomes in MDS. New agents and combinations aim to improve on the results obtained with the currently available drugs.