Clinical Medicine Insights: Therapeutics

Myelodysplastic syndromes (MDS) are marked by progressive cytopenias and risk of transformation to acute myeloid leukemia. Supportive care with transfusions, antibiotics, and hematopoietic growth factors has long been the mainstay of therapy for MDS, given that most patients are not eligible for more intensive chemotherapy. The hypomethylating agent 5-azacitidine (AZA) was the first chemotherapeutic agent approved by the U.S. Food and Drug Administration for the treatment of MDS, and it represented a real advance in the management of the disease. In Phase III trials, azacitidine demonstrated a higher response rate and a longer overall survival compared to supportive care alone. Importantly, it is a well-tolerated drug that can be given IV or SC in various outpatient schedules. Future studies are expected to evaluate the activity of AZA in combination with other epigenetic modifying agents and to establish the relative efficacy of azacitidine and decitabine. This review summarizes the current treatment landscape in MDS and specifically addresses the role of azacitidine in the management of MDS.