Hypomethylating agents and chemotherapy in MDS

Until recently, the treatment of higher risk myelodysplastic syndrome was based on [1] Intensive chemotherapy using anthracycline-AraC combinations, leading to a lower complete remission rates and a shorter CR duration compared with de novo AML [2], low dose chemotherapy with limited CR rate mainly restricted to patients with normal karyotype. Azacitidine was the first drug to significantly improve survival in higher risk MDS, although it is not curative. Thus, the survival improvement obtained with azacitidine must be the starting point for combination studies, and for utilization of this drug in other situations (before allo SCT, or after chemotherapy or allo SCT as maintenance treatment).