ABSTRACT

The treatment of MDS remains difficult and, apart from allogeneic stem cell transplantation (SCT), is generally not curative. However, some major improvements have been made in the last few years. They arise from improvements in allogeneic SCT, including better donor-recipient matching by allelic HLA typing, nonmyeloablative conditioning that allows transplanting older patients, and more effective anti-infective treatments during the posttransplant period, including antifungal agents (see chap. 21). They also arise from the advent of new drugs like hypomethylating agents (see chap. 20) that, at least for one of themazacytidine-can prolong survival in higher risk MDS, and lenalidomide, which appears to be a “targeted” drug in MDS with del(5q) (chap. 19). Further progress results from more rationale use of erythroid stimulating agents (ESAs) including EPO and darbepoietin (chap. 17), and from better recognition of iron overload due to RBC transfusions and its reversal by adequate chelation therapy (chap. 7).