Transplantation for myelodysplastic syndrome in the era of hypomethylating agents

Abstract

This review summarizes the available data on the role of hypomethylating agents in the setting of hematopoietic cell transplantation (HCT) for myelodysplastic syndrome (MDS). Although hypomethylating agents have been established as standard of therapy for MDS in the nontransplant setting, the role of these agents in patients who are candidates for HCT or are undergoing HCT is less well defined. Hypomethylation therapy has been investigated in both the pre-HCT and post-HCT setting. Patients who are transplant candidates and are given pre-HCT hypomethylating therapy should proceed with HCT when 'best response' is achieved; HCT when hypomethylation has failed is associated with inferior outcome. Only limited data have been presented on the use of hypomethylating agents after HCT. Although this approach may prove to be useful in reducing post-HCT relapse, such therapy should only be given in the setting of clinical trials. Treatment planning for patients with MDS who are HCT candidates should comprise the entire treatment arc including pre-HCT debulking, possibly with hypomethylating agents, conditioning regimen, and potential post-HCT treatment, be it prophylactic, pre-emptive or therapeutic.