Myelodysplastic syndromes (MDS) are malignant clonal stem cell disorders. There are two main treatment options for higher-risk MDS, i.e., eligible and ineligible hematopoietic stem cell transplantation (HSCT). Transplantation-eligible patients should receive HSCT immediately, with or without prior therapy. In contrast, for patients who are ineligible for HSCT, azacitidine (AZA) may now be the first choice of treatment, which significantly prolongs overall survival in responder patients when compared with conventional care regimens. However, there are major problems regarding the management of patients with disease relapse after HSCT and those with loss of response to AZA. In this review, treatment strategies and future perspectives, including the use of novel agents, are presented with the aim of improving the outcome of higher-risk MDS.
Keywords: Azacitidine; Hematopoietic stem cell transplantation; Myelodysplastic syndromes; Novel agents.