Myelodysplastic syndromes (MDS) are hematopoietic stem cell neoplasms, which are characterized as bone marrow failure with a propensity to develop acute myeloid leukemia (AML). Patients with MDS tend to be of higher age, with usually poor antineoplastic chemotherapy response. The life prognosis of MDS is poor, and its curative treatment is limited to allogeneic hematopoietic cell transplantation. Recent advances in hematopoietic cell transplantation, including alternative donors or reduced-intensity conditioning regimens, resulted in an expanded treatment indication for patients of higher age. Moreover, several novel therapeutic agents are implemented after the elucidation of pathological processes in MDS. Therapeutic agents that can alter the disease course, including azacitidine and lenalidomide, are approved and available in Japan. Additionally, other therapeutic agents for MDS, such as erythropoiesis-stimulating agents and oral iron chelators, are also available, and clinical trials for novel agents are also underway. This study discussed the updated risk-stratified treatment strategies for MDS and the development of novel agents.
Keywords: IPSS-R; Myelodysplastic syndromes.