Abstract:
Myelodysplastic syndromes (MDS) are a group of common myeloid neoplasms, the incidence of which increases with age. The treatment options for patients with MDS differ primarily according to the prognosis group in which they fall, and patients with different risk stratifications have different treatment goals. Most patients belong to the lower-risk group and their presentation is complicated with anemia. Therefore, improving both the anemia and quality of life is the main therapeutic goal. Owing to the harm of repeated transfusions as well as the lack of patient response to classical drugs such as erythropoiesis-stimulating agents (ESAs) and lenalidomide, the development of more effective and safer drugs for anemia is needed. Luspatercept, which is a red blood cell maturation agent, has been approved nationally and abroad for the treatment of patients with beta-thalassemia. In this article, we review the mechanism of action of luspatercept and clinical studies related to its use in the treatment of MDS as well as its efficacy and safety. Additionally, further guidance for its use in clinical practice is provided.