Luspatercept in Myelodysplastic Syndromes: Who and When?

Anemia is the most common clinical manifestation of myelodysplastic syndrome (MDS), and most patients become red blood cell transfusion dependent. Defective erythropoiesis includes impaired terminal erythroid maturation. There are limited options for treatments of anemia in lower-risk MDS after failure of erythroid-stimulating agents. Luspatercept is an activin receptor type IIB fusion ligand trap novel agent. Luspatercept showed promising activity for treating anemia in patients with MDS with ring sideroblast subtypes. This article reviews the mechanism of impaired erythropoiesis in MDS. It summarizes clinical data with luspatercept and foresees how to best use this treatment in practice.