Luspatercept, a first-in-class erythroid maturation agent is approved by the European Medicine Agency (EMA) for the treatment of adult patients with transfusion-dependent anaemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts, who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy. Luspatercept showed promising activity for treating anaemia in lower risk myelodysplastic syndrome with ring sideroblasts. Both the PACE and MEDALIST trial showed high rates of durable red blood cell transfusion independence and hematological improvement with luspatercept and a manageable toxicity profile.

(BELG J HEMATOL 2021;12(8):344–8)