Luspatercept: A Review in Transfusion-Dependent Anaemia due to Myelodysplastic Syndromes or β-Thalassaemia

Luspatercept (Reblozyl(R)), a first-in-class erythroid maturation agent, is approved in several countries worldwide for the treatment of adults with transfusion-dependent anaemia due to myelodysplastic syndromes (MDS), who have failed prior erythropoiesis-stimulating therapy, or beta-thalassaemia. In pivotal, placebo-controlled, phase III trials, subcutaneous luspatercept significantly reduced red blood cell (RBC) transfusion requirements in patients with MDS or beta-thalassaemia. Luspatercept had a generally manageable tolerability profile in clinical trials. Adverse events of special interest include thromboembolic events, hypertension and bone pain. Thus, luspatercept is an emerging treatment option in adults with transfusion-dependent anaemia due to MDS or beta-thalassaemia.

Automated summary

Luspatercept, a novel erythroid maturation agent, has shown significant reduction in red blood cell transfusion requirements for patients with MDS or beta-thalassaemia in pivotal phase III trials. It has a generally manageable tolerability profile with adverse events of special interest including thromboembolic events, hypertension, and bone pain. As a result, Luspatercept is an emerging treatment option for adults with transfusion-dependent anaemia due to MDS or beta-thalassaemia.