Hemoglobinopathies

At the 63rd Annual Meeting of the American Society of Hematology (ASH), an international team of researchers presented results of the phase 2 BEYOND trial (poster 3081), which indicate improved quality of life (QoL) outcomes and reduced red blood cell transfusion (RBCT) burden in patients with non-transfusion-dependent beta-thalassemia (NTDT) treated with the recombinant fusion protein luspatercept (Reblozyl). Luspatercept is an activin receptor ligand trap that binds to select transforming growth factor-β superfamily ligands to reduce aberrant Smad2/3 signaling; it has been shown to enhance late-stage erythropoiesis in beta-thalassemia.