Hemoglobinopathies

Investigators for the phase 2 BEYOND study reported at the European Hematology Association 2021 Virtual Congress in June that 77% of non–transfusion-dependent thalassemia patients treated with luspatercept had a hemoglobin (Hb) increase of at least 1 g/dL, compared with no patients assigned to placebo (P < .0001). In addition, results of the randomized, double-blind, multicenter study showed 52.1% of patients assigned tp luspatercept achieved a mean Hb increase of 1.5 g/dL or greater during weeks 13 to 24 vs no patients who received placebo (P < .0001). Luspatercept is a first-in-class erythroid maturation agent approved to treat anemia in selected adults with lower-risk myelodysplastic syndrome.