Luspatercept for Transfusion-Dependent β-Thalassemia

Patients with transfusion-dependent β-thalassemia are sustained by transfusions, yet this supportive therapy adds to the risk for iron overload, mandating iron chelation therapy. Additional therapies such as splenectomy are associated with short-term surgical complications along with risk for thrombosis and pulmonary hypertension. Although gene therapy is of great promise, it is not yet available outside of a clinical trial setting (N Engl J Med 2018; 378:1479). Therefore, additional supportive therapies are of interest. One potential option, luspatercept, enhances later stages of erythropoiesis and has been shown to reduce transfusion burden in a phase II study including patients with β-thalassemia (NEJM JW Oncol Hematol Mar 2019 and Blood…