The use of luspatercept for thalassemia in adults

Abstract

Luspatercept is an activin receptor ligand trap that has been shown to enhance late-stage erythropoiesis in animal models of b-thalassemia. A multicenter, international, phase 2 dosefinding study was initiated in adult patients with b-thalassemia, either non-transfusiondependent thalassemia (NTDT) or transfusion-dependent thalassemia (TDT). Positive results of the phase 2 study paved the way to a randomized phase 3 clinical trial (BELIEVE) to assess the efficacy and safety of luspatercept. The BELIEVE trial is a randomized, double-blind, placebo-controlled phase 3 trial. Three hundred thirty-six patients aged $18 years with TDT (regularly transfused, 6-20 red blood cell units within 24 weeks before randomization) were included in the trial. Patients received luspatercept or placebo subcutaneously every 21 days for$48weeks and best supportive care. Forty-eight of 224 patients (21.4%) in the luspatercept group achieved the primary end points (≥33% reduction in transfusion burden) compared with those in the placebo group (4.5%; P