Evaluating luspatercept responders in the phase 3, randomised, double-blind, placebo-controlled BELIEVE trial of luspatercept in adult beta-thalassaemia patients who require regular red blood cell transfusions

Introduction: β-thalassemia is an inherited hemoglobinopathy characterized by ineffective erythropoiesis and anemia. Luspatercept is a first-in-class erythroid maturation agent that binds to select TGF-β superfamily ligands to reduce aberrant Smad2/3 signaling and enhance late-stage erythropoiesis.