“Luspatercept: a lifesaving therapy for transfusion-dependent thalassemia patients with alloimmunization complications”

Abstract

This case demonstrates the benefits of luspatercept for thalassemic patients who are unable to receive adequate blood transfusions. It acts as a selective ligand trap and inhibits TGF- signaling via Smad2/3 to promote differentiation and maturation of late-stage erythroid precursors. Phase III clinical data showed that a 33% reduction in transfusion burden from baseline was achieved. The administration of luspatercept was a challenge due to the coexistence of antiphospholipid syndrome, portal vein thrombosis and splenectomy. Since initiating luspatercept, the patient has not experienced any adverse events and her general condition has improved.