Emanuele Tanzi, Simona Maria Di Modica, Jessica Bordini, Violante Olivari, Alessia Pagani, Valeria Furiosi, Laura Silvestri, Alessandro Campanella, Antonella Nai
β-thalassemia is a disorder characterized by anemia, ineffective erythropoiesis (IE), and iron overload, whose treatment still requires improvement. The activin receptor-ligand trap Luspatercept, a novel therapeutic option for β-thalassemia, stimulates erythroid differentiation inhibiting the transforming growth factor β pathway. However, its exact mechanism of action and the possible connection with erythropoietin (Epo), the erythropoiesis governing cytokine, remain to be clarified. Moreover, Luspatercept does not correct all the features of the disease, calling for the identification of strategies that enhance its efficacy...
April 17, 2024: American Journal of Hematology