Rationale of ferroportin inhibition for β-thalassemia management

beta-Thalassemia is associated with decreased synthesis of beta-globin chains leading to anemia and ineffective erythropoiesis. The ineffective erythropoiesis contributes to the anemia and causes iron overabsorption to support the increased iron demand for hemoglobin synthesis, leading to organ iron overload. As a result, these patients require chronic red blood cell transfusions to survive, and iron chelation therapy to counterbalance the iron intake and prevent iron accumulation and subsequent organ damage. Alongside with currently available therapeutic approaches, there are numerous new agents targeting impaired iron metabolism in beta-thalassemia. In this manuscript, we review the potential of ferroportin inhibition in preventing iron overload in these patients.

Automated summary

beta-Thalassemia is a condition characterized by reduced synthesis of beta-globin chains, leading to anemia and ineffective erythropoiesis. Ineffective erythropoiesis contributes to the anemia and causes excessive absorption of iron, resulting in iron overload in organs. This necessitates chronic red blood cell transfusions and iron chelation therapy for these patients. In addition to current therapeutic approaches, there are several new agents that target impaired iron metabolism in beta-thalassemia.