Red cell transfusion has been a mainstay in treatment of both beta-thalassemia and sickle cell disease, but recent research is further exploring the role for ineffective erythropoiesis not only in thalassemia but also in sickle cell disease. Dr. Wassim El Nemer will describe the mechanisms for ineffective erythropoiesis in sickle cell disease, and its implications for treatment with hydroxyurea, voxelotor, and gene therapy. Dr. Yelena Ginzburg will discuss new insights into ineffective erythropoiesis in beta-thalassemia, and the role of newly approved activin II receptor traps as well as other emerging therapies, such as JAK2 inhibitors and hepcidin mimetics.
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Identify new research findings regarding ineffective erythropoiesis in thalassemia and sickle cell disease.
Assess the implications of these findings for treatment of these two diseases.
Explain mechanisms of action against ineffective erythropoiesis with current and emerging therapies.
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AM21-25: New Insights and Drug Development for Ineffective Erythropoiesis in Thalassemia and Sickle Cell Disease Evaluation