Treating thalassemia: ‘thinking out of the box’


Strategies to improve beta-thalassemia are iron restriction, use of Hamp agonists, activators of ferroportin inhibitors, improving effective erythropoiesis, and using TGF-beta/SMAD modulators or GATA1 modulators. Can a combination of erythroid stimulating agents with iron restriction drugs further improve anemia in this disease?

“Erythropoietin can improve anemia, but also increase the number of progenitor erythroid cells, aggravating the splenomegaly,” stated dr. Stefano Rivella (Children’s Hospital Philadelphia, USA) in the Plenary session on Sunday. “Iron restriction agents improve anemia without increasing the number of erythroid progenitors. Combinations of agents that increase erythropoiesis (by erythropoietin) and iron restriction have the potential to improve anemia in beta-thalassemia, limit splenomegaly and decrease iron overload.”

His research suggests that acute or tailored administration of erythroid stimulating agents could be utilized, in combination with drugs that limit iron intake, to improve anemia, splenomegaly and iron overload. This is just a proof of principle, thinking ‘out of the box’.”

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