Article
Eltrombopag for the treatment of the inherited thrombocytopenia deriving from MYH9 mutations.
Blood - 23 Dec 2010
Pecci Alessandro, Gresele Paolo, Klersy Catherine, Savoia Anna, Noris Patrizia, Fierro Tiziana, Bozzi Valeria, Mezzasoma Anna Maria, Melazzini Federica, Balduini Carlo L
Abstract excerpt
Platelet transfusion is currently the primary medical treatment for reducing thrombocytopenia in patients with inherited thrombocytopenias. To evaluate whether stimulating megakaryopoiesis could increase platelet count in these conditions, we treated patients with a severe thrombocytopenia induced by MYH9 mutations (MYH9-related disease) with a nonpeptide thrombopoietin receptor agonist, eltrombopag. Twelve adult...
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