Eltrombopag for the treatment of the inherited thrombocytopenia deriving from MYH9 mutations.

@article{Pecci2010EltrombopagFT,
  title={Eltrombopag for the treatment of the inherited thrombocytopenia deriving from MYH9 mutations.},
  author={Alessandro Pecci and Paolo Gresele and Catherine Klersy and Anna Savoia and Patrizia Noris and Tiziana Fierro and Valeria Bozzi and Anna Maria Mezzasoma and Federica Melazzini and Carlo Luigi Balduini},
  journal={Blood},
  year={2010},
  volume={116 26},
  pages={5832-7}
}
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 patients with MYH9-RD and platelet counts of less than 50 × 10(9)/L received 50 mg… CONTINUE READING