Thrombocytopenia is a frequent symptom in patients with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). Eltrombopag is a small molecule thrombopoietin receptor agonist that might be a new option to treat thrombocytopenia in these diseases, provided that it can be shown does not stimulate malignant hematopoiesis. Unfortunately, there is no significant literature to support Promacta in the setting of ongoing treatment for AML or in MDS. Currenlty PROMACTA is not indicated for the treatment of thrombocytopenia due to causes of thrombocytopenia (eg, myelodysplasia or chemotherapy) other than chronic ITP. Several studies showed that Eltrombopag was capable of increasing megakaryocytic differentiation and formation of normal megakaryocytic colonies in patients with AML and MDS but the clinical significance of this observation remains unclear. Promacta is currenlty in a study: Eltrombopag in Myelodysplastic Syndrome (MDS) Patients With Thrombocytopenia, NCT01286038.
Will B, Kawahara M, Luciano JP, Bruns I, Parekh S, Erickson-Miller CL, Aivado MA, Verma A, Steidl U.
Effect of the nonpeptide thrombopoietin receptor agonist Eltrombopag on bone marrow cells from patients with acute myeloid leukemia and myelodysplastic syndrome. Blood. 2009 Oct 29;114(18):3899-908
S. Wroblewski, W. Shi, P. Mudd Jr., M. Aivado;Eltrombopag in thrombocytopenic patients with advanced myelodysplastic syndromes (MDS) or secondary acute myeloid leukemia after MDS: A phase I/II study.J Clin Oncol 28:15s, 2010 (suppl; abstr TPS184)
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