Aplastic Anemia and Eltrombopag

Aplastic anemia which was once considered as rare and invariably fatal disease. Over the years the understanding of its pathophysiology, its relationship with constitutional bone marrow failure syndrome and evolution to myelodysplastic syndrome and leukemia has improved. Evolution of standard immunotherapy and bone marrow transplantation has dramatically improved the survival of patients over the years. The optimum immunotherapy with antithymocyte globulin and cyclosporine has been developed and patients who failed even to second course of immunotherapy were also subjected to bone marrow transplantation. These strategies have achieved and improved the event free but significant numbers of cases were refractory to these treatments. Initial studies of thrombopoietin-MPL system revealed their activity to increase the number of committed progenitor cells along with their proliferation in all three lineages. Peptide agonists such as eltrombopag can bind to MPL region to initiate lineage responses. Initially it was shown to induce significant stimulus to increase platelets in patients of chronic idiopathic thrombocytopenic purpura. Subsequent clinical trials have revealed that eltrombopag induced erythropoietic stimulus even in patients of refractory aplastic anemia and resulted in improved event free and overall survivals. In the present manuscript the role of eltrombopag along with standard immunotherapy has been briefly reviewed in aplastic anemia.