Acquired Amegakaryocytic Thrombocytopenia With Evolution Toward Aplastic Anemia Treated With Thrombopoietin Receptor Agonists: A Case Report.

Source: PubMed, NCBI / U.S. National Library of Medicine

Amegakaryocytic thrombocytopenia is a rare hematological disorder that is characterized by absent or near-absent megakaryocytes without evidence of other hematological abnormalities. The disease can either be congenital or acquired. Given that less than a hundred cases of acquired amegakaryocytic thrombocytopenia (AAMT) have been reported in the literature, its pathophysiology and treatment remain poorly understood. Various treatments have been proposed for AAMT, including glucocorticoids and other immunosuppressive agents, such as cyclosporine, antithymocyte globulin, and rituximab. To date, there are limited data on the role of thrombopoietin receptor agonists (TPO-RAs) for the treatment of AAMT. We present the case of a patient who initially presented with ecchymoses, purpura, and petechiae and was ultimately diagnosed with AAMT. He was treated with eltrombopag and had an increase in platelets with decreased bleeding after approximately 6 months of eltrombopag therapy. He did not experience any side effects from eltrombopag or require any platelet transfusions during follow-up. Treatment overlapped with cyclosporine and prednisone, but these were discontinued due to adverse effects. Our case demonstrates that TPO-RAs can be effective novel therapeutic agents and improve patient outcomes for this rare condition.