The FDA has granted a breakthrough therapy designation to eltrombopag (Promacta) for use in combination with standard immunosuppressive therapy as a first-line treatment for patients with severe aplastic anemia (SAA).
SAA is a rare blood disorder in which a patient's bone marrow fails to produce enough red blood cells, white blood cells and platelets, leading to symptoms such as fatigue, dyspnea, recurring infections, and abnormal bruising or bleeding. About two-thirds of patients with SAA achieve some degree of hematologic response with conventional immunosuppressive therapy, most often the combination of ATG and cyclosporine. However, response rates have been stable for 3 decades despite ongoing efforts to improve outcomes in the condition.
Townsley DM, Dumitriu B, Scheinberg P, et al. Eltrombopag added to standard immunosuppression for aplastic anemia accelerates count recovery and increases response rates. Presented at: 57th American Society of Hematology Annual Meeting; Orlando, Florida; December 5-8, 2015. Abstract LBA-2.
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