Marrowforums - Promacta Approved for U.S. Treatment of Severe Aplastic Anemia

On August 26, 2014, the U.S. Food and Drug Administration approved use of Promacta (eltrombopag) for treatment of severe aplastic anemia when the patient has had an insufficient response to immunosuppressive therapy such as ATG, and when the patient is not a transplant candidate.

Promacta increases production of blood cells by inducing stem cell proliferation and differentiation in the bone marrow. It is taken orally, once daily. In a trial, about 40 percent of patients who had not responded to immunosuppression had clinically significant responses to Promacta. Some patients were able to discontinue taking Promacta while maintaining their blood counts across all three blood lines.

Promacta is generally well tolerated although one-fifth to one-third of patients may experience nausea, fatigue, cough, diarrhea, or headaches.

References:

GlaxoSmithKline Press Release
AA&MDSIF Press Release