Successful treatment of amegakaryocytic thrombocytopenia with azathioprine.

Acquired amegakaryocytic thrombocytopenia (AAMT) is an entity characterized by severe thrombocytopenia with a significantly reduced number of megakaryocytes in the bone marrow. AAMT is rare and poorly defined. Therefore, standard treatment is not well established. In general, steroids are considered the frontline treatment while anti-thymocyte globulin and cyclosporine are reported to be effective in scattered reports. We report a case of AAMT which was successfully treated with azathioprine 3 mg/kg/day. The clinical bleeding tendency resolved after treatment for 4 weeks and complete remission was documented after 6 weeks. Azathioprine treatment for AAMT is low risk, convenient, and cost-effective. Our successful experience suggests that azathioprine is potentially the treatment of choice after steroid failure.