National Center for Advancing and Translational Sciences Genetic and Rare Diseases Information Center, a program of the National Center for Advancing and Translational Sciences

Acquired amegakaryocytic thrombocytopenia

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I have acquired amegakaryocytic thrombocytopenia. Can you help me find more information on this disease, its treatment, and its long-term prognosis?

The following information may help to address your question:


What is acquired amegakaryocytic thrombocytopenia?

Acquired amegakaryocytic thrombocytopenia is a rare blood disorder that causes severe thrombocytopenia with no other blood abnormalities. It is so named because the level of large bone marrow cells that produce platelets, called megakaryocytes, are significantly lower or absent.[1][2] Signs and symptoms of the condition include prolonged bleeding; easy bruising; rash (pinpoint red spots called petechia); bleeding in the mouth and gums; and/or frequent nosebleeds.[3][4] There are many potential causes of the condition. Although standard treatment guidelines have not been established, various immunosuppressive treatment approaches have been utilized with success in affected people.[1][2] 
Last updated: 10/26/2015

What causes acquired amegakaryocytic thrombocytopenia?

There are many potential causes of acquired amegakaryocytic thrombocytopenia. For example, the condition can be associated with:[1][2]
In some people, the condition is an early sign of a more severe progressive disease, such as aplastic anemia, myelodysplasia, or leukemia.[1]
Last updated: 10/26/2015

How might acquired amegakaryocytic thrombocytopenia be treated?

Standard treatment guidelines have not been established for acquired amegakaryocytic thrombocytopenia. However, various immunosuppressive treatment approaches have been utilized in affected people. In several case reports, affected people were successfully treated with cyclosporine either alone or in combination with other immunosuppressive medications (i.e. antithymocyte globulin). Other therapies for acquired amegakaryocytic thrombocytopenia have included rituximab, danazol, azathioprine, and bone marrow transplant with variable success.[1]

To determine the best treatment for you or a family member, please speak with a healthcare provider.
Last updated: 10/26/2015

What is the long-term outlook for people with acquired amegakaryocytic thrombocytopenia?

The long-term outlook (prognosis) for people with acquired amegakaryocytic thrombocytopenia varies based on the underlying cause. Some people respond well to treatment and long-term remissions have been documented in several case reports.[1] In others, the condition progresses rapidly to aplastic anemia or myelodysplasia.[5] There are currently no good predictors to aid in assessing the likelihood of response to therapy or overall prognosis.[6]
Last updated: 10/26/2015

We hope this information is helpful. We strongly recommend you discuss this information with your doctor. If you still have questions, please contact us.

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GARD Information Specialist

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  1. Brown GE, Babiker HM, Cantu CL, Yeager AM, Krishnadasan R.. "Almost bleeding to death": the conundrum of acquired amegakaryocytic thrombocytopenia. Case Rep Hematol. 2014. 2014; Accessed 10/26/2015.
  2. Patel M, Kalra A, Surapaneni R, Schwarting R, Devereux L. Acquired amegakaryocytic thrombocytopenia in a patient with occupational chemical exposure. Am J Ther. January-February 2014; 21(1):e17-20.
  3. Thrombocytopenia. National Heart, Lung, and Blood Institute. September 2012; http://www.nhlbi.nih.gov/health/health-topics/topics/thcp.
  4. Thrombocytopenia. MedlinePlus. February 2014; https://www.nlm.nih.gov/medlineplus/ency/article/000586.htm.
  5. Bulcahndani D et al.,. Acquired pure megakaryocytic aplasia: Report of a single case treated with Mycophenolate Mofetil. American Journal of Hematology. 2007;
  6. Felderbauer P et al.,. Case Report: Acquired pure megakaryocytic aplasia: a seoarate haematological disease entity or a syndrome with multiple causes?. Eur J Haematol. 2004;