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Genetic and Rare Diseases Information Center (GARD)

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Thrombocytopenia, acquired amegakaryocytic

Other Names for this Disease
  • AAT
  • Acquired pure megakaryocytic aplasia
  • Severe thrombocytopenia and selective, marked decrease or absence of megakaryocytes
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Your Question

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

Our Answer

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What is acquired amegakaryocytic thrombocytopenia?

Acquired amegakaryocytic thrombocytopenia is characterized by severe thrombocytopenia (lack of platelets) with no other blood abnormalities.[1] It is so named because the level of the large bone marrow cells that produce platelets, called megakaryocytes, are significantly lower or absent. The course of the condition is variable, but the condition can become life-threatening. Its cause is currently unknown.[1]
Last updated: 6/10/2009

What causes acquired amegakaryocytic thrombocytopenia?

The exact cause of acquired amegakaryocytic thrombocytopenia is unknown. Exposure to toxins, drug sensitivity, infectious diseases, and genetic and immune cytotoxicity may play a role in some cases.[2] In some people the absent megakaryocytes are early signs of a more severe progressive disease, such as aplastic anemia or myelodysplasia.[2] This illustrates that there may be more than one underlying cause. This in turn makes it challenging to know how to predict the course of the disease.
Last updated: 6/10/2009

How is acquired amegakaryocytic thrombocytopenia treated?

Limited information is available regarding treatment approaches for acquired pure megakaryocytic aplasia. Treatment with the following medications (often used in combination) to suppress the immune system has been reported in the medical literature:[1][2]

Cyclosporine or mycophenolate mofetil
Antithymocyte globulin (ATG) or antilymphocyte globulin (ALG)

Some people with acquired pure megakaryocytic aplasia have responded well to immunosuppression, however failure with this treatment is not uncommon. Non-response to treatment may indicate that the absent megakaryocytes are an early sign of a more serious progressive disease. An alternative treatment to immune suppressive therapies (particularly for those with progressive disease) is bone marrow transplantation.[1][2]
Last updated: 6/10/2009

What is the prognosis (chance of recovery) of people with acquired amegakaryocytic thrombocytopenia?

While there is little information regarding the prognosis of people with acquired amegakaryocytic thrombocytopenia, the course of this condition has been described as “variable.”[1] Some people respond well to treatment and long term remissions have been documented in the literature. In others the condition progresses rapidly to a aplastic anemia or myelodysplasia.[1] There are currently no good predictors to aid in assessing the likelyhood of response to therapy or overall prognosis.[2]
Last updated: 6/10/2009

  • Bulcahndani D et al.,. Acquired pure megakaryocytic aplasia: Report of a single case treated with Mycophenolate Mofetil. American Journal of Hematology. 2007;
  • 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;