Aplastic anemia medical therapy

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Editor-In-Chief: Aric Hall, M.D., Beth Israel Deaconess Medical Center, Boston, MA [1]

Overview

Medical therapy of aplastic anemia often includes a short course of anti-thymocyte globulin (ATG or anti-lymphocyte globulin) and several months of treatment with cyclosporin to modulate the immune system. Mild chemotherapy with agents such as cyclophosphamide and vincristine may also be effective. Antibodies therapy, such as ATG, targets T-cells, which are believed to attack the bone marrow. Steroids are generally ineffective.

Supportive Care in treatment of aplastic anemia (DONOT EDIT)

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Supportive Care Prophylactic platelet transfusions should be given when the platelet count is <10 x 109/L (or <20 x 109/L in the presence of fever). Irradiated blood products should be given routinely to all patients having antithymocyte globulin (ATG) treatment. Transfusion of irradiated granulocyte transfusions may be considered in patients with life-threatening neutropenic sepsis. The routine use of recombinant human erythropoietin (rHuEPO) in aplastic anaemia is not recommended. A short course of granulocyte colony-stimulating factor (G-CSF) may be considered for severe systemic infection that is not responding to intravenous antibiotics and anti-fungal drugs, but should be discontinued after 1 week if there is no increase in the neutrophil count. Prophylactic antibiotic and antifungal drugs should be given to patients with neutrophil count <0.2 x 109/L. Systemic antifungal therapy should be introduced into the febrile neutropenia regimen early if fevers persist. Iron chelation therapy should be considered when the serum ferritin is >1000 µg/L.

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Specific Treatment of Aplastic Anaemia: General Comments (DONOT EDIT)

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General Comments Infection or uncontrolled bleeding should be treated first before giving immunosuppressive therapy. This also applies to patients scheduled for bone marrow transplant (BMT), although it may sometimes be necessary to proceed straight to BMT in the presence of severe infection as a BMT may offer the best chance of early neutrophil recovery. Haemopoietic growth factors, such as rHuEPO or G-CSF, should not be used on their own in newly diagnosed patients in an attempt to 'treat' the aplastic anaemia. Prednisolone should not be used to treat patients with aplastic anaemia because it is ineffective and encourages bacterial and fungal infection.

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