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Two antithymocyte globulin (ATG) agents licensed for clinical use in the United States are Thymoglobulin (rabbit ATG, rATG, Genzyme) and Atgam (equine ATG, eATG, Pfizer). Thymoglobulin and Atgam are currently licensed for use in the treatment of renal allograft rejection; Atgam is additionally licensed for use in the treatment of aplastic anemia.
Aplastic anemia [2] (AA) [3] is a severe hematologic condition in which the body fails to make blood cells in sufficient numbers. Blood cells are produced in the bone marrow by stem cells that reside there. [4] Aplastic anemia causes a deficiency of all blood cell types: red blood cells, white blood cells, and platelets. [5] [6]
While acquired aplastic anemia with an unknown cause is rare, it is commonly permanent and life-threatening as half of those with this condition die within the first six months. [ 10 ] The prevalence of bone marrow failure is over three times higher in Japan and East Asia than in the United States and Europe. [ 10 ]
The treatments for cytopenia vary depending on the type of cytopenia. The treatment for anemia is rest and a diet consisting of high iron foods. Medication can also be used such as: [citation needed] Epoetin alfa, a synthetic erythropoietin that stimulates stem cells to produce red blood cells.
The goal is to treat the underlying condition if it can be identified and provide supportive care. If symptomatic anemia develops, blood products may be replaced. Disease specific management may include glucocorticoids, IVIG, immunosuppressive agents, stem cell transplant, or other treatments depending on the etiology of reticulocytopenia. [7]
Candidates for HSCTs include pediatric cases where the patient has an inborn defect such as severe combined immunodeficiency or congenital neutropenia with defective stem cells, and also children or adults with aplastic anemia [17] who have lost their stem cells after birth.
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