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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. Both drugs are used in off-label applications, especially as immunosuppression induction agents before and/or during kidney transplantation.
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]
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]
Two types of Congenital amegakaryocytic thrombocytopenia have been identified with type I being more severe. [1] Treatment is mostly supportive, consisting of multiple platelet transfusions. Hematopoietic stem cell transplantation is the only known cure for CAMT. [1] Once pancytopenia develops, the prognosis is poor.
Pure red cell aplasia (PRCA) or erythroblastopenia refers to a type of aplastic anemia affecting the precursors to red blood cells but usually not to white blood cells. In PRCA, the bone marrow ceases to produce red blood cells. There are multiple etiologies that can cause PRCA. The condition has been first described by Paul Kaznelson in 1922. [1]
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One in ten individuals with bone marrow failure have unsuspected Fanconi anemia (FA). [14] FA is the most common inherited bone marrow failure with an incidence of one to five episodes per million individuals. [14] The carrier frequency for FA is 1 in 200 to 300, however this differs by ethnicity. [14]