Aplastic anemia in younger patients is generally immune mediated and is treated with immunosupression. When there is no sibling donor, cyclosporine(CSA) is recommended, usually in combination with other drugs but it can also be used alone(1). First-line treatment approaches include immunosuppressive treatment using the combination of antithymocyte globulin and cyclosporine A for patients without a sibling donor and HLA identical sibling transplant for patients younger than age 40 with a donor(2). Despite a theoretical rationale for its use, sirolimus did not improve the response rate in patients with severe aplastic anemia when compared to standard h-ATG/CsA(3). CsA is usually dosed on day 1 to a target trough level between 200 and 400 ng/mL, starting at a dose of 10 mg/kg per day (in children, 15 mg/kg per day).
2. Jakob R. Passweg et al, Aplastic Anemia: First-line Treatment by Immunosuppression and Sibling Marrow Transplantation ASH Education Book December 4, 2010 vol. 2010 no. 1 36-42
3.Scheinberg P, Wu CO, Nunez O, et al.(2009) Treatment of severe aplastic anemia with a combination of horse antithymocyte globulin and cyclosporine, with or without sirolimus: a prospective randomized study. Haematologica 94:348354.
4.Phillip Scheinberg1and Neal S. Young, .How I treat acquired aplastic anemia Blood August 9, 2012 vol. 120 no. 6 1185-1196
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Promacta for Aplastic Anemia
Red cell growth factors for Aplastic Anemia