Cyclosporin A in severe aplastic anemia in children

Severe aplastic anemia should be treated with bone marrow transplantation if possible. Various clinical and experimental data support the view that the major pathogenetic defect in SAA is a dysregulated cellular immune response which in turn has a negative effect upon hematopoiesis. Therefore a large percentage of patients react favorably to treatment with immunosuppressive agents as antithymocyte globulin and high-dose methylprednisolone. Data concerning the efficiency of Cyclosporine A treatment are limited until now. We present in this report our own experience with Cyclosporine A treatment in three children with severe aplastic anemia who lack a bone marrow donor. Included is also a review of the present therapeutic possibilities.