Severe acquired aplastic anemia in children: 11-year experience with bone marrow transplantation and immunosuppressive therapy.

We reviewed the management and outcome of severe acquired aplastic anemia in 36 patients younger than 18 years old between March 1977 and June 1987. In most patients, no cause could be found. The best therapeutic results were achieved with allogeneic bone marrow transplantation from histocompatible related donors (14 patients), with a cure rate of 79%. Bone marrow transplantation from mismatched related donors was attempted in two patients and failed in both. Immunosuppression using antithymocyte globulin and/or high-dose methylprednisolone represented an alternative treatment in the absence of a suitable bone marrow donor (12 patients) and induced complete remission in 25% of the patients. Children treated with supportive care only (eight patients) had a 75% mortality with a very short survival time. These data confirm the superiority of allogeneic bone marrow transplantation over immunosuppressive therapy in children with severe aplastic anemia.