Abstract
Between 1/91 and 12/96, total of 33 children with acquired aplastic anemia received immunosuppressive therapy (IS) (n = 23) or a matched bone marrow transplant (BMT) (n = 11) in a single institution. One child failed IS therapy and was rescued by BMT.
Etiology was idiopathic in 23 children, hepatitis in 9 and drug-induced in one patient. Nineteen children suffered from severe aplastic anemia, 10 children from very severe aplastic anemia and 4 children from non-severe aplastic anemia.
In 9 children an allogeneic BMT from HLA-identical sibling was performed. In one case the donor was monozygotic twin and in one HLA identical mother respectively.
Nineteen children received IS therapy consisted of antilymphocyte globulin (ALG) or antithymocyte globulin (ATG) + cyclosporine A + prednisone + G-CSF/GM-CSF. In 4 patients with non-severe aplastic anemia only cyclosporine A was used.
With median follow-up of 3.3 years (1 - 6.8 y) 28 (85%) children are alive and transfusion independent. Probability of 5-years survival is 91% in BMT group and 82% in IS group of children.
Seven (25%) children suffered from relapse of aplastic anemia and all achieved remission by IS (n = 6) or BMT (n = 1) therapy. Five children died due to the complications of the disease or the therapy.
In contrast, only 32% of patients are alive from historical group treated by the help of IS therapy at the same institution in 1980s. Bone marrow transplantation and the progress in the supportive therapy offer the chance of cure for the majority of children with this rare and potentially fatal disorder.