Bone marrow transplantation in children

From 1975 to 1987, 82 children underwent bone marrow transplantation at Huddinge Hospital. In 21 children transplanted for acute leukemia in first remission, or chronic myeloid leukemia in chronic phase with HLA‐identical marrow, the actuarial 5‐year leukemia‐free survival was 85% and the probability of relapse was 15%. These survival figures were better when compared to a 30% probability of relapse‐free survival (p < 0.001) and a 52% relapse rate (p = 0.01) in 27 children undergoing marrow transplantation for leukemia at later stages. Seven out of 9 children with severe aplastic anemia are alive from 3 to 9 yr after transplantation. Successful marrow transplantations were performed in 3 children with Gaucher disease and a child with a T‐cell defect. An attempt to induce tolerance in a patient with Hurler's disease by infusion of mixed T cell‐depleted autologous and allogeneic marrow resulted in autologous recovery. Twenty children received marrow from HLA‐mismatched donors. One out of 2 mismatched recipients with Fanconi anemia is alive and well. Recipients of HLA‐mismatched marrow had an increased death rate by graft‐versus‐host disease (GVHD) (p < 0.01) and a decreased survival (p < 0.001) compared to recipients of HLA‐matched marrow. HLA‐mismatched patients treated with a combination of methotrexate and cyclosporin had a decreased incidence of grade II‐IV acute GVHD (0/7) compared with those treated with either methotrexate or cyclosporin (7/11, p = 0.01). Late side‐effects of total‐body‐irradiation included cataract, delayed growth and puberty, and thyroid gland deficiency.