Bone marrow transplantation for severe aplastic anemia

Fifteen patients (nine male, six female) with severe aplastic anemia (SAA) undergoing HLA-indentical allogeneic bone marrow transplantation (BMT) received preparative regimens consisting of cyclophosphamide and total lymphoid irradiation. Patients were aged from eight to 26 years (median 15 years). Prophylaxis of graft versus host disease (GVHD) including cyclosporine and short couse methotrexate was administered. One early death occurred at day 8 post BMT. Among the other 14 patients, one died of sepsis at day 53 with no evidence of engraftment, 13 were engrafted despite the number of donors exposed in transfusions of previous blood components. Eleven patients have survived from six to 100 months (median, 54 months), post BMT. The remaining two patients died of acute GVHD-related infections on day 44 and 63. Acute GVHD occurred among eight of 13 engrafted patients, five of whom were grades II-IV clinically. Chronic GVHD developed among five patients, three of whom were clinically progresive and extensive. Two of three patients with extensive chronic GVHD had received transfusion of donor's buffy coat after BMT. Our data indicate an engraftment rate of 87% (13/15). The projected probability of disease-free survival was 73% (11/15) at 9.3 years after BMT according to the Kaplan-Meier model. Further efforts must be made to eliminate GVHD and to control fatal infections.