BMT is used with increased frequency to treat aplastic anemia (AA) and acute leukemia (AL). We report the results of 90 BMT performed on children at UCLA through June, 1980. Thirty-three BMT were done on previously transfused AA patients; 18 recipients survive (55%). In 17 of these 33 patients, low-dose (3Gy) total body irradiation (TBI) was added to the cyclophosphamide conditioning regimen (CY) to decrease the risk of graft rejection. Thirteen of these patients survive (76%, median = 791 + d). Failure to use TBI was associated with a 43% rejection rate, while use of 10Gy TBI was associated with an 83% risk of fatal graft versus host disease (GVHD). Fifty-seven patients had BMT for AL. Three of 34 patients transplanted in relapse survive (>3.5 y). There were 6 early deaths; 17 of the remaining 28 patients developed GVHD (16 fatal), and 9 of the remaining 12 patients relapsed. Twenty-three had BMT during remission: there were 2 early deaths, 6 cases of fatal GVHD and 6 patients relapsed. Nine patients survive (39%, median = 264 + d). No relapses occurred among 6 patients who had BMT for AML in first remission, using a matched sibling donor.
We conclude that BMT is the treatment of choice for children with severe AA, if a compatible sibling donor is available. Addition of low-dose TBI to CY improves the survival of previously transfused AA patients. BMT offers the possibility of prolonged disease-free survival to patients with ultra-high-risk AL.