BONE-MARROW TRANSPLANTATION FOR HIGH-RISK NEUROBLASTOMA AT THE CHILDRENS-HOSPITAL-OF-PHILADELPHIA - AN UPDATE

A bone marrow transplant (BMT) protocol including surgical excision, local and total body irradiation, and high dose multiagent chemotherapy based on melphalan and bone marrow rescue has been in effect for children with high risk or relapsed neuroblastoma at the Children's Hospital of Philadelphia since 1979. The initial results were reported in 1984 [August et al.: J Clin Oncol 2:609-616,1984]. This report updates the initial results and those that followed changes in the original conditioning regimen. Forty-two patients were treated between May 1979 and November 1987, and included 27 whose disease had relapsed and 15 who received BMT as part of primary treatment. Allogeneic marrow was given to 12 and autologous marrow to 30; in 7 of these 30, the marrow was purged with monoclonal antibodies and magnetic beads. The 4-year actuarial survival rate is 29%. Ten patients died of early treatment-related complications, 18 died of progressive disease, and 2 died of late complications (1 AIDS and 1 acute myelogenous leukemia). Censoring the two late complications the actuarial 4-year relapse-free survival rate becomes 32%. The longest interval after BMT to relapse was 20 months. There was no significant difference in the survival for patients transplanted following relapse or in first remission. The better survival for patients rescued with autologous marrow (30%) is not statistically significantly different from the result with allogeneic marrow (17%). (C) 1994 Wiley-Liss, Inc.