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

A bone marrow transplant (BMT) protocol including surgical excision, l ocal and total body irradiation, and high dose multiagent chemotherapy based on melphalan and bone marrow rescue has been in effect for chil dren with high risk or relapsed neuroblastoma at the Children's Hospit al of Philadelphia since 1979. The initial results were reported in 19 84 [August et al.: J Clin Oncol 2:609-616,1984]. This report updates t he initial results and those that followed changes in the original con ditioning regimen. Forty-two patients were treated between May 1979 an d November 1987, and included 27 whose disease had relapsed and 15 who received BMT as part of primary treatment. Allogeneic marrow was give n 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 actua rial survival rate is 29%. Ten patients died of early treatment-relate d complications, 18 died of progressive disease, and 2 died of late co mplications (1 AIDS and 1 acute myelogenous leukemia). Censoring the t wo 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 trans planted following relapse or in first remission. The better survival f or patients rescued with autologous marrow (30%) is not statistically significantly different from the result with allogeneic marrow (17%). (C) 1994 Wiley-Liss, Inc.