Encouraging results are reported with high-dose
chemotherapy and total body irradiation followed by autologous
bone marrow transplantation in the treatment of advanced
neuroblastoma. However, relapse remains a significant problem. We used high-dose
chemotherapy, surgery, intraoperative radiation and an autologous bone marrow transplant treated in vitro to remove
tumor cells followed by
13-cis-retinoic acid to treat 36 children with advanced
neuroblastoma. This comprehensive treatment appears to improve the survival rate of patients with advanced
neuroblastoma, including those with N-myc amplification and bony involvement. The disease-free survival rate was 66% (95% confidence interval, 49-84%) at 3 years. All patients who received
13-cis-retinoic acid developed
cheilitis, but no bone marrow depression occurred in these patients. Five patients developed
hemolytic uremic syndrome (HUS) post-transplant. This may have been related to the procedure used for total body irradiation. Patients who had their kidneys shielded during this procedure did not develop this syndrome. Patients who received local irradiation at the primary site showed no evidence of relapse in this region, indicating that such
therapy may help to prevent a relapse. These data suggest a high rate of 3 year disease-free survival with this treatment strategy. The nonrandomized nature of the study and use of multiple modalities precludes analysis of the specific contribution of each.