OBJECTIVETo investigate the efficacy of high dose chemotherapy combined with autologous peripheral blood stem cell transplantation (APBSCT) for the treatment of neural ectodermal solid tumor originated from neural crest in children.

METHODSTwenty-three children at a medium age of 5.8 + or - 3.5 years with neural ectodermal solid tumor originated from neural crest were enrolled. Of the 23 children, 20 with stage IV neuroblastoma (9 were in complete remission, 7 were in partial remission and 4 were in progressive disease), 2 with stage IV primitive neuroectodermal tumor (PNET) in complete remission, and 1 with retinoblastoma in partial remission. Before APBSCT the children received 8.0 + or - 4.3 courses of chemotherapy. During chemotherapy the autologous peripheral blood stem cells were harvested and the tumor excision was performed. Then APBSCT was performed.

RESULTSThe reconstruction of the hematopoietic system was noted in 19 of 20 children with stage IV neuroblastoma 16.5 + or - 0.9 days after transplantation. A follow-up (median 15.8 months) was done in these children. The follow-up showed that the survival rate in children in complete remission before transplantation was 100%, 57% in those in partial remission, and none of children in progressive disease survived (P<0.05). The total survival rate was 67% in children with neuroblastoma. The child with retinoblastoma had complete remission in a 6-months follow-up. The tumors recurred in children with PNET 5 to 8 months after transplantation and all died within one year after transplantation.

CONCLUSIONSHigh dose chemotherapy combined with APBSCT can result in a good outcome in children with neural ectodermal solid tumor originated from neural crest in complete remission before transplantation and can improve the outcome in patients in partial remission before transplantation. However, the children with PNET, even in complete remission before transplantation, do not respond to the therapy.