Abstract:OBJECTIVE: Neuroblastoma is a highly malignant tumor.Stage IV neuroblastoma has a very poor long-term outcome by conventional chemotherapy and surgery and better therapies are essential. This study aimed to explore the long-term effect of high dose induction chemotherapy combined with autologous peripheral blood stem cell transplantation and 13-cis retinoid acid treatment on stage IV neuroblastoma in children. METHODS: Twenty-eight children with stage IV neuroblastoma,aged 2.1-11.5 years(mean 3.3±1.9 years),were employed for the study.Primary sites of the tumors included adrenal(n=23), chest(n=3), chest-abdomen(n=1) and sacrum(n=1).Before autologous peripheral blood stem cell transplantation the patients received 6 courses of intensive induction chemotherapy. During chemotherapy the autologous peripheral blood stem cells were harvested and the tumor excision was done. After transplantation the local radiation and 13-cis retinoid acid therapy were administered. RESULTS: After 6 courses of induction chemotherapy 13 patients got complete remission(CR), 11 got partial remission(PR), and 4 had no response. The 24 patients who received CR or PR completed the full therapy.A 3.5±0.7 years follow-up showed that the 4-year event-free survival of the CR and PR patients was 29.2%.The median no-relapse survival time in CR patients was 4.1±0.7 years but 2.8±0.5 years in PR patients(t= 3.9, P<0.01). CONCLUSIONS: High dose chemotherapy combined with autologous peripheral stem cell transplantation and 13 cis-retinoid acid treatment can improve the long-term outcome of patients with stage IV neuroblastoma. The patients in CR before transplantation had better outcomes than those in PR.
