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.