目的：目前IV期神经母细胞瘤患儿无论采用何种方法治疗均疗效差,长期生存率低,需要探索新的治疗途径。该文采用大剂量化疗、自体外周血造血干细胞移植及13-顺式维甲酸治疗等方法,试图提高IV期神经母细胞瘤的长期疗效。方法：选择IV期神经母细胞瘤患儿28例,年龄2.1~11.5岁,平均3.3±1.9岁,发病时间1~7个月,平均3.1±0.7个月。原发部位:肾上腺23例,胸部3例,胸腹联合1例,骶骨1例。强烈化疗6疗程,期间进行外周血造血干细胞采集、手术切除,然后进行自体外周血造血干细胞移植,术后行局部放疗及13-顺式维甲酸治疗,定期随访。结果：28例患儿诱导化疗结束时13例取得完全缓解,11例取得部分缓解,4例化疗中病情进展。完全缓解及部分缓解的24例患儿完成治疗进入本研究。随访3.5±0.7年,两组4年无病生存率29.2%。完全缓解组中位无复发生存时间4.1±0.7年;部分缓解组中位无复发生存时间2.8±0.5年,两组中位无复发生存时间差异有显著性(t=3.9,P<0.01)。结论：大剂量化疗、自体外周血造血干细胞移植及13-顺式维甲酸治疗IV期神经母细胞瘤可取得较好疗效,4年无病生存率29.2%,移植前达到完全缓解时可取得更好疗效

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.