大剂量化疗结合自体外周血干细胞移植治疗神经外胚层来源的儿童实体瘤疗效分析

Therapeutic effects of high dose chemotherapy combined with autologous peripheral blood stem cell transplantation for neural ectodermal solid tumor originated from neural crest in children

摘要:

目的：探讨大剂量化疗结合自体外周血造血干细胞移植（APBSCT）治疗神经外胚层来源晚期实体瘤的疗效。方法：选择神经外胚层来源的肿瘤患儿23例，平均年龄5.8±3.5岁。移植前化疗8.0±4.3疗程。其中IV期神经母细胞瘤20例，移植时完全缓解9例，部分缓解7例，处于肿瘤进展期4例；原始神经外胚叶肿瘤2例，移植期间为完全缓解。视网膜母细胞瘤1例，移植时部分缓解。进行外周血造血干细胞采集、手术切除，然后进行APBSCT。结果：①20例神经母细胞瘤患儿中，19例移植后造血功能获得满意重建，重建时间为16.5±0.9 d。术后中位随访时间为15.8月。移植前获完全缓解的患儿生存率为100%，部分缓解患儿为57%，进展期患儿为0（P<0.05）。3组15.8月总生存率67%。②视网膜母细胞瘤患儿随访6个月获完全缓解。③原始神经外胚叶肿瘤患儿于术后5~8个月原发灶复发，1年内均死亡。结论：大剂量化疗结合APBSCT治疗神经外胚层来源实体瘤，在移植前达到完全缓解者可取得较好疗效，部分缓解者可提高缓解率。其中原始神经外胚叶肿瘤预后最差，即使在完全缓解时移植死亡率仍高。［中国当代儿科杂志，2010，12（4）：244-247］

Abstract:

OBJECTIVE: To 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. METHODS: Twenty-three children at a medium age of 5.8±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±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. RESULTS: The reconstruction of the hematopoietic system was noted in 19 of 20 children with stage IV neuroblastoma 16.5±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. CONCLUSIONS: High 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,［Chin J Contemp Pediatr, 2010, 12 (4):244-247］