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目的 常规化疗对小儿晚期神经母细胞瘤效果欠佳 ,本文探讨CEM方案 (卡铂 +依托泊苷 +马法兰 )做预处理结合自体外周血干细胞移植治疗晚期神经母细胞瘤的疗效及毒副反应。方法 5例神经母细胞瘤患儿 ,Ⅲ期 2例 ,Ⅳ期 3例。肿瘤转移部位为骨 3例 ,骨髓 3例 ,肺转移 1例 ,肝转移 1例。移植时完全缓解 (CR) 4例 ,部分缓解 (PR) 1例。用化疗加重组人粒细胞集落刺激因子 (rhG CSF)动员。所采集单个核细胞 (MNC)为 (5 7～ 8 5 6 )× 10 8/kg。CD34+ 细胞为 (2 4 2～ 38 2 4 )× 10 6/kg。以CEM(马法兰 +卡铂 +依托泊苷 )作为预处理方案 ,未用TBI。结果 5例患儿成功地完成了动员、采集和回输 ,渡过了危险的骨髓抑制期。无严重毒副反应 ,无移植相关死亡。 1例患儿移植术后 11个月颅内转移外 ,余 4例患儿均存活 ,并能正常生活和学习。随诊时间分别为 8、10、36及 72个月。结论 CEM方案做预处理结合自体外周血干细胞移植治疗高危晚期神经母细胞瘤毒性小、复发率低 ,可明显改善其生存状况
Objective To evaluate the efficacy and toxicity of CEM regimen combined with autologous peripheral blood stem cell transplantation in the treatment of advanced neuroblastoma. Methods Five children with neuroblastoma were included in this study. There were 2 cases in stage Ⅲ and 3 cases in stage Ⅳ. The site of tumor metastasis was 3 cases of bone, 3 cases of bone marrow, 1 case of lung metastasis and 1 case of liver metastasis. Four patients had complete remission (CR) at transplantation and one patient had partial remission (PR). Chemotherapy with aggravated human granulocyte colony-stimulating factor (rhG CSF) mobilization. The collected mononuclear cells (MNC) were (57 ~ 856) × 10 8 / kg. CD34 + cells were (224 ~ 382 4) × 10 6 / kg. CEM (melphalan + carboplatin + etoposide) as a preconditioning regimen without TBI. Results Five children were successfully mobilized, collected, and reinfused, and survived the dangerous period of myelosuppression. No serious side effects, no transplant-related deaths. One patient had intracranial metastasis 11 months after transplantation, and the remaining four patients survived and were able to live and study normally. Follow-up time were 8,10,36 and 72 months. Conclusion CEM pretreatment combined with autologous peripheral blood stem cell transplantation for the treatment of late-stage neuroblastoma with low toxicity and low recurrence rate can significantly improve its survival