CEM为预处理方案的自体外周血造血干细胞移植治疗晚期神经母细胞瘤相关毒性及疗效观察

Curative effects and regimen-related toxicity of CEM chemotherapy as a conditioning regimen in autologous peripheral blood stem cell transplantation for the treatment of high-risk neuroblastoma

目的 观察以CEM (卡铂 +VP 16 +马法兰 )为预处理方案的自体外周血造血干细胞移植治疗晚期神经母细胞瘤患儿的毒性和疗效。方法 研究经大剂量放、化疗及手术治疗达完全缓解的IV期神经母细胞瘤患儿 6例 ,采用CEM预处理方案 (卡铂每日 4 2 5mg/m2 共 4d ,VP 16每日 338mg/m2 共 4d ,马法兰每日 70mg/m2 共 3d)的自体外周血造血干细胞移植 ,并依据Bearman标准对预处理毒性进行评价 ,对其造血重建、并发症及预后进行观察。结果 预处理后髓外毒性除 1例为I级肝损害外 ,主要表现在口腔粘膜 (I级 6例 )和胃肠道 (I级 5例 ,II级 1例 )损害。白细胞于移植后 3± 0 .5d达到 0 ,移植后 33± 3.1d稳定于 1× 10 9/L以上。除 1例患儿移植后 11个月颅内转移外 ,余 5例患儿均健康存活。随诊时间分别为 8、10、2 3、36及 4 8个月。结论 以CEM为预处理方案的自体外周血造血干细胞移植可以安全、有效的治疗IV期神经母细胞瘤。

Objective To evaluate the effects and regimen-related toxicity (RRT) of CEM (carboplatin, etoposide, melphalan) chemotherapy as a conditioning regimen in autologous peripheral blood stem cell transplantation (APBSCT) in children with high-risk neuroblastoma. Methods Six patients in stage IV neuroblastoma, who had gotten a complete remission after receiving high-dose chemotherapy, radiation and surgery were enrolled in this study. All the patients received CEM chemotherapy (carboplatin 425 mg/m 2 per day for 4 days; etoposide 338 mg/m 2 per day for 4 days; melphalan 70 mg/m 2 per day for 3 days) as a conditioning regimen in APBSCT. Transplantation related complications were observed and RRT was graded according to Bearman's proposal. Results The RRT was principally manifested as stomatitis (grade I in 6 cases) and gastrointestional symptoms (grade I in 5 cases and grade II in 1), and only 1 case had RRT in liver (grade I). The RRT symptoms were improved about 16 days after transplantation. The amount of white blood cells was recovered to over 1×10 9/L after about 33 days of transplantation. The follow-up visit (ranging from 8 months to 48 months ) revealed that five patients had event free survival and 1 died due to brain metastasis after APBSCT. Conclusion CEM chemotherapy as a conditioning regimen in APBSCT might be effective and safe for children with high-risk neuroblastoma.