自体外周血造血干细胞移植在小儿恶性晚期实体肿瘤的临床应用(英文)

Effect of autologous peripheral blood stem cell transplantation on advanced malignant solid tumors in children

摘要:

目的 小儿晚期实体肿瘤对常规化疗效果欠佳 ,该文探讨大剂量化疗并自体外周血干细胞移植(APBSCT)治疗小儿高危晚期实体瘤的可行性及疗效。方法 1 3例恶性实体肿瘤患儿 (恶性淋巴瘤 7例、神经母细胞瘤 6例 ) ,在其完全缓解 (1 2例 ) ,部分缓解 (1例 )后进行了APBSCT治疗。移植时病程中位时间 1 0月。 1 1例用化疗加重组人粒 单细胞集落刺激因子 (rhGM CSF)或重组人粒细胞集落刺激因子 (rhG CSF)动员 ,2例采用常规化疗方案作为动员剂。所采集单个核细胞 (MNC)为 (6 .85± 2 .6 5 )× 1 0 8/kg。CD34+ 细胞为 (1 5 .82± 1 2 .93)×1 0 6/kg。CFU GM集落为 (1 7.87± 1 7.94 )个 / 1 0 4细胞。预处理方案中 6例基本方案为全身放疗加环磷酰胺。 7例未用TBI ,仅以马法兰为主做为预处理方案 (马法兰 +卡铂 +足叶乙甙 5例 ,白消胺 +马法兰 2例 )。结果 移植后白细胞 >0 .5× 1 0 9/L、>1 .0× 1 0 9/L、血小板 >2 0× 1 0 9/L的中位时间分别为 1 2天、1 5天、1 9天。中位随访时间4 8月 (1月～ 1 4 4月 )。至今总生存率 77% (1 0 / 1 3) ,死亡率 2 3% (3/ 1 3) ,无移植相关死亡。结论 APBSCT是治疗小儿晚期实体肿瘤 ,明显改善其预后的重要治疗方法。

Abstract:

Objective The advanced solid tumor in children is not responsive to regular chemotherapy. This paper aims at studying the feasibility and effect of high dose chemotherapy combined with autologous peripheral stem cell transplantation (APBSCT) in the treatment of high risk advanced solid tumors in children. Methods Thirteen patients, including 7 cases of malignant lymphoma and 6 cases of neuroblastoma, were given APBSCT after receiving complete remission (CR) or partial remission (PC). The median disease duration before transplantation was 10 months. Before transplantation, 12 patients had CR and 1 had PR. Mobilization of stem cells was performed with chemotherapy plus G CSF or GM CSF in 11 and chemotherapy alone in 2 patients. The collecting mean number of MNCs, CD34 + cell and CFU GM was ( 6.85 ± 2.65 )×10 8/kg, ( 15.82 ± 12.93 )×10 6/kg and 17.87 ± 17.94 colons/10 4 cell respectively. The conditioning regimen consisted of cyclophosphamide plus TBI as basic program in 6 patients, melphalan plus etoposide, carboplatin in 5 patients and busulfan plus melphalan in 2 patients. Results The median times of the amount of WBC being more than 0.5 ×10 9/L and 1.0 ×10 9/L and of thrombocytopenia being more than 20×10 9/L were 12, 15 and 19 days respectively. A follow up of 48 months (ranging from 1 month to 144 months) showed that survival and death rates after transplantation were 77% (10/13) and 23% (3/13) respectively. No death due to the transplantation was found. Conclusion APBSCT can significantly improve the prognosis of patients with advanced solid tumors.