COG方案大剂量化疗联合串联自体造血干细胞移植治疗高危神经母细胞瘤的疗效及安全性  

Efficacy and safety of high-dose chemotherapy with COG regimen combined with serial autologous hematopoietic stem cell transplantation in the treatment of high-risk neuroblastoma

作　　者：程杰 陈天平 高姗 路幼佳 刘野天 屈丽君 汪俭 刘洪军 CHENG Jie;CHEN Tianping;GAO Shan;LU Youjia;LIU Yetian;QU Lijun;WANG Jian;LIU Hongjun(Department of Hematology and Oncology,Anhui Provincial Children's Hospital(Anhui Hospital,Pediatric Hospital of Fudan University),Anhui Hefei 230051,China)

机构地区：[1]安徽省儿童医院血液肿瘤科,复旦大学附属儿科医院安徽医院,安徽合肥230051

出　　处：《现代肿瘤医学》2024年第9期1694-1698,共5页Journal of Modern Oncology

基　　金：安徽省自然科学基金项目(编号:2108085MH268)。

摘　　要：目的:探讨以北美儿童肿瘤专业组(Children's Oncology Group,COG)方案大剂量化疗(high-dose chemotherapy,HDC)联合串联自体造血干细胞移植(autologous hematopoietic stem cell transplantation,ASCT)治疗儿童高危神经母细胞瘤(neuroblastoma,NB)的安全性和疗效。方法:研究纳入自2022年07月至2023年06月于我科接受ASCT治疗的9例高危NB患者,均按照COG高危NB自体移植方案给药,首次移植预处理采用大剂量塞替派(thiotepa,TT)联合环磷酰胺(cyclophosphamide,CTX)为预处理方案,第二次移植预处理给予大剂量美法兰(melphalan,Mel)、卡铂(carboplatin,CBP)联合依托泊苷(etoposide,VP16)方案(CEM方案),移植后分别对其造血重建、并发症及疗效进行观察评估。结果:9例均为男孩,发病时大多伴多发骨转移,其中Ⅳ期8例,Ⅲ期1例,发病时除1例未检外其余8例至少均有2种分子遗传学异常;第一次移植预处理后,髓外不良反应主要表现为口腔黏膜炎、腹泻和呕吐等。中性粒细胞植入平均时间为10.44天,血小板(platelet,PLT)植入中位时间为15天。同一组患者接受串联第二次移植时与第一次移植时植入速度、不良反应的发生程度等无明显统计学差异。中位随访时间330天,至观察期末均无进展生存,疾病评估较移植前好转。结论:以COG方案HDC联合串联ASCT治疗儿童高危NB安全性好,疗效佳,双次移植较单次移植后观察到了更好的临床疗效,仍需扩大样本量延长观察时间,以确定该治疗策略的长期疗效。Objective:To investigate the safety and efficacy of high-dose chemotherapy(HDC)based preconditioning combined with tandem autologous hematopoietic stem cell transplantation(ASCT)in the treatment of high-risk neuroblastoma(NB)in children according to the regimen of North American Children's Oncology Group(COG).Methods:This study included in 9 children with high-risk NB treated with ASCT in our department from July 2022 to June 2023,all of whom were treated according to COG high-risk NB autologous transplantation protocol.The first transplantation received high-dose thiotepa(TT)combined with cyclophosphamide(CTX)followed by ASCT.High-dose melphalan(Mel)and carboplatin(CBP)combined with etoposide(VP16)regimen(CEM regimen)was administered in the second transplantation pretreatment.Hematopoietic reconstruction,complications and efficacy were observed and evaluated after transplantation.Results:All the 9 cases were boys,and most of them were accompanied by multiple bone metastases,including 8 cases in stageⅣand 1 case in stageⅢ,8 patients had at least two kinds of molecular genetic abnormalities except one who didn't receive the examination for some reason.The extramedullary side effects after the first conditioning regimen were mainly oral mucositis,diarrhea,and vomiting.The mean time of neutrophil implantation was 10.44 days,and the median time of platelet implantation was 15 days.There was no significant statistical difference in the rate of implantation and the degree of adverse reactions between the patients receiving the second tandem transplantation and the first transplantation in the same group.The median follow-up time was 330 days,and the PFS was 100%to the end of the observation period,and the disease evaluation was better than that before transplantation.Conclusion:COG protocol HDC combined with ASCT in the treatment of high-risk NB in children is safety.Better clinical efficacy was observed after double transplantation than after single transplantation.It is still necessary to expand the sample size a

关 键 词：高危神经母细胞瘤 大剂量化疗 预处理方案 自体造血干细胞移植 串联移植 

分 类 号：R739.4[医药卫生—肿瘤]