483例神经母细胞瘤基于危险因素分层治疗结果分析  

Outcome analysis of neuroblastoma based upon risk-adapted therapy:a report of 483 cases

作　　者：孙晓非[1] 甄子俊[1] 王娟[1] 高远红[2] 刘卓炜[3] 朱佳[1] 张玉[4] 路素英[1] 孙斐斐[1] 黄俊廷[1] Sun Xiaofei;Zhen Zijun;Wang Juan;Gao Yuanhong;Liu Zhuowei;Zhu Jia;Zhang Yu;Lu Suying;Sun Feifei;Huang Junting(State Key Laboratory of Oncology in South China,Department of Pediatric Oncology,Sun Yat-Sen University Cancer Center,Guanghzou 510060,China;State Key Laboratory of Oncology in South China,Department of Radiotherapy,Sun Yat-Sen University Cancer Center.Guanghzou 510060,China;State Key Laboratory of Oncology in South China,Department of Urology Surgery,SunYat-Sen University Cancer Center.Guanghzou 510060,China;State Key Laboratory of Oncology in South China,Department of Pathology,Sun Yat-Sen University Cancer Center.Guanghzou 510060,China)

机构地区：[1]华南肿瘤学国家重点实验室,中山大学肿瘤防治中心儿童肿瘤科,广州510060 [2]华南肿瘤学国家重点实验室,中山大学肿瘤防治中心放疗科,广州510060 [3]华南肿瘤学国家重点实验室,中山大学肿瘤防治中心泌尿外科,广州510060 [4]华南肿瘤学国家重点实验室,中山大学肿瘤防治中心病理科,广州510060

出　　处：《中华小儿外科杂志》2022年第3期193-200,共8页Chinese Journal of Pediatric Surgery

摘　　要：目的评估和分析神经母细胞瘤(neuroblastoma,NB)患儿采用危险因素分层治疗的结果。方法收集2002年9月至2018年12月中山大学肿瘤防治中心收治的483例采用危险因素分层治疗的初诊为NB患儿的临床资料。其中,男290例[60.0%(290/483)],女193例[40.0%(193/483)];患儿的中位年龄为3.48岁,年龄范围为7 d至18岁;按国际NB分期系统分期,1期19例,2期25例,3期95例,4期334例,4S期10例。按照年龄、临床分期、病理类型和N-MYC基因状态分为低危、中危和高危组。低危组46例[9.5%(46/483)],中危组102例[21.1%(102/483)],高危组335例[69.4%(335/483)]。低危组采用手术+观察或手术+低强度化疗4~6个疗程的治疗方案;中危组采用手术+中等强度化疗6~8个疗程+放疗(按需实施)的治疗方案;高危组2013年前采用"环磷酰胺、长春新碱、吡柔比星"与"顺铂、依托泊苷"方案交替化疗8个疗程+手术+自体造血干细胞移植(autologous hematopoietic stem cell transplantation,ASCT)(按需实施)+放疗+生物治疗(按需实施)+异维甲酸维持治疗1年的治疗方案;2013年后采用"环磷酰胺、长春新碱、吡柔比星"与"依托泊苷、异环磷酰胺、顺铂"方案交替化疗8个疗程+手术+放疗+生物治疗(按需实施)+口服化疗药物维持治疗1年的治疗方案。复发患儿采用再次手术、化疗或放疗等挽救治疗。结果全组322例行N-MYC基因检测,N-MYC基因扩增阳性率为22.4%(72/322),高危组N-MYC基因扩增阳性率为33.8%(72/213)。高危患儿行ASCT仅15例,占4.5%(15/335)。中位随访时间为38.1个月,范围在2.5~208.0个月。低危组3年无事件生存率(event free survival,EFS)和总生存率(overall survival,OS)分别为(86.5±5.1)%和100%,中危组为(79.1±4.3)%和(89.2±3.3)%,高危组为(30.6±2.7)%和(60.3±2.9)%。335例高危组单因素和多因素生存分析显示,临床分期4期、单纯化疗和(或)腹部原发病灶是影响EFS和OS的独立不良预后因素(P均<0.05)。N-MYC基因�Objective To evaluate the results of neuroblastoma(NB)based upon risk-adapted therapy.Methods From September 2002 to December 2018,clinical data were reviewed for 483 newly diagnosed NB patients aged≤18 years based on risk-adapted therapy.They were stratified into low,intermediate and high-risk groups by age,stage,histological category and MYCN status.There were 290 males and 193 females with a median diagnostic age of 3.5 years(7 days to 18 years).The stages were 1(n=19),2(n=25),3(n=95),4(n=334)and 4S(n=10).Surgery alone or surgery plus low intensive chemotherapy for low-risk group(n=46,9.5%);Surgery plus moderate intensive chemotherapy with or without radiotherapy for intermediate-risk group(n=102,21.1%);For high-risk group(n=335,69.4%),before 2013,the treatment strategies included 8 cycles of alternating chemotherapy of cyclophosphamide(CTX)+vincristine(VCR)+pirarubicin(THP)and cisplatin(DDP)+etoposide(VP16),surgery,with or without autologous hematopoietic stem cell transplantation(ASCT),radiotherapy,with or without biotherapy and isotretinoin maintenance therapy for 1 year;after 2013,8 cycles of alternating chemotherapy of CTX+VCR+THP and VP16+ifosfamide+DDP protocol,surgery,radiotherapy,with or without biological therapy and oral maintenance chemotherapy drugs for 1 year.Recurrent patients received salvage therapy.Results MYCN amplification was 22.4%(72/322)in all groups and 33.8%(72/213)in high-risk group.ASCT was performed for only 15 cases.During a median follow-up period of 38(2.5-208)months,3-year event-free survival(EFS)and overall survival(OS)were(86.5±5.1)%and 100%for low-risk group;(79.1±4.3)%and(89.2±3.3)%for intermediate-risk group;(30.6±2.7)%and(60.3±2.9)%for high-risk group respectively.Univariate survival analysis of 335 high-risk patients indicated that stage 4 and chemotherapy alone were poor prognostic factors for EFS/OS.Multivariate survival analysis showed that stage 4 and chemotherapy alone were independent adverse prognostic factors for EFS in high-risk NB patients while stage 4,c

关 键 词：神经母细胞瘤 危险因素 治疗 

分 类 号：R739.4[医药卫生—肿瘤]