儿童及青少年横纹肌肉瘤多中心临床研究——CCCG-RMS-2016中期研究报告  

Multicenter clinical study of rhabdomyosarcoma in children and adolescents——An interim report of CCCG-RMS-2016 protocol

作　　者：段超[1] 张伟令[2] 孙青[3] 刘江华 马典庆 徐艳丽[6] 郭雪梅 彭晓敏[8] 陈开澜[9] 赵艳霞[10] 徐娜 李静[2] 孙立荣[10] 李建新[9] 黎阳[8] 方拥军[7] 袁晓军[6] 屈丽君 刘炜 赵卫红[3] 黄东生[2] 倪鑫[1] 马晓莉[1] DUAN Chao;ZHANG Weiling;SUN Qing;LIU Jianghua;MA Dianqing;XU Yanli;GUO Xuemei;PENG Xiaolin;CHEN Kailan;ZHAO Yanxia;XU Na;LI Jing;SUN Lirong;LI Jianxin;LI Yang;FANG Yongjun;YUAN Xiaojun;QU Lijun;LIU Wei;ZHAO Weihong;HUANG Dongsheng;NI Xin;MA Xiaoli(Medical Oncology Department, Pediatric Oncology Center, Beijing Children′s Hospital, Capital Medical University, National Center for Children′s Health, Beijing Key Laboratory of Pediatric Hematology Oncology, Key Laboratory of Major Disease in Children, Ministry of Education, Beijing 100045, China;Capital Medical University, Beijing Tongren Hospital, Beijing 100730,China;First Hospital, Peking University, Beijing 100034, China;Henan children′Hospital, Zhengzhou 450018, China;Anhui Children′s Hospital, Hefei 230000, China;Xinhua Hospital Affiliated to Shanghai Jiaotong University School of Medicine, Shanghai 200092, China;Nanjing Medical University, Nanjing Children′s Hospital, Nanjing 100191, China;SUN YAT-SEN Memorial Hospital, SUN YAT-SEN University, Guangzhou 510000, China;Tongji Medical College of Huazhong University of Science and Technology, Wuhan Children′s Hospital, Wuhan 430030, China;Pediatric Hematology/Oncology Department, the Hospital Affiliated to Qingdao University, Qingdao 266003, China)

机构地区：[1]国家儿童医学中心(北京),首都医科大学附属北京儿童医院,儿童肿瘤中心肿瘤内科,北京市儿童血液肿瘤重点实验室,儿科重大疾病研究教育部重点实验室,北京100045 [2]首都医科大学附属北京同仁医院儿科,北京100730 [3]北京大学第一医院儿科,北京100034 [4]河南省儿童医院血液肿瘤科,郑州450018 [5]安徽省儿童医院血液肿瘤科,合肥230000 [6]上海交通大学医学院附属新华医院小儿血液肿瘤科,上海200092 [7]南京医科大学附属儿童医院血液肿瘤科,南京100191 [8]中山大学孙逸仙纪念医院儿科肿瘤专科,广州510000 [9]华中科技大学同济医学院附属武汉儿童医院血液肿瘤科,武汉430030 [10]青岛大学附属医院儿童血液肿瘤科,青岛266003

出　　处：《中国小儿血液与肿瘤杂志》2022年第2期78-82,96,共6页Journal of China Pediatric Blood and Cancer

基　　金：北京市医院管理中心“登峰”计划(DFL20191201);北京市医院管理中心儿科学协同发展中心专项(XTYB201803)。

摘　　要：目的总结中国抗癌协会小儿肿瘤专业委员会CCCG-RMS-2016方案中期研究数据。方法本研究为多中心前瞻性临床研究,对符合入组条件的新确诊RMS患儿,按照CCCG-RMS-2016方案进行治疗、评估及随诊,采集数据进行统计学分析。结果共213例新诊断的RMS患儿纳入研究,男122例(57.3%),女91例(42.7%)。中位年龄47.5(3~155)个月。低危组31(14.6%)例,中危组100(46.9%)例,高危组42(19.7%)例,中枢侵犯组40(18.8%)例。中位随访时间25(1~61)个月。4年总生存率为73.5±3.6%,4年无进展生存率为63.0±4.0%,其中低、中、高危组及中枢侵犯组4年OS分别为:100%、(80.9±4.5)%、(56.1±8.6)%及(32.2±15.1)%;4年PFS分别为:(93.5±3.2)%、(69.4±5.6)%、(39.5±8.8)%及(32.2±15.1)%。患儿的OS及PFS与原发部位、病理亚型、淋巴结转移、浸润程度及手术切除程度相关(P<0.05)。多因素COX回归分析显示:危险度分组及肿瘤浸润(T)程度是影响预后的独立危险因素(P<0.05)。结论CCCG-RMS-2016中期报告数据反应了我国当前RMS的诊治现状。Objective To summarize the mid-term research data of CCCG-RMS-2016.Methods This is a multicenter prospective clinical study,newly diagnosed patients eligible for the CCCG-RMS-2016 inclusion criteria were included in this study,they were treated,evaluated and followed up according to CCCG-RMS-2016 protocol.Clinical features and survival data of them were analyzed.Results A total of 213 newly diagnosed Rhabdomyosarcoma(RMS)patients were included in this study,including 122(57.3%)males and 91(42.7%)females,the age ranged from 3 to 155 months,the median age was 47.5 months old.There were 31(14.6%)patients in low-risk group,100(46.9%)in intermediate risk group,and 42(19.7%)in high-risk group,40(18.8%)in central invasion group.The follow-up time ranged from 1 to 61 months,with the median follow-up time 25 months.The 4 years overall survival(OS)rate and event free survival(EFS)rate were(73.5±3.6)%and(63.0±4.0)%respectively.The 4-year OS rate in low,intermediate,high and central invasion risk group was 100%,(80.9±4.5)%,(56.1±8.6)%and(32.2±15.1)%,respectively,and the 4-year EFS rate was(93.5±3.2)%,(69.4±5.6)%,(39.5±8.8)%and(32.2±15.1)%,respectively.OS and PFS rates were correlated with primary site,pathological subtype,lymph node metastasis,infiltration and surgical resection(P<0.05).Multivariate Cox regression analysis showed that the risk group and the degree of tumor invasion(T)were independent risk factors affecting the prognosis(P<0.05).Conclusions CCCG-RMS-2016 interim report data reflect the current diagnosis and treatment status of RMS in China.

关 键 词：横纹肌肉瘤 多中心前瞻性临床研究 儿童 青少年 

分 类 号：R73[医药卫生—肿瘤]