儿童恶性横纹肌样瘤8例临床分析  

Clinical analysis of malignant rhabdoid tumor in 8 children

作　　者：顾华丽[1] 王一卓[1] 黄东生[1] 张伟令[1] 张谊[1] 周燕[1] GU Huali;WANG Yizhuo;HUANG Dongsheng;ZHANG Weiling;ZHANG Yi;ZHOU Yan(Department of Pediatrics, Beijing Tongren Hospital, Capital Medical University, Beijing 100176, China)

机构地区：[1]首都医科大学附属北京同仁医院儿科,北京100176

出　　处：《中国小儿血液与肿瘤杂志》2020年第4期214-219,共6页Journal of China Pediatric Blood and Cancer

摘　　要：目的研究儿童恶性横纹肌样瘤(MRT)的临床特点、治疗方法及预后情况。方法回顾性分析北京同仁医院儿科病房2005年1月1日—2017年1月1日收治的确诊为MRT的8例儿童病例资料和预后。结果8例MRT患儿中,男2例,女6例;中位发病年龄16(9~68)个月,≤1岁者3例,>1岁者5例。发病部位:肾脏原发5例,其中左肾3例,右肾2例;肾外部位包括盆腔1例,腹膜后1例,椎管1例。肿瘤分期Ⅲ期5例,Ⅳ期3例;肾恶性横纹肌样瘤(MRTK)包括Ⅲ期3例、Ⅳ期2例,肾外非中枢神经系统恶性横纹肌样瘤(EERT)包括Ⅲ期2例,非典型畸胎瘤样/横纹肌样瘤(AT/RT)Ⅳ期1例。本组患儿接受包括化疗、手术、放疗及靶向治疗的综合治疗。中位随访时间11.5(5~33)个月,5例死亡,中位生存时间8(5~13)个月,3例患儿无事件生存(EFS)37.5%(3/8)。3例≤1岁的患儿均死亡(100%);5例>1岁患儿死亡2例,占40%(2/5)。肿瘤分期Ⅲ期者存活3/5,Ⅳ期存活0/3;MRTK存活2/5,EERT存活1/2,AT/RT存活0/1。结论儿童MRT临床可因发病部位不同而表现多样,恶性程度极高,预后差,年龄及分期均可影响预后。目前尚无MRT标准治疗方案,靶向治疗研究可能是改善MRT预后的突破点。Objective To explore the clinical features,treatment methods and the prognosis of malignant rhabdoid tumor(MRT)in children.Methods The clinical data of the 8 patients of MRT treated in Tongren Hospital from Jan 1,2005 to Jan 1,2017 were retrospectively analyzed.Results A total of 8 MRT patients were enrolled,including 2 male and 6 female.The median age was 16 months,with a range from 9 to 68 months.Three cases were younger than 1-year-old,and 5 cases were elder than 1-year-old.The primary lesion location,included kidney(5 cases,including 3 cases in left and 2 cases in right),pelvic(1 case),retroperitoneal(1 case)and spinal canal(1 case).There were 5 cases of Renal malignant rhabdomoid tumor(MRTK),including 3 cases of StageⅢand 2 cases of StageⅣ,2 cases of extrarenal non-central nervous system malignant rhabdomoid tumor(EERT)both in StageⅢand 1 case of atypical teratoma-like/rhabdomoid tumor(AT/RT)in StageⅣ.Treatment strategies included surgery,chemotherapy,radiotherapy,and targeted therapy.The median follow-up time was 11.5 months,with a range from 5 to 33 months.During follow up,5 cases died and the median survival time was 8 months with a range from 5 to 13 months.Three cases(3/8,37.5%)were event-free survival.The fatality rates of patients younger than 1-year-old was 3/3 and patients elder than 1-year-old was 2/5.Survival rates of patients in Stage III was 3/5 and StageⅣwas 0/3.Survival rates of patients with MRTK,EERT and AT/RT were 2/5,1/2 and 0/1,respectively.Conclusions The clinical manifestations of MRT in children varies due to different sites of tumor.MRT is highly malignant with poor prognosis.Both age and stage are prognostic factors.Currently,there is no standard treatment regimen for MRT,and targeted therapy might be the breakthrough to improve the prognosis of MRT.

关 键 词：恶性横纹肌样瘤 肾恶性横纹肌样瘤 肾外非中枢神经系统恶性横纹肌样瘤 非典型畸胎瘤样/横纹肌样瘤 预后 

分 类 号：R73[医药卫生—肿瘤]