AVDC/ICE方案治疗10例儿童颅外恶性横纹肌样瘤的临床疗效分析  

Clinical efficacy analysis of AVDC/ICE regimen in the treatment of 10 pediatric extracranial malignant rhabdoid tumors

作　　者：封淑月 张姮 孙梦娇 方拥军[1] FENG Shuyue;ZHANG Heng;SUN Mengjiao;FANG Yongjun(Department of Hematology and Oncology,Children’s Hospital of Nanjing Medical University,Nanjing 210003,Jiangsu,China)

机构地区：[1]南京医科大学附属儿童医院血液肿瘤科,江苏南京210003

出　　处：《临床儿科杂志》2024年第12期1032-1038,共7页Journal of Clinical Pediatrics

基　　金：江苏省卫生健康委员会2020年度医学科研立项项目(No.ZDB2020018)。

摘　　要：目的探讨AVDC/ICE方案治疗儿童颅外恶性横纹肌样瘤(MRT)的疗效。方法对2020年1月至2023年1月接受AVDC/ICE方案治疗的10例颅外MRT患儿进行回顾性分析。结果10例颅外MRT患儿,男女发病比例为1∶1,中位发病年龄为24.5(1~138)月龄。3例患儿初诊时发生远处转移,最常见的转移部位是肺。初诊肿瘤平均大小为(9.87±5.42)cm。2例肾恶性横纹肌样瘤(MRTK)患儿术前出现肿瘤破溃。10例患儿明确病理后均接受AVDC/ICE方案化疗。截至末次随访时间,7例患儿处于完全缓解(CR)状态,1例患儿处于部分缓解(PR)状态,目前予贝伐珠单抗+索拉菲尼+环磷酰胺维持治疗中。2例肾外非中枢神经系统恶性横纹肌样瘤(EERT)患儿死于肿瘤转移。中位随访时间为14(5~28)个月,1年总体生存率(OS)为78.8%,1年无事件生存率(EFS)为68.6%。10例患儿治疗过程中均未发生与治疗相关的严重不良事件。结论AVDC/ICE方案是治疗儿童颅外MRT安全有效的化疗选择。Objective This study aims to assess the therapeutic effectiveness of the AVDC/ICE regimen for children diagnosed with extracranial malignant rhabdoid tumors(MRT).Methods A retrospective analysis was conducted on 10 pediatric cases of extracranial MRT treated with the AVDC/ICE from January 2020 to January 2023.Results Clinical data from 10 pediatric patients with extracranial MRT were analyzed.The gender distribution was balanced(1:1),with a median age at diagnosis of 24.5 months(range 1-138 months).Three patients had distant metastases at diagnosis,predominantly in the lungs.The average size of primary tumors at diagnosis was(9.87±5.42)cm.Two patients with malignant rhabdoid tumor of the kidney(MRTK)experienced tumor rupture before surgery.Following pathological confirmation,all patients received the AVDC/ICE chemotherapy regimen.At the last follow-up,seven patients achieved complete remission(CR),one patient achieved partial remission(PR)and was receiving maintenance therapy with bevacizumab,sorafenib,and cyclophosphamide.Two patients with extra-renal extracranial rhabdoid tumor(EERT)succumbed to metastasis.The median follow-up was 14 months(range 5-28 months),with a one-year overall survival(OS)rate of 78.8%and a one-year eventfree survival(EFS)rate of 68.6%.No severe treatment-related adverse events were observed.Conclusion The AVDC/ICE regimen emerges as a safe and effective chemotherapy option for managing extracranial MRT in pediatric patients.

关 键 词：恶性横纹肌样瘤 治疗 预后 儿童 

分 类 号：R730.5[医药卫生—肿瘤]