儿童横纹肌肉瘤的靶向治疗临床试验进展  

Advances in clinical trials of targeted therapy of pediatric rhabdomyosarcoma

作　　者：吴纪恒 王金湖[3] 茅君卿[3] 倪韶青[1,2] WU Ji-heng;WANG Jin-hu;MAO Jun-qing;NI Shao-qing(Office of Drug Clinical Trial Institution of Children's Hospital Affiliated to Medical College of Zhejiang University,Hangzhou 310052,China;National Clinical Medicine Research Center for Child Health and Disease,Hangzhou 310052,China;Department of Pediatric Oncology,Children's Hospital Affiliated to Medical College of Zhejiang University,Hangzhou 310052,China)

机构地区：[1]浙江大学医学院附属儿童医院药物临床试验机构办公室,杭州310052 [2]国家儿童健康与疾病临床医学研究中心,杭州310052 [3]浙江大学医学院附属儿童医院儿童肿瘤外科,杭州310052

出　　处：《中国新药杂志》2022年第15期1480-1486,共7页Chinese Journal of New Drugs

基　　金：国家自然科学基金资助项目(81573516)。

摘　　要：儿童横纹肌肉瘤(rhabdomyosarcoma, RMS)是高度恶性的软组织肿瘤,目前的国内外诊疗指南中的标准治疗方案均以手术、放疗、化疗药治疗为主导。相关药物已经使用了几十年,虽然能够产生一定疗效,但复发难治性儿童RMS患者的预后生存依然不理想。如今,靶向治疗的出现为RMS患者带来了新的选择。本文就目前全球范围内正在进行的儿童RMS靶向药物治疗及临床试验进展做一综述,以期为国内相关临床研究提供参考。Pediatric rhabdomyosarcoma is a highly malignant tissue sarcoma. The current standard therapy in treatment guidelines is dominated by surgery, radiotherapy and chemotherapy, which have been used for decades. Although the chemotherapeutic agents have certain curative effect, the prognostic survival of patients with relapsed and refractory pediatric rhabdomyosarcoma is unsatisfactory. The emergence of targeted therapy has brought new options for patients with rhabdomyosarcoma. The review summarizes the ongoing trials of targeted drugs in the treatment of pediatric rhabdomyosarcoma, in order to provide references for the development of relevant clinical trials in China.

关 键 词：儿童横纹肌肉瘤 靶向治疗 临床试验 

分 类 号：R979.1[医药卫生—药品]