病理完全缓解在肿瘤临床试验的应用进展  

Application of Pathological Complete Response in Tumor Clinical Trials

作　　者：卓俊城 宫晓 杜海坚 张声鹏 ZHUO Jun-cheng;GONG Xiao;DU Hai-jian;ZHANG Sheng-peng(Department of Medicine,Guangzhou Yufeng Tiwei Medical Research Co.,Ltd,Guangzhou 510420,China;Department of Biostatistics,Guangzhou Jeeyor Pharmaceutical Research Co.,Ltd,Guangzhou 510896,China;Second Department of Oncology,Guangzhou Clifford Hospital,Guangzhou 511495,China)

机构地区：[1]广州驭峰钛纬医学研究有限公司医学部,广州510420 [2]广州静远医药研究有限公司生物统计部,广州510896 [3]广东祈福医院肿瘤二科,广州511495

出　　处：《循证医学》2021年第4期225-231,共7页The Journal of Evidence-Based Medicine

基　　金：广州市民生科技攻关项目(201903010016)。

摘　　要：近年来,新辅助治疗备受人们关注,除了使不可切除肿瘤病变降期、提高保乳率等已明确的获益外,临床实践还发现,新辅助治疗对研发新药有潜在优势。术前治疗为评估生物等效性效应提供机会,使新药可能在初治的、更敏感人群中进行测试。此外,早期疗效替代指标,如病理完全缓解(pathological complete response,pCR)和残余肿瘤负荷,可提供长期预后相关的近似指标,从而潜在地缩短寻找有效辅助治疗的时间。目前,为促进抗肿瘤药物的研发,国内已陆续颁布了一系列用于指导抗肿瘤药物临床试验研究终点设计的指导原则,旨在希望能加速识别有治疗前景的新疗法,但pCR准确预测及长期预后的能力尚缺乏高级别的证据,仍需要进行反复验证。In recent years,neoadjuvant therapy has attracted much attention. It can not only reduce the stage of unresectable tumor and improve the breast conserving rate,but also has some potential advantages in the development of new drugs. Preoperative treatment provides an opportunity to assess biological effects,making it possible for new drugs to be tested in more sensitive,untreated populations. In addition,early therapeutic alternatives,such as pathological complete response(pCR)and residual tumor burden,can provide approximate indicators related to long-term prognosis,thus potentially shortening the time to find effective adjuvant therapy. At present,in order to promote the research and development of anti-tumor drugs,a series of guidelines have been issued in China to guide the end-point design of these clinical trials. However,the ability of pCR to accurately predict and long-term prognosis is still lacking of high-level evidence,which still needs to be repeatedly verified.

关 键 词：新辅助治疗 病理完全缓解 总生存率 研究终点 

分 类 号：R730.7[医药卫生—肿瘤]