基于真实世界样本的结直肠癌靶向药物治疗比较研究

Comparative Study of Targeted Drug Therapy for Colorectal Cancer Based on Real World Samples

目的比较浙江大学医学院附属第一医院收治的晚期转移结直肠癌患者一线使用西妥昔单抗的疗效与国际多中心大型随机对照试验(RCT)结果的异同,以定位基于基因状态的靶向药物治疗在我国结直肠癌治疗中的适宜性和科学性。方法采用真实世界研究方法,回顾性分析2014年1月—2018年6月收治的晚期转移结直肠癌一线治疗使用西妥昔单抗的病例,系统评估其客观缓解率、疾病控制率、不良反应等,并与现有国际多中心RCT FIRE-3结果进行比较。结果统计发现,本次纳入研究的真实世界样本病例与FIRE-3纳入研究病例的既往化疗方案存在差异,而在疗效方面,一线治疗使用西妥昔单抗的晚期转移结直肠癌患者近期疗效明显低于FIRE-3的研究结果,客观缓解率为47.1%vs 71.3%(P=0.006),疾病控制率76.5%vs91.8%(P=0.011),不良反应发生频率和程度则相当。结论基于笔者所在医院结直肠癌靶向药物治疗真实世界研究数据较国际多中心RCT存在差距,提示肿瘤的靶向治疗与基线数据、人群差异等多种因素相关。

OBJECTIVE To compare the efficacy of cetuximab as the first-line treatment for advanced colorectal cancer between the First Affiliated Hospital of Medical College of Zhejiang University and international multicenter randomized controlled trial(RCT), so as to discuss the suitability and feasibility of targeted drug therapy based on gene status in colorectal cancer treatment in China. METHODS By adopting the method of real world research, a retrospective analysis was conducted on the first-line treatment with cetuximab in patients with advanced colorectal cancer hospitalized hospital from January 2014 to June 2018. The objective response rate, disease control rate, adverse reactions and so on were systematically evaluated, and compared with those from the existing international multicenter RCT FIRE-3. RESULTS The data showed that chemotherapy regimens were different between real world sample cases and trial cases in the FIRE-3. The short-term efficacy of cetuximab as the first-line treatment for advanced colorectal cancer given was significantly lower than that of FIRE-3. For examples, the objective response rate was 47.1% compared to 71.3% in the FIRE-3(P=0.006), disease control rate was 76.5% compared to91.8% in the FIRE-3(P=0.011), and the frequency and degree of adverse reactions were similar between the hospital and the FIRE-3. CONCLUSION There is a gap between the real world research of the hospital and international multicenter RCT,which suggests that the efficacy of targeted therapy is related to baseline data, population difference, and other factors.