UCL Cancer Institute, University College London, London, UK.
Department of Molecular and Clinical Cancer Medicine, University of Liverpool, Liverpool, UK.
Liver Int. 2017 Jul;37(7):1047-1055. doi: 10.1111/liv.13359. Epub 2017 Feb 2.
BACKGROUND & AIMS: Response Evaluation Criteria in Solid Tumors (RECIST) has been shown to be a poor surrogate for survival benefit with targeted therapy in advanced hepatocellular carcinoma (HCC).
We investigated whether response evaluated using modified RECIST (mRECIST) predicted overall survival (OS) using data from two Phase II clinical trials. Analyses were conducted on pooled data from 188 patients with advanced HCC treated with nintedanib or sorafenib, of whom 180 were evaluable for response. Cox regression and Kaplan-Meier survival analyses were used to explore differences in OS between the responders and non-responders according to RECIST 1.0 and mRECIST criteria. Multivariate Cox proportional hazards models, including factors known to influence survival, were used to compare survival according to RECIST and mRECIST response.
Discordance between RECIST and mRECIST evaluation was most common for assessment of partial response (12.2%) and stable disease (13.3%). OS was significantly longer in patients with response compared to patients without response-RECIST: hazard ratio (HR) 0.325 (95% confidence interval [CI] 0.130-0.815), P=.0122; mRECIST: HR 0.544 (95% CI 0.335-0.881), P=.0122. HRs from the multivariate models used to evaluate response by RECIST or by mRECIST as predictors of OS approached significance for RECIST (0.40 [95% CI 0.16-1.01]; P=.053) and for mRECIST (0.62 [95% CI 0.38-1.01]; P=.053).
Response according to RECIST or mRECIST is associated with improved survival and should be considered as a valid endpoint for use in HCC clinical trials.
在晚期肝细胞癌（HCC）中，与靶向治疗相关的实体瘤反应评价标准（RECIST）已被证明不能很好地替代生存获益。
我们使用来自两项 II 期临床试验的数据，调查使用改良 RECIST（mRECIST）评估的反应是否可以预测总生存期（OS）。对接受尼替西农或索拉非尼治疗的 188 例晚期 HCC 患者的汇总数据进行了分析，其中 180 例可评估反应。使用 Cox 回归和 Kaplan-Meier 生存分析根据 RECIST 1.0 和 mRECIST 标准，探索反应者和无反应者之间 OS 的差异。使用包括已知影响生存的因素在内的多变量 Cox 比例风险模型，比较根据 RECIST 和 mRECIST 反应的生存情况。
RECIST 和 mRECIST 评估之间的不一致最常见于部分缓解（12.2%）和稳定疾病（13.3%）的评估。与无反应者相比，有反应者的 OS 明显更长-RECIST：风险比（HR）0.325（95%置信区间 [CI] 0.130-0.815），P=.0122；mRECIST：HR 0.544（95% CI 0.335-0.881），P=.0122。使用多变量模型评估 RECIST 或 mRECIST 作为 OS 预测因子的反应的 HR 接近显著性（RECIST：0.40 [95% CI 0.16-1.01]；P=.053）和 mRECIST（0.62 [95% CI 0.38-1.01]；P=.053）。
根据 RECIST 或 mRECIST 的反应与改善的生存相关，应被视为 HCC 临床试验中有效的终点。
