Department of Gastroenterological Surgery, Osaka General Medical Center, Osaka.
Gastroenterology Center, Japanese Foundation for Cancer Research, Cancer Institute Hospital, Tokyo.
ESMO Open. 2023 Aug;8(4):101614. doi: 10.1016/j.esmoop.2023.101614. Epub 2023 Aug 8.
Trifluridine/tipiracil (FTD/TPI) and regorafenib (REG) are standard therapies for refractory metastatic colorectal cancer (mCRC). No results of large real-world data directly comparing FTD/TPI + bevacizumab (BEV) with FTD/TPI or REG monotherapy have been reported. We evaluated the efficacy and safety of FTD/TPI + BEV in a real-world setting.
This retrospective study used a Japanese claims database provided by Medical Data Vision Co., Ltd. (Tokyo, Japan). Eligible patients were aged 20 years and over with a diagnosis of mCRC, and received their first dose of FTD/TPI or REG from 2014 to 2021. The primary endpoint was overall survival (OS) in a propensity score matching (PSM) population in which PSM was carried out by matching using a 1 : 1 ratio for the FTD/TPI + BEV group and the control group (FTD/TPI or REG) by propensity score. To enhance robustness, sensitivity analyses of OS were carried out using the inverse probability treatment weighted (IPTW) approach and the analysis in the all eligible population. Secondary endpoints included time to treatment discontinuation (TTD), incidence of adverse events, and post-treatment.
Eligible population was 2369 for the FTD/TPI + BEV group and 9318 for the control group. The PSM population was 1787 for each group. Median OS (mOS) was longer in the FTD/TPI + BEV group compared to the control group [17.0 versus 11.6 months, hazard ratio (HR) = 0.70, P < 0.001] in the PSM population. Similarly, mOS was longer for the FTD/TPI + BEV group compared to that for the control group in IPTW analyses and in the all eligible population (both HRs = 0.68). Median TTD was 3.3 months for the FTD/TPI + BEV group and 1.8 months for the control group in the PSM population (P < 0.001).
Real-world data showed that FTD/TPI + BEV was significantly associated with OS and TTD compared to FTD/TPI or REG. In clinical practice, FTD/TPI + BEV can be a favorable regimen for refractory mCRC.
替匹嘧啶/鲁比卡丁（FTD/TPI）和瑞戈非尼（REG）是治疗难治性转移性结直肠癌（mCRC）的标准疗法。尚无大样本真实世界数据直接比较 FTD/TPI+贝伐珠单抗（BEV）与 FTD/TPI 或 REG 单药治疗的结果。我们在真实环境中评估了 FTD/TPI+BEV 的疗效和安全性。
这项回顾性研究使用了由 Medical Data Vision Co., Ltd.（日本东京）提供的日本索赔数据库。符合条件的患者年龄在 20 岁及以上，患有 mCRC，并在 2014 年至 2021 年期间首次接受 FTD/TPI 或 REG 治疗。主要终点是倾向评分匹配（PSM）人群中的总生存期（OS），PSM 通过对 FTD/TPI+BEV 组和对照组（FTD/TPI 或 REG）进行 1:1 比例的倾向评分匹配来进行。为了增强稳健性，还使用逆概率处理加权（IPTW）方法和所有合格人群中的分析对 OS 进行了敏感性分析。次要终点包括治疗终止时间（TTD）、不良事件发生率和治疗后。
FTD/TPI+BEV 组符合条件的人群为 2369 人，对照组为 9318 人。每组 PSM 人群为 1787 人。与对照组相比，FTD/TPI+BEV 组的 PSM 人群中位 OS（mOS）更长[17.0 个月比 11.6 个月，风险比（HR）=0.70，P<0.001]。同样，在 IPTW 分析和所有合格人群中，FTD/TPI+BEV 组的 mOS 也长于对照组（HRs=0.68）。FTD/TPI+BEV 组的中位 TTD 为 3.3 个月，对照组为 1.8 个月，在 PSM 人群中（P<0.001）。
真实世界数据显示，与 FTD/TPI 或 REG 相比，FTD/TPI+BEV 与 OS 和 TTD 显著相关。在临床实践中，FTD/TPI+BEV 可能是难治性 mCRC 的一种有利治疗方案。
