Background: For patients with refractory metastatic colorectal cancer (mCRC), trifluridine/tipiracil (FTD–TPI) has been associated with a significant improvement in overall survival (OS). However, data are lacking regarding the activity of FTD–TPI in patients withBRAF-mutated mCRC. Methods: This retrospective, multicenter, international cohort included patients withBRAF-mutated mCRC treated with FTD–TPI in a real-life setting in Spain and Italy. Survival analysis was performed using Kaplan–Meier methods and Cox proportional hazard models and according to established prognostic groups: good prognosis characteristics (GPC; < 3 metastatic sites and time from metastases to FTD–TPI ≥ 18 months) and poor prognosis characteristics (PPC; ≥ 3 metastatic sites or time from metastases to FTD–TPI < 18 months). Results: In the 26 patients included, the median age was 61 years, 13 (50%) were female, and 20 (77%) had an Eastern Cooperative Oncology Group (ECOG) performance status of 1. Fourteen (56%) patients had right-sided tumors, six (23%) had microsatellite instability tumors, and thirteen (50%) had liver metastases. Median progression-free survival was 2.3 months (95% CI 2.0–3.2), and median OS (mOS) was 6.6 months (95% CI 4.4–12.0). mOS was 7.6 vs. 4.2 months (HR 1.64, 95% CI 0.65–4.10,p= 0.3) for GPC and PPC patients, respectively. Exploratory analyses identified ECOG as the only feature associated with survival. The most frequent grade 3–4 adverse events were neutropenia (8%), anemia (8%), and asthenia (4%). Conclusions: Patients withBRAFmutant mCRC achieved modest benefits with FTD–TPI; however, patients with GPC and ECOG 0 achieved longer OS compared with those with PPC or ECOG 1–2, thus warranting further exploration in prospective cohorts.
背景：对于难治性转移性结直肠癌（mCRC）患者，曲氟尿苷/替匹嘧啶（FTD-TPI）与总生存期（OS）的显著改善相关。然而，目前缺乏关于FTD-TPI在BRAF突变mCRC患者中疗效的数据。方法：这项回顾性、多中心、国际队列研究纳入了西班牙和意大利在真实世界临床实践中接受FTD-TPI治疗的BRAF突变mCRC患者。采用Kaplan-Meier法和Cox比例风险模型进行生存分析，并根据既定预后分组进行分析：良好预后特征组（GPC；转移部位<3个且从转移至开始FTD-TPI治疗的时间≥18个月）和不良预后特征组（PPC；转移部位≥3个或从转移至开始FTD-TPI治疗的时间<18个月）。结果：在纳入的26例患者中，中位年龄为61岁，13例（50%）为女性，20例（77%）美国东部肿瘤协作组（ECOG）体能状态评分为1分。14例（56%）患者为右侧肿瘤，6例（23%）为微卫星不稳定性肿瘤，13例（50%）存在肝转移。中位无进展生存期为2.3个月（95% CI 2.0–3.2），中位总生存期（mOS）为6.6个月（95% CI 4.4–12.0）。GPC组和PPC组患者的mOS分别为7.6个月和4.2个月（HR 1.64，95% CI 0.65–4.10，p=0.3）。探索性分析确定ECOG评分是与生存相关的唯一特征。最常见的3-4级不良事件为中性粒细胞减少症（8%）、贫血（8%）和乏力（4%）。结论：BRAF突变mCRC患者从FTD-TPI治疗中获益有限；然而，与PPC组或ECOG 1-2分患者相比，GPC组和ECOG 0分患者获得了更长的OS，因此值得在前瞻性队列中进一步探索。
肿瘤(癌症)患者之家