Background: Several narrative reports document long-term responses to regorafenib treatment in patients with metastatic colorectal cancer (mCRC). However, no large-scale study has assessed long-term responses and there are no established predictors of potential long-term benefit. We carried out an observational study of characteristics of patients treated in real-world clinical practice in the USA using duration of treatment (DoT) as a surrogate for treatment response. Patients and Methods: This retrospective cohort study used a de-identified electronic health record-derived database and included patients aged ≥18 years with mCRC who initiated regorafenib monotherapy between 1 July 2013 and 30 June 2023. Patient cohorts were defined by DoT ≥4 months (LTR4), ≥5 months (LTR5), or ≥6 months (LTR6) and are not mutually exclusive. Results: Of 2444 patients who initiated regorafenib monotherapy during the study, those with long-term response were analyzed: 544 had LTR4 (22%), 367 had LTR5 (15%), and 250 had LTR6 (10%). Most patients with long-term responses had left-sided tumors (65–70%), Eastern Cooperative Oncology Group performance status of 0/1 (67–68%), and liver metastases (55–61%) and had received prior bevacizumab treatment (60–67%). The median age in each group was 66 years, and patients most frequently initiated regorafenib as third-line treatment (31–33%). Median time to regorafenib discontinuation was 6.0–9.3 months among long-term responders. Conclusions: Most patients with long-term responses to regorafenib had favorable performance status at treatment initiation, left-sided tumors, and liver metastases and had received prior bevacizumab treatment. The study highlights that patients in the real-world setting were able to tolerate and maintain long-term responses to regorafenib treatment.
背景：已有若干叙述性报告记录了转移性结直肠癌（mCRC）患者对瑞戈非尼治疗的长期反应。然而，尚无大规模研究评估长期反应，且缺乏公认的潜在长期获益预测因子。本研究基于美国真实世界临床实践，以治疗持续时间（DoT）作为治疗反应的替代指标，对接受治疗的患者特征进行观察性分析。 患者与方法：这项回顾性队列研究采用去识别化的电子健康记录衍生数据库，纳入2013年7月1日至2023年6月30日期间开始瑞戈非尼单药治疗、年龄≥18岁的mCRC患者。根据DoT≥4个月（LTR4）、≥5个月（LTR5）或≥6个月（LTR6）定义患者队列（各组非互斥）。结果：在研究期间开始瑞戈非尼单药治疗的2444例患者中，对长期反应者进行分析：544例（22%）为LTR4，367例（15%）为LTR5，250例（10%）为LTR6。多数长期反应者具有左侧原发肿瘤（65–70%）、美国东部肿瘤协作组体力状态评分0/1分（67–68%）、肝转移（55–61%）且既往接受过贝伐珠单抗治疗（60–67%）。各组中位年龄均为66岁，患者最常将瑞戈非尼作为三线治疗（31–33%）。长期反应者的中位停药时间为6.0–9.3个月。 结论：对瑞戈非尼产生长期反应的患者多数在治疗开始时体力状态良好，具有左侧原发肿瘤和肝转移，且既往接受过贝伐珠单抗治疗。本研究提示，真实世界中的患者能够耐受瑞戈非尼治疗并维持长期反应。
肿瘤(癌症)患者之家