RESORCE-III 试验表明，与安慰剂相比，接受索拉非尼治疗后病情进展并接受瑞戈非尼二线治疗的晚期肝细胞癌 (HCC) 患者的总生存期有所改善。后来，免疫检查点抑制剂（ICIs）联合抗血管生成抗体的免疫疗法已发展成为健康患者的首选一线治疗方法。我们的目的是探讨瑞戈非尼单独作为一线药物或与 ICI 联合治疗晚期 HCC 患者的疗效和安全性。我们确定了 50 名接受瑞戈非尼作为一线药物治疗的晚期 HCC 患者。两名患者失访并被排除。从电子病历中记录基线因素、剂量、ICI 的同时使用、毒性和治疗结果。26 名患者接受瑞戈非尼单药治疗，22 名患者接受瑞戈非尼 ICI 联合治疗。在整个队列中，中位无进展生存期 (mPFS) 为 7.7 个月，中位总生存期 (mOS) 为 16.7 个月 (P=0.02)。根据实体瘤疗效评价标准（RECIST）1.1版评估的客观缓解率（ORR）和疾病控制率（DCR）分别为21%和73%。瑞戈非尼单药治疗组的 mPFS 为 5.9 个月，mOS 为 13.9 个月；联合组的 mPFS 为 7.8 个月，mOS 为 23.6 个月。单药治疗组的 ORR 和 DCR 分别为 15% 和 65%，联合治疗组分别为 27% 和 82%。瑞戈非尼与 ICI 联合使用具有温和的安全性，可改善反应，并使 mOS 几乎翻倍与单一疗法相比，需要在随机研究中进行进一步的前瞻性评估。2024 年胃肠肿瘤学杂志。版权所有。

The RESORCE-III trial demonstrated that advanced hepatocellular carcinoma (HCC) patients who progressed on sorafenib and had second-line therapy with regorafenib improved overall survival compared with placebo. Later, immunotherapy with immune checkpoint inhibitors (ICIs) combined with antiangiogenetic antibodies has evolved as the preferred first-line treatment for fit patients. We aimed to explore the efficacy and safety of regorafenib as a first-line agent alone or in combination with ICIs in patients with advanced HCC.We identified 50 patients with advanced HCC treated with regorafenib as a first-line agent. Two patients were lost to follow-up and excluded. Baseline factors, dosing, concomitant use of ICIs, toxicity and outcome of treatment were recorded from electronic medical records.Twenty-six patients received regorafenib as monotherapy and twenty-two received regorafenib + ICI in combination. In the total cohort, the median progression-free survival (mPFS) was 7.7 months and the median overall survival (mOS) was 16.7 months (P=0.02). Objective response rate (ORR) and disease control rate (DCR) assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 were 21% and 73%. In the regorafenib monotherapy group, mPFS was 5.9 months, and mOS was 13.9 months; in the combination group, mPFS was 7.8 months, and mOS was 23.6 months. ORR and DCR were 15% and 65% in the monotherapy group, and 27% and 82% in the combined treatment group, respectively.Regorafenib used in combination with ICIs had a mild safety profile and resulted in improved response and an almost doubling of mOS compared to monotherapy, warranting further prospective evaluation in a randomized study.2024 Journal of Gastrointestinal Oncology. All rights reserved.