Background & Aims: The outcome of patients with hepatocellular carcinoma (HCC) who achieved complete response (CR) to immune-checkpoint-inhibitor (ICI)-based systemic therapies is unclear. Approach & Results: Retrospective study of patients with HCC who had CR according to mRECIST to ICI-based systemic therapies from 28 centers in Asia, Europe, and the United States. Of 3933 patients with HCC treated with ICI-based non-curative systemic therapies, 174 (4.4%) achieved CR according to mRECIST (CR-mRECIST) and 97 (2.5%) had CR according to RECISTv1.1 (CR-RECISTv1.1) as well. The mean age of the total cohort (male, 85%; BCLC-C, 70%) was 65.9±9.8 years. The majority (83%) received ICI-based combination therapies. Median follow-up was 32.2 (95%CI, 29.9-34.4) months. One- and 3-year overall survival (OS) rates were 98% and 86%. One- and 3-year recurrence-free survival (RFS) rates were excellent in patients with CR-mRECIST-only and CR-RECISTv1.1 (78% and 55%; 70% and 42%). Among patients who discontinued ICIs for reasons other than recurrence, those who received immunotherapy for ≥6 months after first mRECIST CR had a longer RFS than those who discontinued immunotherapy earlier (p=0.008). Of 9 patients who underwent curative surgical conversion therapy, 8 (89%) had pathological CR (CR-RECISTv1.1, n= 2/2; CR-mRECIST-only, n= 6/7). Conclusion: OS and RFS of patients with CR-mRECIST-only and CR-RECISTv1.1 were excellent, and 6 of 7 patients with CR-mRECIST-only who underwent surgical conversion therapy had pathological CR. Despite potential limitations, these findings support the use of mRECIST in the context of immunotherapy for clinical decision-making. When considering ICI discontinuation, treatment for at least 6 months beyond CR seems advisable.

中文翻译：

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对基于免疫治疗的全身治疗达到完全反应的肝细胞癌患者的结局和管理


背景和目标：肝细胞癌（HCC）患者对免疫检查点抑制剂（ICI）为基础的全身疗法达到完全缓解（CR）的结果尚不清楚。方法和结果：回顾性研究，对来自亚洲、欧洲和美国的28个中心接受基于ICI的全身治疗的mRECIST对CR的HCC患者进行研究。在接受基于 ICI 的非治愈性全身治疗的 3933 例 HCC 患者中，174 例 （4.4%） 根据 mRECIST （CR-mRECIST） 达到 CR，97 例 （2.5%） 根据 RECISTv1.1 （CR-RECISTv1.1） 达到 CR。整个队列的平均年龄（男性，85%;BCLC-C，70%）为 65.9±9.8 岁。大多数 （83%） 接受了基于 ICI 的联合治疗。中位随访时间为 32.2 （95% CI，29.9-34.4） 个月。1 年和 3 年总生存率 （OS） 分别为 98% 和 86%。仅 CR-mRECIST 和 CR-RECISTv1.1 患者的 1 年和 3 年无复发生存率 （RFS） 非常好 （78% 和 55%;70% 和 42%）。在因复发以外的原因停止 ICIs 的患者中，在首次 mRECIST CR 后接受免疫治疗 ≥ 的患者比提前停止免疫治疗的患者 RFS 更长 （p=0.008）。在接受根治性手术转化治疗的 9 例患者中，8 例 （89%） 病理 CR （CR-RECISTv1.1，n= 2/2;仅限 CR-mRECIST，n= 6/7）。结论： 仅 CR-mRECIST-和 CR-RECISTv1.1 患者的 OS 和 RFS 均极好，7 例接受手术转化治疗的 CR-mRECIST-only 患者中有 6 例出现病理性 CR。尽管存在潜在的局限性，但这些发现支持在免疫疗法的背景下使用 mRECIST 进行临床决策。当考虑停用 ICI 时，似乎建议在 CR 之后至少治疗 6 个月。