免疫检查点抑制剂（ICI）在晚期肝细胞癌（HCC）患者中的使用已变得广泛，在新辅助治疗中取得了令人鼓舞的结果。围移植环境中的安全性和意向治疗 (ITT) 结果目前基于小型且异质的单中心报告。美国第一项多区域研究（2016-2023）包括 117 名连续接受 LT 评估并在术前接受 ICI 治疗的 HCC 患者。进行意向治疗 ITT 和生存分析，并评估 LT 后排斥率。总共有 86 名患者 (73.5%) 超过 MC，65 名患者 (75.6%) 在中位 5.6 个月内成功降期 (DS)。 43 例 (36.7%) 接受了移植，其中 18 例 (15.4%) 处于 MC 范围内，23 例 (19.7%) 最初超出 DS 范围。总体而言，94% 的队列接受了同步 ICI 和局部治疗。等候名单上没有发生4-5级不良事件。 MC 范围内的 3 年累积辍学概率为 28%，超出 MC 范围的为 48%。脱落的独立预测因素包括：超出 MC（p<0.001）、AFP 较基线翻倍（p=0.014）和放射学反应（p<0.001）。 3 年 ITT 生存率为 71.1%（MC 内为 73.5%，MC 外为 69.7%，p=0.329），LT 后 3 年生存率为 85%。 7 名患者发生了 LT 后排斥反应，其中 6 名患者在 LT 前不到 3 个月内接受了最后一剂 ICI，导致 1 名移植物丢失。对 LT 前接受 ICI 的 HCC 患者进行的首次多中心评估显示出良好的生存和安全结果，证明了这一点在临床实践中继续利用并进一步评估该策略。高肿瘤负荷、AFP 水平加倍和放射学反应被确定为不良肿瘤结果的预测因素。首次对肝细胞癌移植前免疫检查点抑制剂进行多中心评估，显示出有希望的意向治疗生存率、安全性和排斥反应费率。免疫检查点抑制剂，无论是单独使用还是与 LRT 联合使用，都显示出可靠的疗效。这种术前策略可能对高危患者特别有益，包括那些需要降期或尽管接受局部治疗但 AFP 水平仍升高的患者。这些发现填补了当前的知识空白，并为移植前使用免疫检查点抑制剂的可行性提供了令人放心的证据，等待正在进行的试验结果。版权所有 © 2024。由 Elsevier B.V. 出版。

The use of immune checkpoint inhibitors (ICI) in patients with advanced hepatocellular carcinoma (HCC) has become widespread with encouraging outcomes in the neoadjuvant setting. Safety and intention to treat (ITT) outcomes in the peri transplant setting are currently based on small and heterogenous single center reports.This first multiregional US study (2016-2023) included 117 consecutive HCC patients assessed for LT and treated preoperatively with ICIs. Intention to treat ITT and survival analyses were conducted with evaluation of post LT rejection rates.In total, 86 (73.5%) patients exceeded MC and 65 (75.6%) were successfully downstaged (DS) within a median of 5.6 months. 43 (36.7%) underwent transplantation, including 18 (15.4%) within MC and 23 (19.7%) initially beyond and DS. Overall, 94% of the cohort received concurrent ICIs and locoregional therapies. No grade 4-5 adverse events occurred on the waiting list. The 3-year cumulative probability of dropout was 28% for those within MC and 48% for those beyond. Independent predictors of dropout included: being beyond MC (p<0.001), AFP doubling from baseline (p=0.014) and radiographic responses (p<0.001). The 3-year ITT survival was 71.1% (73.5% within MC vs 69.7% beyond MC, p=0.329), with 3-year post LT survival rate of 85%. Post-LT rejection occurred in 7 patients, six received their last dose of ICI less than 3 months prior to LT, resulting in one graft loss.The first multicenter evaluation of HCC patients receiving ICI pre-LT demonstrates favorable survival and safety outcomes, justifying continued utilization and further evaluation of this strategy in clinical practice. High tumor burden, doubling of AFP levels, and radiographic response were identified as predictors of unfavorable oncologic outcomes.The first multicenter evaluation of pre-transplant immune-checkpoint-inhibitors in hepatocellular carcinoma to show promising intention-to-treat survival, safety and rejection rates. Immune-checkpoint-inhibitors, either alone or combined with LRT, demonstrate reliable efficacy. This preoperative strategy could be particularly beneficial for high-risk patients, including those requiring downstaging or with elevated AFP levels despite locoregional treatment. These findings fill current knowledge gaps and offer reassuring evidence for the feasibility of pre-transplant use of immune-checkpoint-inhibitors, pending results from ongoing trials.Copyright © 2024. Published by Elsevier B.V.