Background: While immune checkpoint inhibitors (ICIs) have redefined systemic therapy in hepatocellular carcinoma (HCC), pivotal trials have not yet included patients with advanced liver disease. Real-world data are needed to assess treatment outcomes in advanced liver disease populations. Methods: We conducted a retrospective analysis of 53 HCC patients treated with ICIs at a large single center between January 2017 and June 2023. Clinical characteristics, liver function scores [Child–Turcotte–Pugh (CTP) and albumin–bilirubin (ALBI)], treatment history, and survival outcomes were analyzed. Primary endpoints included progression-free survival (PFS), survival from ICI initiation (OS-ICI), and overall survival (OS). Secondary endpoints included incidence and predictors of immune-related adverse events (irAEs). Results: Among 53 HCC patients treated with ICIs, the median OS, OS-ICI, and PFS were 18.7 months (m), 7.4 m, and 4.6 m, respectively. On multivariable analysis, a higher ALBI grade and history of alcohol use were independently associated with worse PFS and OS-ICI, while prior locoregional therapy (LRT) significantly improved OS (HR: 0.43;p: 0.012). The ALBI grade outperformed the CTP score in predicting outcomes, highlighting its utility as a more objective liver function marker. Patients receiving atezolizumab–bevacizumab showed improved OS-ICI compared to other regimens (HR: 0.37;p= 0.021). irAEs occurred in 19% of patients, most commonly in those with CTP-A, and were generally manageable. Conclusions: These real-world insights into the efficacy and safety of ICI-based therapies across a more diverse HCC population are usually not represented in clinical trials.
背景：尽管免疫检查点抑制剂（ICIs）已重新定义了肝细胞癌（HCC）的系统性治疗，但关键性临床试验尚未纳入晚期肝病患者。需要真实世界数据来评估晚期肝病人群的治疗结局。 方法：我们对2017年1月至2023年6月期间在一家大型单中心接受ICIs治疗的53例HCC患者进行了回顾性分析。分析了临床特征、肝功能评分[Child–Turcotte–Pugh（CTP）和白蛋白-胆红素（ALBI）]、治疗史和生存结局。主要终点包括无进展生存期（PFS）、自ICI启动起的生存期（OS-ICI）和总生存期（OS）。次要终点包括免疫相关不良事件（irAEs）的发生率和预测因素。 结果：在接受ICIs治疗的53例HCC患者中，中位OS、OS-ICI和PFS分别为18.7个月、7.4个月和4.6个月。多变量分析显示，较高的ALBI分级和饮酒史与较差的PFS和OS-ICI独立相关，而既往接受过局部区域治疗（LRT）则显著改善OS（HR：0.43；p：0.012）。在预测结局方面，ALBI分级优于CTP评分，突显了其作为更客观肝功能标志物的实用性。与其他方案相比，接受阿替利珠单抗-贝伐珠单抗治疗的患者OS-ICI有所改善（HR：0.37；p=0.021）。19%的患者发生了irAEs，最常见于CTP-A级患者，且通常可控。 结论：这些真实世界数据揭示了基于ICI的疗法在临床试验通常未涵盖的、更具多样性的HCC人群中的疗效和安全性。
肿瘤(癌症)患者之家