ImportanceWhether patients with Child-Pugh class B (CP-B) cancer with unresectable hepatocellular carcinoma (uHCC) benefit from active anticancer treatment vs best supportive care (BSC) is debated.ObjectiveTo evaluate the association of immune checkpoint inhibitor (ICI)–based therapies vs BSC with overall survival (OS) of patients with uHCC and CP-B liver dysfunction.Design, Setting, and ParticipantsThis retrospective, multicenter, international clinical case series examined data of patients with CP-B with uHCC who were receiving first-line ICI-based regimens from September 2017 to December 2022 whose data were extracted from an international consortium and compared with a cohort of patients with CP-B receiving BSC. Patients were treated in tertiary care centers across Europe, US, and Asia in routine clinical practice. After applying the inclusion criteria, 187 and 156 patients were left in the ICI and BSC groups, respectively. The propensity score was calculated for the following variables: age, alpha-fetoprotein levels, Child-Pugh score, extrahepatic spread, portal vein tumor thrombosis, cirrhosis, ascites, and baseline Eastern Cooperative Oncology Group performance status.ExposuresPatients in the ICI group received first-line systemic therapy with either atezolizumab plus bevacizumab (A+B) (n = 141) or nivolumab (n = 46).Main Outcomes and MeasuresOS in the inverse probability of treatment weighting (IPTW) populations was the main outcome, and it was estimated with Kaplan-Meier method; univariable Cox regression test was used to make comparisons between the 2 groups.ResultsThe median age was 66 (IQR, 61-72) and 73 (IQR, 66-81) years in the ICI (33 women [18%]) and BSC groups (41 women [26%]), respectively. In the IPTW populations, median OS was significantly longer in the ICI group (7.50 months; 95% CI, 5.62-11.15) compared with BSC (4.04 months; 95% CI, 3.03-5.03; hazard ratio, 0.59; 95% CI, 0.43-0.80; P < .001). Multivariable analysis confirmed that ICI exposure was associated with a reduction of approximately 50% in the risk of death (hazard ratio, 0.55; 95% CI, 0.35-0.86; P < .001), and the presence of portal vein tumor thrombosis, an Eastern Cooperative Oncology Group performance score of greater than 1, and alpha-fetoprotein levels of 400 ng/mL or greater were associated with increased risk of death.Conclusions and RelevanceThe results of this case series provide comparative evidence of improved survival in association with ICI treatment compared with BSC in patients with uHCC with CP-B liver dysfunction.

中文翻译：

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免疫疗法与最佳支持治疗对 Child-Pugh B 功能障碍肝细胞癌患者的治疗


重要性存在争议：伴有不可切除肝细胞癌 （uHCC） 的 Child-Pugh B 级 （CP-B） 癌症患者是否受益于积极的抗癌治疗与最佳支持治疗 （BSC）。目的评价基于免疫检查点抑制剂 （ICI） 的治疗与 BSC 与 uHCC 和 CP-B 肝功能不全患者总生存期 （OS） 的相关性。设计、设置和参与者本回顾性、多中心、国际临床病例系列检查了 2017 年 9 月至 2022 年 12 月接受一线基于 ICI 的方案的 CP-B 伴 uHCC 患者的数据，其数据是从国际联盟中提取的，并与接受 BSC 的 CP-B 患者队列进行了比较。患者在欧洲、美国和亚洲的三级医疗中心接受常规临床实践治疗。应用纳入标准后，ICI 组和 BSC 组分别留有 187 例和 156 例患者。计算以下变量的倾向评分： 年龄、甲胎蛋白水平、 Child-Pugh 评分、肝外扩散、门静脉肿瘤血栓形成、肝硬化、腹水和基线东部肿瘤合作组体能状态。暴露ICI 组患者接受 atezolizumab 加贝伐珠单抗 （A+B） （n = 141） 或 nivolumab （n = 46） 的一线全身治疗。主要结局和措施治疗加权逆概率 （IPTW） 人群的 OS 是主要结局，采用 Kaplan-Meier 方法估计;采用单因素 Cox 回归检验对 2 组进行比较。结果ICI 组 （33 名女性 [18%]）和 BSC 组 （41 名女性 [26%]）的中位年龄分别为 66 岁 （IQR，61-72） 和 73 岁 （IQR，66-81） 岁。 在 IPTW 人群中，ICI 组的中位 OS （7.50 个月;95% CI，5.62-11.15） 与 BSC 组 （4.04 个月;95% CI，3.03-5.03;风险比，0.59;95% CI，0.43-0.80;P < .001）。多变量分析证实，ICI 暴露与死亡风险降低约 50% 相关（风险比，0.55;95% CI，0.35-0.86;P < .001），以及门静脉肿瘤血栓形成、东部肿瘤合作组绩效评分大于 1 和甲胎蛋白水平 400 ng/mL 或更高与死亡风险增加相关。结论和相关性该病例系列的结果提供了比较证据，表明与 BSC 相比，ICI 治疗可改善 CP-B 肝功能不全的 uHCC 患者的生存率。