Purpose

To compare the efficacy and safety of transcatheter arterial chemoembolization (TACE) in combination with tyrosinkinase inhibitors (TKI) and PD-1 inhibitors, versus TACE monotherapy for the treatment of ruptured hepatocellular carcinoma (HCC).

Materials and Methods

This study included 104 patients with ruptured HCC receiving either combination therapy or TACE monotherapy at two centers between June 2015 and June 2022. Propensity score matching (PSM) analysis was used at a 1:2 ratio to reduce bias between the two groups. The primary outcome measures were overall survival (OS) and progression-free survival (PFS), and the secondary outcome measures were the occurrence of adverse events (AEs, Common Terminology Criteria for AEs, version 5.0.) and the peritoneal metastasis rate.

Results

A total of 69 patients were enrolled after PSM, including 23 patients in the combination group and 46 patients in the monotherapy group. The combination group exhibited a significantly longer median OS (553 days, 95% confidence interval [CI] 222.6–883.9) compared to the monotherapy group (105 days, 95% CI 81.2–128.7; P < 0.001). Similarly, the combination group showed a better median PFS (356 days, 95% CI 299.5–412.4) compared to the monotherapy group (97 days, 95% CI 75.9–118.1; P < 0.001). Moreover, there was no significant difference in the peritoneal metastasis rate (combination group: 8.6% vs. monotherapy group: 26.1%, P = 0.119). Grade 3 AEs occurred at a rate of 21.7% and 13% in combination and monotherapy groups, respectively. No Grade 4/5 AEs were observed in either group.

Conclusions

Our study demonstrated that the combination of TACE with TKI and PD-1 inhibitors significantly enhances OS and PFS compared to TACE monotherapy in ruptured HCC patients. Furthermore, this combined approach exhibited an acceptable safety profile.

Graphical abstract

中文翻译：

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经动脉化疗栓塞联合酪氨酸激酶抑制剂和 PD-1 抑制剂治疗自发性破裂性肝细胞癌

目的

比较经导管动脉化疗栓塞 (TACE) 联合酪氨酸激酶抑制剂 (TKI) 和 PD-1 抑制剂与 TACE 单药治疗破裂性肝细胞癌 (HCC) 的疗效和安全性。

材料和方法

这项研究纳入了 2015 年 6 月至 2022 年 6 月期间在两个中心接受联合治疗或 TACE 单药治疗的 104 名破裂 HCC 患者。以 1:2 的比例使用倾向评分匹配 (PSM) 分析，以减少两组之间的偏差。主要结局指标是总生存期 (OS) 和无进展生存期 (PFS)，次要结局指标是不良事件的发生率（AE，AE 通用术语标准，5.0 版）和腹膜转移率。

结果

PSM后共有69例患者入组，其中联合组23例，单药组46例。与单药治疗组（105 天，95% CI 81.2–128.7； P < 0.001 ）相比，联合治疗组的中位 OS 显着更长（553 天，95% 置信区间 [CI] 222.6–883.9） 。同样，与单药治疗组（97 天，95% CI 75.9–118.1；P  < 0.001）相比，联合治疗组显示出更好的中位 PFS（356 天，95% CI 299.5–412.4）。此外，腹膜转移率无显着差异（联合组：8.6% vs 单药组：26.1%，P  = 0.119）。联合治疗组和单一治疗组的 3 级 AE 发生率分别为 21.7% 和 13%。两组均未观察到 4/5 级 AE。

结论

我们的研究表明，与 TACE 单药治疗相比，TACE 联合 TKI 和 PD-1 抑制剂可显着提高破裂 HCC 患者的 OS 和 PFS。此外，这种组合方法表现出可接受的安全性。

图形概要