Bi Huaqiang, Pei Jun, Ma Kuansheng, Zhang Yanling, Aprile Giuseppe, Moris Dimitrios, Méndez-Sánchez Nahum, Samant Hrishikesh, Sun Ximin, Xia Feng
Department of Hepatobiliary Surgery, First Affiliated Hospital of Third Military Medical University (Army Medical University), Chongqing, China.
Department of Oncology, First Affiliated Hospital of Third Military Medical University (Army Medical University), Chongqing, China.
J Gastrointest Oncol. 2024 Feb 29;15(1):368-376. doi: 10.21037/jgo-23-618. Epub 2024 Feb 28.
Most advanced hepatocellular carcinoma (HCC) cases administered molecular targeted agents and/or anti-programmed cell death-1 (PD-1) inhibitors have no response or develop resistance. Moreover, second-line therapies still cannot provide beneficial clinical outcomes. A pilot study assessing combined regorafenib and PD-1 inhibitor as second-line treatment of advanced HCC reported promising effectiveness.
The current single-center, retrospective, real-world study was carried out between January 2019 and July 2021. Advanced HCC cases were administered second-line regorafenib combined with a PD-1 inhibitor or regorafenib alone were assessed. Progression-free survival (PFS), objective response rate (ORR), and disease control rate (DCR) were determined.
Totally 46 HCC cases were analyzed, most of whom underwent previous systemic treatment comprising targeted therapy and immunotherapy. Tumor response was evaluated in 25 and 21 individuals in the regorafenib + PD-1 inhibitor and regorafenib monotherapy groups, respectively: ORRs were 21.7% and 8.7%, and DCRs were 47.8% and 32.6%, respectively. Median PFS was markedly longer in the regorafenib plus PD-1 inhibitor group (11.5 months) compared with the regorafenib monotherapy group (5.1 months, P=0.049).
This study suggested regorafenib and a PD-1 inhibitor in combination may provide significant clinical benefits in HCC cases showing progression following first-line treatment. Further analysis in real-world studies with large cohorts is warranted to confirm these findings.
大多数接受分子靶向药物和/或抗程序性细胞死亡蛋白1（PD-1）抑制剂治疗的晚期肝细胞癌（HCC）患者无反应或产生耐药性。此外，二线治疗仍无法提供有益的临床结果。一项评估瑞戈非尼联合PD-1抑制剂作为晚期HCC二线治疗的试点研究报告了有前景的疗效。
本单中心、回顾性、真实世界研究于2019年1月至2021年7月进行。对接受二线瑞戈非尼联合PD-1抑制剂或单独使用瑞戈非尼治疗的晚期HCC患者进行评估。确定无进展生存期（PFS）、客观缓解率（ORR）和疾病控制率（DCR）。
共分析了46例HCC患者，其中大多数曾接受过包括靶向治疗和免疫治疗在内的全身治疗。瑞戈非尼+PD-1抑制剂组和瑞戈非尼单药治疗组分别有25例和21例患者接受了肿瘤反应评估：ORR分别为21.7%和8.7%，DCR分别为47.8%和32.6%。与瑞戈非尼单药治疗组（5.1个月，P=0.049）相比，瑞戈非尼联合PD-1抑制剂组的中位PFS明显更长（11.5个月）。
本研究表明，瑞戈非尼与PD-1抑制剂联合使用可能为一线治疗后出现进展的HCC患者提供显著的临床益处。有必要在更大队列的真实世界研究中进行进一步分析以证实这些发现。
