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Background <p>Hepatectomy is considered advisable for some patients with intermediate or advanced hepatocellular carcinoma (HCC). The efficacy and safety of neoadjuvant transarterial chemoembolization with tyrosine kinase and immune checkpoint inhibitors (neoadjuvant triple therapy) for these patients remain unclear.</p> Methods <p>583 patients who met the resectable criteria and were assigned to receive neoadjuvant triple therapy (<i>n</i> = 205) or direct hepatectomy (<i>n</i> = 378) at 20 Chinese medical centers (2019–2023) were retrospectively compared in terms of overall survival (OS), event-free survival (EFS), adverse events, and postoperative complications. The subgroup stratified include<Emphasis Type="Underline">s</Emphasis> 106 patients who underwent neoadjuvant triple therapy followed by hepatectomy or 99 patients who received it without subsequent hepatectomy.</p> Results <p>Compared to patients who undergo direct hepatectomy, those who receive neoadjuvant triple therapy show significantly higher OS (hazard ratio [HR] 0.70, 95%CI 0.53–0.92) and significantly longer median EFS (19.7 vs 10.9 months). Similar results are obtained after propensity score matching (PSM). Among patients who undergo hepatectomy, those with prior neoadjuvant triple therapy have significantly better OS (HR 0.45, 95%CI 0.31–0.66) and EFS (HR 0.49, 95%CI 0.38–0.63) than those with direct hepatectomy. Similar results are obtained after PSM. Among patients who receive neoadjuvant triple therapy, OS is significantly better among those who subsequently underwent hepatectomy (HR 0.40, 95%CI 0.24-0.67). neoadjuvant triple therapy results in a complete pathologic response rate of 34.0%. However, the regimen is associated with high rates of serious adverse events and postoperative complications, including hepatic insufficiency, bile leakage, and ascites.</p> Conclusions <p>Neoadjuvant triple therapy offers OS and EFS benefits for patients with resectable intermediate or advanced HCC, but is associated with an increased risk of adverse events and postoperative complications.</p>

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Neoadjuvant transarterial chemoembolization with tyrosine kinase and immune checkpoint inhibitors improves survival from resectable hepatocellular carcinoma in a multicenter, retrospective study (GUIDANCE002)

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  • 发表日期：

    2026年3月12日

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  • Da-Long Yang,
  • Chuang Qin,
  • Ning Peng,
  • Shao-Ping Liu,
  • Ya-Qun Yu,
  • Jun-Liang Nong,
  • Fan-Jian Zeng,
  • Ze Su,
  • Yong-Yu Yang,
  • Fu-Quan Yang,
  • Mian-Jing Li,
  • Ming-Song Wu,
  • Xue-Yao Wang,
  • Yong-Rong Liang,
  • Fu-Xin Li,
  • Shu-Chang Chen,
  • Yong-Cheng Lai,
  • Qing-Qing Pang,
  • Teng-Meng Zhong,
  • Yi-He Yan,
  • Ying-Yu Zhang,
  • Lin Ye,
  • Xiao-Feng Dong,
  • Wen-Feng Li,
  • Jie Liu,
  • Hong-Bing Yao,
  • Pei-Sheng Wu,
  • Xian-Shuang Mao,
  • Jun-Jie Ou,
  • Yao-Zhi Chen,
  • Guo-Dong Wang,
  • Kang Chen,
  • Zhu-Jian Deng,
  • Jia-Yong Su,
  • Guo-Gao Qiu,
  • Zhi-Dong Liu,
  • Jin-Min Wang,
  • Yi Zhang,
  • Xiu-Mei Liang,
  • Bang-De Xiang,
  • Liang Ma,
  • Jian-Hong Zhong,
  • Jian-Hong Zhong,
  • Liang Ma,
  • Bang-De Xiang,
  • Da-Long Yang,
  • Ya-Qun Yu,
  • Lin Ye,
  • Shu-Qun Li,
  • Shao-Ping Liu,
  • Zhen Liu,
  • Chuang Qin,
  • Zhi-Jun Jiang,
  • Ning Peng,
  • Jia-Liang Wei,
  • Ze Su,
  • Jun-Liang Nong,
  • Yi-He Yan,
  • Kang Chen,
  • Xiao-Feng Dong,
  • Yong-Yu Yang,
  • Jun-Jie Ou,
  • Shu-Chang Chen,
  • Ying Xue Wu,
  • Hong-Bing Yao,
  • Xue-Yao Wang,
  • Fan-Jian Zeng,
  • Jie Liu,
  • Mian-Jing Li,
  • Pei-Sheng Wu,
  • Yong-Rong Liang,
  • Yao-Zhi Chen,
  • Yong-Cheng Lai,
  • Ming-Song Wu,
  • Wen-Feng Li,
  • Tian-Man Li,
  • Fu-Quan Yang,
  • Zhi-Yin Liang,
  • Teng-Meng Zhong,
  • Xian-Shuang Mao,
  • Fu-Xin Li,
  • Teng-Xian Zhou,
  • Guang-Cai Zheng,
  • Yao-Ming Wang,
  • Min Luo,
  • Guo-Dong Wang,
  • Qing-Qing Pang,
  • Xiao-Kai Shen,
  • Lei Liu,
  • Si-Cong Lu,
  • Wen-Hai He,
  • Xian-Jian Wu,
  • Lin-Hong Xie,
  • Jian-Yuan Meng,
  • Zhi-Cheng Li,
  • Ting Quan

摘要

Background

Hepatectomy is considered advisable for some patients with intermediate or advanced hepatocellular carcinoma (HCC). The efficacy and safety of neoadjuvant transarterial chemoembolization with tyrosine kinase and immune checkpoint inhibitors (neoadjuvant triple therapy) for these patients remain unclear.

Methods

583 patients who met the resectable criteria and were assigned to receive neoadjuvant triple therapy (n = 205) or direct hepatectomy (n = 378) at 20 Chinese medical centers (2019–2023) were retrospectively compared in terms of overall survival (OS), event-free survival (EFS), adverse events, and postoperative complications. The subgroup stratified includes 106 patients who underwent neoadjuvant triple therapy followed by hepatectomy or 99 patients who received it without subsequent hepatectomy.

Results

Compared to patients who undergo direct hepatectomy, those who receive neoadjuvant triple therapy show significantly higher OS (hazard ratio [HR] 0.70, 95%CI 0.53–0.92) and significantly longer median EFS (19.7 vs 10.9 months). Similar results are obtained after propensity score matching (PSM). Among patients who undergo hepatectomy, those with prior neoadjuvant triple therapy have significantly better OS (HR 0.45, 95%CI 0.31–0.66) and EFS (HR 0.49, 95%CI 0.38–0.63) than those with direct hepatectomy. Similar results are obtained after PSM. Among patients who receive neoadjuvant triple therapy, OS is significantly better among those who subsequently underwent hepatectomy (HR 0.40, 95%CI 0.24-0.67). neoadjuvant triple therapy results in a complete pathologic response rate of 34.0%. However, the regimen is associated with high rates of serious adverse events and postoperative complications, including hepatic insufficiency, bile leakage, and ascites.

Conclusions

Neoadjuvant triple therapy offers OS and EFS benefits for patients with resectable intermediate or advanced HCC, but is associated with an increased risk of adverse events and postoperative complications.