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Peer-reviewedCC BY-NCJournal of hepatocellular carcinoma

The Efficacy and Safety of Combined Locoregional and Systemic Therapy versus Systemic Therapy Alone in Patients with Hepatocellular Carcinoma and Portal Vein Tumor Thrombosis: A Multicenter Real-World Retrospective Cohort Study.

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7 affiliations
  1. 1Department of Hepatic Surgery II, Third Affiliated Hospital of Naval Medical University (Eastern Hepatobiliary Surgery Hospital), Shanghai, People's Republic of China.
  2. 2Department of Hepatopancreatobiliary Surgery, Mengchao Hepatobiliary Hospital of Fujian Medical University, Fuzhou, People's Republic of China.
  3. 3Hepato-Pancreato-Biliary Center, Zhongda Hospital, School of Medicine, Southeast University, Nanjing, People's Republic of China.
  4. 4Department of Hepatobiliary Surgery, The Fifth Clinical Medical College of Henan University of Chinese Medicine (Zhengzhou People's Hospital), Zhengzhou, Henan, People's Republic of China.
  5. 5Department of Hepatic Surgery IV, Third Affiliated Hospital of Naval Medical University (Eastern Hepatobiliary Surgery Hospital), Shanghai, People's Republic of China.
  6. 6Department of Liver Surgery and Transplantation, Liver Cancer Institute and Zhongshan Hospital, Fudan University, Shanghai, People's Republic of China.
  7. 7Department of General Surgery, Jinling Hospital, Affiliated Hospital of Medical School, Nanjing University, Nanjing, People's Republic of China.

Abstract

Background

To compare the efficacy and safety of combined locoregional and systemic therapy vs systemic therapy alone in patients with hepatocellular carcinoma (HCC) and portal vein tumor thrombosis (PVTT).Methods

This multicenter retrospective study included 337 patients with HCC and PVTT who received systemic therapy alone (n=121) or combined locoregional and systemic therapy (n=216). Propensity score matching (PSM) was performed to reduce baseline imbalances. Overall survival (OS), progression-free survival (PFS), tumor response and adverse events (AEs) were compared. When the proportional hazards assumption was violated, time-varying Cox and restricted mean survival time (RMST) analyses were performed.Results

In the whole cohort, combined therapy was associated with a lower hazard of death (hazard ratio [HR]=0.57, 95% confidence interval [CI]=0.38-0.86, P=0.007), and this association remained after PSM (HR=0.54, 95% CI=0.34-0.86, P=0.009). Conventional Kaplan-Meier analysis did not show a significant PFS difference after PSM (median PFS, 12.97 vs 10.20 months; P=0.394); however, the proportional hazards assumption for PFS was violated. RMST analyses showed significant cumulative PFS advantages favoring combined therapy at 12 months (+1.48 months; P=0.008) and 16 months (+1.69 months; P=0.010). After PSM, the objective response rate according to the modified Response Evaluation Criteria in Solid Tumors (mRECIST) was higher with combined therapy (61.1% vs 45.4%, P=0.029). In patients with Vp3-4 PVTT, combined therapy was associated with a lower hazard of death (HR=0.60, 95% CI=0.39-0.93, P=0.023). Grade 3-4 AEs were comparable between groups (29.2% vs 31.4%, P=0.759).Conclusion

Combined locoregional and systemic therapy was associated with longer OS and an early, time-dependent PFS advantage compared with systemic therapy alone in patients with HCC and PVTT, with a more apparent OS benefit signal in Vp3-4 disease and no clear increase in grade 3-4 AEs.

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BibTeX
@article{liu2026efficacy,
  title = {{The Efficacy and Safety of Combined Locoregional and Systemic Therapy versus Systemic Therapy Alone in Patients with Hepatocellular Carcinoma and Portal Vein Tumor Thrombosis: A Multicenter Real-World Retrospective Cohort Study.}},
  author = {Jianwei Liu and Yuxuan Lin and Kongying Lin and Kang Liang and Zhengqing Lei and Hui Xue and Huifeng Wang and Yong Xia and Hui-Chuan Sun and Anfeng Si and Yongyi Zeng and Kui Wang and China Liver Cancer Study Group Young Investigators (CLEAP)},
  journal = {Journal of hepatocellular carcinoma},
  year = {2026},
  month = oct,
  doi = {10.2147/jhc.s624104},
  url = {https://europepmc.org/article/MED/42921173},
  note = {CC BY-NC},
}
RIS
TY  - JOUR
TI  - The Efficacy and Safety of Combined Locoregional and Systemic Therapy versus Systemic Therapy Alone in Patients with Hepatocellular Carcinoma and Portal Vein Tumor Thrombosis: A Multicenter Real-World Retrospective Cohort Study.
AU  - Jianwei Liu
AU  - Yuxuan Lin
AU  - Kongying Lin
AU  - Kang Liang
AU  - Zhengqing Lei
AU  - Hui Xue
AU  - Huifeng Wang
AU  - Yong Xia
AU  - Hui-Chuan Sun
AU  - Anfeng Si
AU  - Yongyi Zeng
AU  - Kui Wang
AU  - China Liver Cancer Study Group Young Investigators (CLEAP)
PY  - 2026
DA  - 2026/10/16
JO  - Journal of hepatocellular carcinoma
DO  - 10.2147/jhc.s624104
UR  - https://europepmc.org/article/MED/42921173
AB  - Background  To compare the efficacy and safety of combined locoregional and systemic therapy vs systemic therapy alone in patients with hepatocellular carcinoma (HCC) and portal vein tumor thrombosis (PVTT).Methods  This multicenter retrospective study included 337 patients with HCC and PVTT who received systemic therapy alone (n=121) or combined locoregional and systemic therapy (n=216). Propensity score matching (PSM) was performed to reduce baseline imbalances. Overall survival (OS), progression-free survival (PFS), tumor response and adverse events (AEs) were compared. When the proportional hazards assumption was violated, time-varying Cox and restricted mean survival time (RMST) analyses were performed.Results  In the whole cohort, combined therapy was associated with a lower hazard of death (hazard ratio [HR]=0.57, 95% confidence interval [CI]=0.38-0.86, P=0.007), and this association remained after PSM (HR=0.54, 95% CI=0.34-0.86, P=0.009). Conventional Kaplan-Meier analysis did not show a significant PFS difference after PSM (median PFS, 12.97 vs 10.20 months; P=0.394); however, the proportional hazards assumption for PFS was violated. RMST analyses showed significant cumulative PFS advantages favoring combined therapy at 12 months (+1.48 months; P=0.008) and 16 months (+1.69 months; P=0.010). After PSM, the objective response rate according to the modified Response Evaluation Criteria in Solid Tumors (mRECIST) was higher with combined therapy (61.1% vs 45.4%, P=0.029). In patients with Vp3-4 PVTT, combined therapy was associated with a lower hazard of death (HR=0.60, 95% CI=0.39-0.93, P=0.023). Grade 3-4 AEs were comparable between groups (29.2% vs 31.4%, P=0.759).Conclusion  Combined locoregional and systemic therapy was associated with longer OS and an early, time-dependent PFS advantage compared with systemic therapy alone in patients with HCC and PVTT, with a more apparent OS benefit signal in Vp3-4 disease and no clear increase in grade 3-4 AEs.
SN  - 2253-5969
ER  -