Application of immune checkpoint inhibitors in hepatocellular carcinoma: a landscape analysis of clinical trial databases
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Junjie Cao1,2, Yanru Chen1,2, Lichuan Zhang1,2
1Department of Emergency, Affiliated Hospital of North Sichuan Medical College, Nanchong, Sichuan, China.
Background:
Hepatocellular carcinoma (HCC) is a leading global malignant tumor with poor prognosis. Immune checkpoint inhibitors (ICIs) have become a breakthrough treatment for HCC, but a systematic landscape analysis of global ICI clinical trials is lacking.
Methods:
Four international clinical trial databases were systematically searched up to February 14, 2026. After screening, 132 eligible trials were included and analyzed for geographic distribution, molecular targets, clinical phases, trial status and result publication rate using R 4.5.1.
Results:
Trials displayed a China-US dual-core pattern (95 vs. 16). PD-1/PD-L1 inhibitors were the predominant targets, while CTLA-4 inhibitor trials were scarce. Phase II was the most frequent clinical phase (59 trials). Over 80% were interventional studies; most were recruiting or of unknown status, with only 6 terminated or withdrawn. The overall result publication rate was extremely low, with 71 PD1-targeting trials and 71.4% CTLA-4 trials were unpublished.
Conclusion:
Global HCC ICIs trials are highly concentrated geographically and by target, with stable research progress. However, major challenges include low result translation efficiency, unbalanced target development, insufficient early/late-phase trials and uneven global collaboration. Targeted optimization is needed to promote clinical translation.
Insights
Global clinical trials for hepatocellular carcinoma (HCC) using immune checkpoint inhibitors (ICIs) show concentrated research, but face challenges in result publication and target development. Optimization is needed to improve clinical translation for this liver cancer.
Area of Science:
- Oncology
- Clinical Trials
- Immunotherapy
Background:
- Hepatocellular carcinoma (HCC) is a significant global cancer with a poor prognosis.
- Immune checkpoint inhibitors (ICIs) represent a promising treatment for HCC.
- A comprehensive analysis of global ICI clinical trials in HCC is currently lacking.
Purpose of the Study:
- To systematically analyze the global landscape of clinical trials for immune checkpoint inhibitors in hepatocellular carcinoma.
- To identify trends in geographic distribution, molecular targets, clinical phases, and trial status.
- To assess the result publication rates of these trials.
Main Methods:
- Systematic search of four international clinical trial databases up to February 14, 2026.
- Inclusion and analysis of 132 eligible trials.
- Data analysis using R 4.5.1 for geographic distribution, molecular targets, clinical phases, trial status, and publication rates.
Main Results:
- Trials exhibit a dual-core geographic pattern, primarily concentrated in China and the US.
- PD-1/PD-L1 inhibitors are the dominant targets, with limited trials for CTLA-4 inhibitors.
- Phase II trials are most common, and the overall result publication rate is notably low, particularly for PD1 and CTLA-4 targeting trials.
Conclusions:
- Global HCC ICI trials demonstrate geographical and target-specific concentration with steady research progression.
- Key challenges include low translation efficiency, unbalanced target development, and insufficient early/late-phase trials.
- Strategic optimization is essential to enhance clinical translation and global collaboration in HCC ICI research.
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