Phase Ib Study of Lenvatinib Plus Pembrolizumab in Patients With Unresectable Hepatocellular Carcinoma
Open accessAbstract
PURPOSE: The immunomodulatory effect of lenvatinib (a multikinase inhibitor) on tumor microenvironments may contribute to antitumor activity when combined with programmed death receptor-1 (PD-1) signaling inhibitors in hepatocellular carcinoma (HCC). We report results from a phase Ib study of lenvatinib plus pembrolizumab (an anti–PD-1 antibody) in unresectable HCC (uHCC). PATIENTS AND METHODS: In this open-label multicenter study, patients with uHCC received lenvatinib (bodyweight ≥ 60 kg, 12 mg; < 60 kg, 8 mg) orally daily and pembrolizumab 200 mg intravenously on day 1 of a 21-day cycle. The study included a dose-limiting toxicity (DLT) phase and an expansion phase (first-line patients). Primary objectives were safety/tolerability (DLT phase), and objective response rate (ORR) and duration of response (DOR) by modified RECIST (mRECIST) and RECIST version 1.1 (v1.1) per independent imaging review (IIR; expansion phase). RESULTS: A total of 104 patients were enrolled. No DLTs were reported (n = 6) in the DLT phase; 100 patients (expansion phase; included n = 2 from DLT phase) had received no prior systemic therapy and had Barcelona Clinic Liver Cancer stage B (n = 29) or C disease (n = 71). At data cutoff, 37% of patients remained on treatment. Median duration of follow-up was 10.6 months (95% CI, 9.2 to 11.5 months). Confirmed ORRs by IIR were 46.0% (95% CI, 36.0% to 56.3%) per mRECIST and 36.0% (95% CI, 26.6% to 46.2%) per RECIST v1.1. Median DORs by IIR were 8.6 months (95% CI, 6.9 months to not estimable [NE]) per mRECIST and 12.6 months (95% CI, 6.9 months to NE) per RECIST v1.1. Median progression-free survival by IIR was 9.3 months per mRECIST and 8.6 months per RECIST v1.1. Median overall survival was 22 months. Grade ≥ 3 treatment-related adverse events occurred in 67% (grade 5, 3%) of patients. No new safety signals were identified. CONCLUSION: Lenvatinib plus pembrolizumab has promising antitumor activity in uHCC. Toxicities were manageable, with no unexpected safety signals.
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
The combination of anti-PD-1 and lenvatinib raises the HCC patient response rate from 17% with anti-PD-1 alone to 45%.
“The combination of anti-PD-1 and lenvatinib, a multi-target tyrosine kinase inhibitor targeting vascular endothelial growth factor (VEGF) receptors, raises the HCC patient response rate from 17% with anti-PD-1 alone to 45%.”
From Contrasting cytotoxic and regulatory T cell responses underlying distinct clinical outcomes to anti-PD-1 plus lenvatinib therapy in cancer (2025) · cites this paper as [11]
The Results > Efficacy section reports a 46% confirmed response rate by mRECIST for lenvatinib plus pembrolizumab, close to the claimed 45%. But this was a single-arm study: our copy does not report a 17% anti-PD-1-only response rate or establish that adding lenvatinib raised the rate from 17% to 45%. (LLM, conf=0.90)
Experimental — accuracy not yet measuredICI combinations can provide a higher tumor response rate and better survival.
“ICI combinations can provide a higher tumor response rate and better survival and may be a potential bias in this microbiota study.5 6”
From Gut microbiota and metabolites associate with outcomes of immune checkpoint inhibitor–treated unresectable hepatocellular carcinoma (2022) · cites this paper as [6]
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Cites
Bray, F., Ferlay, J., Soerjomataram, I., Siegel, R. L., Torre, L. A., & Jemal, A. (2018) · CA: A Cancer Journal for Clinicians · doi:10.3322/caac.21492 · Cited by 84,109 · see citing works
Sorafenib in advanced hepatocellular carcinoma
Llovet, J. M., Ricci, S., Mazzaferro, V., Hilgard, P., Gane, E., Blanc, J.-F., de Oliveira, A. C., Santoro, A., Raoul, J.-L., Forner, A., et al. (2008) · New England Journal of Medicine · doi:10.1056/nejmoa0708857 · Cited by 13,097 · cited 3× · see citing works
“AST increased 20 (20) 4 (4) 5 (5) 11 (11) Proteinuria 20 (20) 9 (9) 7 (7) 4 (4) Asthenia 19 (19) 4 (4) 10 (10) 5 (5)”
BackgroundSelf-citation
Flagged first, then by citations
Research works printed but not linked: 24
- [2]Llovet JM Zucman-Rossi J Pikarsky E et al Hepatocellular carcinoma Nat Rev Dis Primers 2 16018 2016 27158749
- [3]Llovet JM Montal R Sia D et al Molecular therapies and precision medicine for hepatocellular carcinoma Nat Rev Clin Oncol 15 599 616 2018 30061739 PMC12452113
- [5]Cheng AL Kang YK Chen Z et al Efficacy and safety of sorafenib in patients in the Asia-Pacific region with advanced hepatocellular carcinoma: A phase III randomised, double-blind, placebo-controlled trial Lancet Oncol 10 25 34 2009 19095497
- [6]Matsui J Funahashi Y Uenaka T et al Multi-kinase inhibitor E7080 suppresses lymph node and lung metastases of human mammary breast tumor MDA-MB-231 via inhibition of vascular endothelial growth factor-receptor (VEGF-R) 2 and VEGF-R3 kinase Clin Cancer Res 14 5459 5465 2008 18765537
- [7]Matsui J Yamamoto Y Funahashi Y et al E7080, a novel inhibitor that targets multiple kinases, has potent antitumor activities against stem cell factor producing human small cell lung cancer H146, based on angiogenesis inhibition Int J Cancer 122 664 671 2008 17943726
- [8]Okamoto K Kodama K Takase K et al Antitumor activities of the targeted multi-tyrosine kinase inhibitor lenvatinib (E7080) against RET gene fusion-driven tumor models Cancer Lett 340 97 103 2013 23856031
- [9]Yamamoto Y Matsui J Matsushima T et al Lenvatinib, an angiogenesis inhibitor targeting VEGFR/FGFR, shows broad antitumor activity in human tumor xenograft models associated with microvessel density and pericyte coverage Vasc Cell 6 18 2014 25197551 PMC4156793
- [10]Kudo M Finn RS Qin S et al Lenvatinib versus sorafenib in first-line treatment of patients with unresectable hepatocellular carcinoma: A randomised phase 3 non-inferiority trial Lancet 391 1163 1173 2018 29433850
- [11]Lencioni R Llovet JM Modified RECIST (mRECIST) assessment for hepatocellular carcinoma Semin Liver Dis 30 52 60 2010 20175033 PMC12268942
- [12]Prieto J Melero I Sangro B Immunological landscape and immunotherapy of hepatocellular carcinoma Nat Rev Gastroenterol Hepatol 12 681 700 2015 26484443
- [13]Ringelhan M Pfister D O’Connor T et al The immunology of hepatocellular carcinoma Nat Immunol 19 222 232 2018 29379119
- [14]El-Khoueiry AB Sangro B Yau T et al Nivolumab in patients with advanced hepatocellular carcinoma (CheckMate 040): An open-label, non-comparative, phase 1/2 dose escalation and expansion trial Lancet 389 2492 2502 2017 28434648 PMC7539326
- [17]Zhu AX, Finn RS, Edeline J, et al: Pembrolizumab in patients with advanced hepatocellular carcinoma previously treated with sorafenib (KEYNOTE-224): A non-randomised, open-label phase 2 trial. Lancet Oncol 19:940-952, 2018 [Erratum: Lancet Oncol 19:e440, 2018] 29875066
- [18]Finn RS Ryoo BY Merle P et al Pembrolizumab as second-line therapy in patients with advanced hepatocellular carcinoma in KEYNOTE-240: A randomized, double-blind, phase III trial J Clin Oncol 38 193 202 2020 31790344
- [19]Schmidt EV Developing combination strategies using PD-1 checkpoint inhibitors to treat cancer Semin Immunopathol 41 21 30 2019 30374524 PMC6323091
- [23]Fukumura D Kloepper J Amoozgar Z et al Enhancing cancer immunotherapy using antiangiogenics: Opportunities and challenges Nat Rev Clin Oncol 15 325 340 2018 29508855 PMC5921900
- [24]Kato Y Tabata K Kimura T et al Lenvatinib plus anti-PD-1 antibody combination treatment activates CD8+ T cells through reduction of tumor-associated macrophage and activation of the interferon pathway PLoS One 14 e0212513 2019 30811474 PMC6392299
- [25]Kimura T Kato Y Ozawa Y et al Immunomodulatory activity of lenvatinib contributes to antitumor activity in the Hepa1-6 hepatocellular carcinoma model Cancer Sci 109 3993 4002 2018 30447042 PMC6272102
- [26]Jiang JF Lao YC Yuan BH et al Treatment of hepatocellular carcinoma with portal vein tumor thrombus: Advances and challenges Oncotarget 8 33911 33921 2017 28430610 PMC5464922
- [27]Schemper M Smith TL A note on quantifying follow-up in studies of failure time Control Clin Trials 17 343 346 1996 8889347
- [28]Faivre S Rimassa L Finn RS Molecular therapies for HCC: Looking outside the box J Hepatol 72 342 352 2020 31954496
- [30]Finn RS, Qin S, Ikeda M, et al: Atezolizumab plus bevacizumab in unresectable hepatocellular carcinoma. N Engl J Med 382:1894-1905, 2020 32402160
- [32]National Comprehensive Cancer Network: NCCN Guidelines: Hepatobiliary cancers—Version 1.2020. https://www.nccn.org/professionals/physician_gls/pdf/hepatobiliary.pdf PMC5557008 28476736
- [34]Ikeda K Kudo M Kawazoe S et al Phase 2 study of lenvatinib in patients with advanced hepatocellular carcinoma J Gastroenterol 52 512 519 2017 27704266 PMC5357473
Cited by
Sorted by citations
Immunotherapies for hepatocellular carcinoma
Llovet, J. M., Castet, F., Heikenwalder, M., Maini, M. K., Mazzaferro, V., Pinato, D. J., Pikarsky, E., Zhu, A. X., & Finn, R. S. (2022) · Nature Reviews Clinical Oncology · doi:10.1038/s41571-021-00573-2 · Cited by 1,843 · see citing works
Self-citationLocoregional therapies in the era of molecular and immune treatments for hepatocellular carcinoma
Llovet, J. M., De Baere, T., Kulik, L., Haber, P. K., Greten, T. F., Meyer, T., & Lencioni, R. (2021) · Nature Reviews Gastroenterology & Hepatology · doi:10.1038/s41575-020-00395-0 · Cited by 1,044 · see citing works
Self-citationQin, S., Chan, S. L., Gu, S., Bai, Y., Ren, Z., Lin, X., Chen, Z., Jia, W., Jin, Y., Guo, Y., et al. (2023) · The Lancet · doi:10.1016/s0140-6736(23)00961-3 · Cited by 746 · see citing works
Kudo, M., Kawamura, Y., Hasegawa, K., Tateishi, R., Kariyama, K., Shiina, S., Toyoda, H., Imai, Y., Hiraoka, A., Ikeda, M., et al. (2021) · Liver Cancer · doi:10.1159/000514174 · Cited by 678 · see citing works
Self-citationKelley, R. K., Rimassa, L., Cheng, A.-L., Kaseb, A., Qin, S., Zhu, A. X., Chan, S. L., Melkadze, T., Sukeepaisarnjaroen, W., Breder, V., et al. (2022) · The Lancet Oncology · doi:10.1016/s1470-2045(22)00326-6 · Cited by 535 · see citing works
Self-citationMolecular pathogenesis and systemic therapies for hepatocellular carcinoma
Llovet, J. M., Pinyol, R., Kelley, R. K., El-Khoueiry, A., Reeves, H. L., Wang, X. W., Gores, G. J., & Villanueva, A. (2022) · Nature Cancer · doi:10.1038/s43018-022-00357-2 · Cited by 498 · see citing works
Self-citationLlovet, J. M., Kudo, M., Merle, P., Meyer, T., Qin, S., Ikeda, M., Xu, R., Edeline, J., Ryoo, B.-Y., Ren, Z., et al. (2023) · The Lancet Oncology · doi:10.1016/s1470-2045(23)00469-2 · Cited by 469 · see citing works
Self-citationKudo, M., Ren, Z., Guo, Y., Han, G., Lin, H., Zheng, J., Ogasawara, S., Kim, J. H., Zhao, H., Li, C., et al. (2025) · The Lancet · doi:10.1016/s0140-6736(24)02575-3 · Cited by 342 · see citing works
Self-citationHack, S. P., Zhu, A. X., & Wang, Y. (2020) · Frontiers in Immunology · doi:10.3389/fimmu.2020.598877 · Cited by 286 · see citing works
Self-citationXia, Y., Tang, W., Qian, X., Li, X., Cheng, F., Wang, K., Zhang, F., Zhang, C., Li, D., Song, J., et al. (2022) · Journal for ImmunoTherapy of Cancer · doi:10.1136/jitc-2022-004656 · Cited by 228 · see citing works
Biomarkers for immunotherapy of hepatocellular carcinoma
Greten, T. F., Villanueva, A., Korangy, F., Ruf, B., Yarchoan, M., Ma, L., Ruppin, E., & Wang, X. W. (2023) · Nature Reviews Clinical Oncology · doi:10.1038/s41571-023-00816-4 · Cited by 227 · see citing works
Lee, P.-C., Wu, C.-J., Hung, Y.-W., Lee, C. J., Chi, C.-T., Lee, I.-C., Yu-Lun, K., Chou, S.-H., Luo, J.-C., Hou, M.-C., et al. (2022) · Journal for ImmunoTherapy of Cancer · doi:10.1136/jitc-2022-004779 · Cited by 217 · see citing works
Zhang, S., Yuan, L., Danilova, L., Mo, G., Zhu, Q., Deshpande, A., Bell, A. T. F., Elisseeff, J., Popel, A. S., Anders, R. A., et al. (2023) · Genome Medicine · doi:10.1186/s13073-023-01218-y · Cited by 140 · see citing works
NASH and Hepatocellular Carcinoma: Immunology and Immunotherapy
Pinter, M., Pinato, D. J., Ramadori, P., & Heikenwalder, M. (2023) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-21-1258 · Cited by 103 · see citing works
Guo, X., Nie, H., Zhang, W., Li, J., Ge, J., Xie, B., Hu, W., Zhu, Y., Zhong, N., Zhang, X., et al. (2025) · Cancer Cell · doi:10.1016/j.ccell.2025.01.001 · Cited by 100 · see citing works
CXCL12+ tumor-associated endothelial cells promote immune resistance in hepatocellular carcinoma
Lu, Y., Liu, Y., Zuo, X., Li, G., Wang, J., Liu, J., Wang, X., Wang, S., Zhang, W., Zhang, K., et al. (2025) · Journal of Hepatology · doi:10.1016/j.jhep.2024.09.044 · Cited by 82 · see citing works
Kudo, M. (2019) · Liver Cancer · doi:10.1159/000503254 · Cited by 70 · see citing works
Self-citationDing, Z., Dong, Z., Chen, Z., Hong, J., Yan, L., Li, H., Yao, S., Yan, Y., Yang, Y., Yang, C., et al. (2021) · Frontiers in Immunology · doi:10.3389/fimmu.2021.733530 · Cited by 47 · see citing works
- Authors
- Finn, R. S., Ikeda, M., Zhu, A. X., Sung, M. W., Baron, A. D., Kudo, M., Okusaka, T., Kobayashi, M., Kumada, H., Kaneko, S., Pracht, M., Mamontov, K., Meyer, T., Kubota, T., Dutcus, C. E., Saito, K., Siegel, A. B., Dubrovsky, L., Mody, K., & Llovet, J. M.
- Affiliations
- University of California, Los Angeles (US)
- National Cancer Center Hospital East (JP)
- Harvard University (US)
- Tisch Cancer Institute (US)
- Sutter Health (US)
- Kindai University Hospital (JP)
- National Cancer Center Hospital (JP)
- Toranomon Hospital (JP)
- Kanazawa University (JP)
- Centre Eugène Marquis (FR)
- Altay Regional Oncological Hospital, Barnaul, Russian Federation
- National Health Service (GB)
- Eisai (Japan) (JP)
- Eisai (United States) (US)
- Merck & Co., Inc., Rahway, NJ, USA (United States) (US)
- Hospital Clínic de Barcelona (ES)
- Corresponding author
- Llovet, Josep M.
- ORCID
- Venue
- Journal of Clinical Oncology
- Published
- 2020-09-10
- Volume
- 38
- Issue
- 26
- Pages
- 2960-2970
- ISSN
- 0732-183X (ISSN-L)
- 1527-7755
- PMC
- PMC7479760
- OpenAlex
- W3045387409
- Cited by
- 1,167
- References
- 2
- Citations by year
- 2026: 67 · 2025: 132 · 2024: 197 · 2023: 257 · 2022: 262 · 2021: 197 · 2020: 42 · 2019: 8 · 2018: 2
- FWCI
- 111.73
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- cc-by-nc-nd
- Locations
- Journal of Clinical Oncology (publishedVersion, cc-by-nc-nd)
- PubMed Central (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- Dipòsit Digital de la Universitat de Barcelona (Universitat de Barcelona) (submittedVersion, cc-by-nc-nd)
- UCL Discovery (University College London) (submittedVersion)
- Funders
- Cancer Research UK (26813)
- Topics
- Health Sciences › Medicine › Hepatology › Hepatocellular Carcinoma Treatment and Prognosis
- Health Sciences › Medicine › Oncology › Cancer Immunotherapy and Biomarkers
- Health Sciences › Medicine › Oncology › Colorectal Cancer Treatments and Studies
- Keywords
- lenvatinib, pembrolizumab, hepatocellular carcinoma, unresectable liver cancer, PD-1 inhibitors, multikinase inhibitor, combination immunotherapy, tumor microenvironment, objective response rate, duration of response, progression-free survival, dose-limiting toxicity, first-line therapy, immune-related adverse events, predictive biomarkers
- MeSH terms
- Progression-Free Survival, Aged, Aged, 80 and over, Antineoplastic Combined Chemotherapy Protocols, Female, Carcinoma, Hepatocellular, Humans, Liver Neoplasms, Male, Middle Aged, Neoplasm Staging, Phenylurea Compounds, Quinolines, Time Factors, Disease Progression, Antibodies, Monoclonal, Humanized
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review