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Liver cancer

Nivolumab and ipilimumab as neoadjuvant therapy for potentially resectable HCC

Nature Reviews Gastroenterology & Hepatology (2025)Cite this article

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A single-arm, open-label clinical trial in Taiwan (NCT03510871) has assessed the efficacy and safety of neoadjuvant immune checkpoint inhibitor-based combination therapy in patients with potentially resectable hepatocellular carcinoma (HCC). Patients (n = 43 enrolled; 37 men and 6 women) received 3 mg/kg nivolumab plus 1 mg/kg ipilimumab every 3 weeks, and tumour response was assessed after the second and fourth cycles. Of these patients, 24 subsequently underwent surgery and 8 had a major pathological response.

Analysis of tumour samples indicated that treatment-induced tertiary lymphoid structure formation in resected tumours was correlated with antitumour immunity, and in vivo mouse observations supported the role of tertiary lymphoid structure formation in mediating immunotherapy treatment efficacy. In addition, biomarker analysis of blood samples (pre-treatment and post-treatment) suggested that monitoring peripheral blood T cell exhaustion may facilitate prediction of response to immunotherapy.

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  1. Nature Reviews Gastroenterology & Hepatology http://www.nature.com/nrgastro/

    Jordan Hindson

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  1. Jordan Hindson
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Correspondence to Jordan Hindson.

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Hindson, J. Nivolumab and ipilimumab as neoadjuvant therapy for potentially resectable HCC. Nat Rev Gastroenterol Hepatol (2025). https://doi.org/10.1038/s41575-025-01136-x

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  • DOI: https://doi.org/10.1038/s41575-025-01136-x

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