Journal Article

DKFZ-2025-02488

http://join2-wiki.gsi.de/foswiki/pub/Main/Artwork/join2_logo100x88.png Case Report: A neoantigen-targeting peptide vaccine combined with checkpoint inhibition induces tumor regression and long-term remission in a pediatric patient with metastatic hepatocellular carcinoma.

Amorelli, G.DKFZ* ; Rabsteyn, A. ; Maier, C.-P. ; Trautner, F. ; Holzer, U. ; Schäfer, J. F. ; Ebinger, M. ; Handgretinger, R. ; Nahnsen, S. ; Rammensee, H.-G. ; Lang, P.DKFZ*

2025
Frontiers Media Lausanne

Abstract: Pediatric hepatocellular carcinoma (HCC) is a rare and aggressive malignancy with limited treatment options and poor prognosis, highlighting the need for innovative therapeutic strategies. Neoantigen-targeting peptide vaccination is a promising treatment approach with potential for combination therapy with checkpoint inhibition (CPI). Here, we present a case study of a pediatric patient with metastatic HCC treated with a neoantigen-derived peptide vaccine combined with CPI therapy after disease recurrence. Immunomonitoring revealed robust vaccine-induced T-cell responses, further enhanced by CPI. T-cell cloning and T-cell receptor (TCR) sequencing confirmed neoepitope specificity and clonality of the vaccine-induced T-cell response. Following immunotherapy, the inoperable metastasis regressed completely, with no further intervention. A subsequent metastasis was surgically resected, and the patient has remained in complete remission since, with an overall survival (OS) of 13 years. These findings underscore the potential of personalized peptide vaccination and demonstrate the feasibility and efficacy of combinatorial strategies in optimizing therapeutic outcome in pediatric HCC. Importantly, this case illustrates a uniquely durable remission in pediatric metastatic HCC, exceeding survival outcomes reported in previous vaccine or CPI monotherapy studies.

Keyword(s): Humans (MeSH) ; Carcinoma, Hepatocellular: immunology (MeSH) ; Carcinoma, Hepatocellular: therapy (MeSH) ; Carcinoma, Hepatocellular: drug therapy (MeSH) ; Carcinoma, Hepatocellular: pathology (MeSH) ; Liver Neoplasms: immunology (MeSH) ; Liver Neoplasms: therapy (MeSH) ; Liver Neoplasms: pathology (MeSH) ; Liver Neoplasms: drug therapy (MeSH) ; Cancer Vaccines: immunology (MeSH) ; Cancer Vaccines: therapeutic use (MeSH) ; Vaccines, Subunit: immunology (MeSH) ; Vaccines, Subunit: therapeutic use (MeSH) ; Immune Checkpoint Inhibitors: therapeutic use (MeSH) ; Antigens, Neoplasm: immunology (MeSH) ; Male (MeSH) ; Remission Induction (MeSH) ; Child (MeSH) ; Neoplasm Metastasis (MeSH) ; Immunotherapy: methods (MeSH) ; Treatment Outcome (MeSH) ; Female (MeSH) ; Protein Subunit Vaccines (MeSH) ; immune checkpoint inhibition ; neoantigen-specific T-cells ; nivolumab ; pediatric hepatocellular carcinoma ; peptide vaccination ; Cancer Vaccines ; Vaccines, Subunit ; Immune Checkpoint Inhibitors ; Antigens, Neoplasm ; Protein Subunit Vaccines

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Contributing Institute(s):

1. DKTK Koordinierungsstelle Tübingen (TU01)

Research Program(s):

1. 899 - ohne Topic (POF4-899) (POF4-899)

Appears in the scientific report 2025

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Database coverage:
; ; ; Article Processing Charges ; Clarivate Analytics Master Journal List ; DOAJ Seal ; Essential Science Indicators ; Fees ; IF >= 5 ; JCR ; SCOPUS ; Science Citation Index Expanded ; Web of Science Core Collection

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