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Cholangiocarcinoma and Gallbladder Cancer Studies / Hepatocellular Carcinoma Treatment and Prognosis · Journal article
Journal of Gastrointestinal Cancer · August 15, 2026
Early or partial results. Treat as a signal, not a conclusion.
This case report documents a single patient with advanced, ruptured HCC (BCLC C) who achieved stable disease over four years with intralesional Pexa-Vec and intravenous Nivolumab, enabling successful liver transplantation with 24-month disease-free survival. The report illustrates potential feasibility of immunotherapy as a bridging strategy but provides no evidence of efficacy, safety profile relative to alternatives, or generalizable patient selection criteria.
Case report. Single patient: 54-year-old male with HCV-related cirrhosis, multifocal unresectable HCC (BCLC C) complicated by hemoperitoneum and tumor rupture; percutaneous biopsies confirmed Edmondson–Steiner grade 2 HCC.. Intervention: Intralesional Pexa-Vec (oncolytic immunotherapy) combined with intravenous Nivolumab (PD-1 inhibitor, 240 mg every 14 days). n = 1. Not stated.
Single 54-year-old male with multifocal, unresectable BCLC C HCC complicated by hemoperitoneum achieved stable disease over 4 years of combination therapy Patient received 3 intralesional Pexa-Vec injections and 93 cycles of Nivolumab with only mild, transient adverse events Liver transplantation performed successfully in September 2023; post-transplant follow-up of 24 months showed no HCC recurrence or graft rejection
No comparative safety or efficacy data versus standard downstaging approaches; adverse event profile limited to 'mild, transient' Patient received 3 intralesional Pexa-Vec injections and 93 cycles of Nivolumab with only mild, transient adverse events
This case suggests immunotherapy may stabilize advanced, ruptured HCC and enable transplant eligibility in exceptional circumstances, but the single success cannot guide patient selection, dosing, or timing in clinical practice. Clinicians should not extrapolate this outcome to inform decisions about other patients without prospective evidence.
A single case report of advanced HCC with rupture treated with combination immunotherapy and successfully transplanted; demonstrates feasibility but lacks comparator, control group, and generalizability needed to establish efficacy or change practice.
As stated by the source record.
Quoted from the source exactly as published.
This case suggests immunotherapy may stabilize advanced, ruptured HCC and enable transplant eligibility in exceptional circumstances, but the single success cannot guide patient selection, dosing, or timing in clinical practice. Clinicians should not extrapolate this outcome to inform decisions about other patients without prospective evidence.
Graded across the dimensions that decide whether you should act, each from what the source actually supports. There is no single score, and where a dimension was not assessed it says so.
Abstract Aim To report a case of advanced hepatocellular carcinoma (HCC) complicated by tumor rupture, successfully downstaged with combined oncolytic immunotherapy (Pexa-Vec) and a PD-1 inhibitor (Nivolumab), ultimately enabling liver transplantation (LT). This report highlights the potential role of immune checkpoint inhibitors (ICIs) as bridging therapy in otherwise ineligible patients. Methods A 54-year-old male with HCV-related cirrhosis presented with multifocal, unresectable HCC (BCLC C) complicated by hemoperitoneum. Surgery was not indicated. He was enrolled in a Phase I/IIa trial of intralesional Pexa-Vec and intravenous Nivolumab (240 mg every 14 days). Percutaneous biopsies confirmed Edmondson–Steiner grade 2 HCC. Clinical, laboratory, and imaging follow-up were performed throughout therapy. Pre-transplant screening was performed after 4 years of stable disease. Results The patient received three intralesional Pexa-Vec injections and 93 cycles of Nivolumab over four years, with only mild, transient adverse events. Imaging showed stable disease without extrahepatic spread. Pre-LT evaluation confirmed preserved liver function (Child-Pugh A5, ECOG 0). Immunotherapy was suspended 12 weeks before LT, and LT was performed successfully in September 2023. Post-transplant follow-up (24 months) revealed no recurrence of HCC or graft rejection. HCV was eradicated with direct-acting antivirals. Explant pathology showed complete necrosis in injected nodules and regressive changes in other lesions. Conclusions ICIs alone or in combination with oncolytic immunotherapy may serve as novel downstaging strategies in advanced, ruptured HCC. Successful LT with long-term disease-free survival challenges traditional concerns about peritoneal seeding. Larger studies are required to define optimal patient selection, timing, and safety of pre-transplant ICIs. Lay summary A 54-year-old man with advanced, ruptured liver cancer had an unexpectedly stable course over four years while receiving immunotherapy and an oncolytic virus. He later underwent a liver transplant and remains cancer-free two years later, illustrating the unpredictable nature of some severe liver cancer cases. Clinical trial registration Not applicable.
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