Study of Immunotherapy-Based Downstaging Treatment for Liver Cancer Beyond Milan Criteria Before Liver Transplantation

What this Trial Is About

Researchers are evaluating a new treatment approach for patients with hepatocellular carcinoma HCC that extends beyond the standard Milan criteria used for liver transplantation. This phase II study aims to assess the safety and effectiveness of a downstaging protocol that includes immunotherapy, specifically Anti-PD-1 inhibitors, combined with other therapies. This approach is designed to improve outcomes for patients who currently face limited options due to tumor size or spread, particularly considering the shortage of donor organs and risk of tumor recurrence after transplantation. The study involves four phases. First, eligible patients receive downstaging treatment with Anti-PD-1 inhibitors every 3 to 4 weeks intravenously, combined with locoregional or targeted therapies decided by a multidisciplinary team. Patients who meet defined criteria for successful downstaging within 12 treatment cycles move to an observation phase lasting at least 3 months, where additional non-immunotherapy treatments are not allowed. Those maintaining tumor control are then placed on a liver transplant waiting list, followed by a bridging phase where optional non-immunotherapy treatments may be given before undergoing liver transplantation. Post-transplant, patients are monitored unless contraindications arise. Participants will undergo various assessments throughout the study, including tumor response evaluations by imaging criteria, blood tests such as alpha-fetoprotein levels, and survival predictions using established models. Researchers will monitor event-free survival at two years after transplantation as the primary outcome, along with overall survival rates, graft function, and rejection rates. The study also tracks the success of tumor downstaging and safety during all phases, with participants followed up through transplantation and beyond for comprehensive evaluation.

Downstaging Protocol Containing Immunotherapy for HCC Beyond the Milan Criteria Before Liver Transplantation