A Retrospective Study of Safety and Efficacy of Locoregional Therapies Combined With Anti-VEGF/TKIs and Immune Checkpoint Inhibitors for All Comers of Intention-to-treat Patients With Hepatocellular Carcinoma Beyond UCSF Criteria Before Liver Transplantation

Ningbo Medical Center Lihuili Hospital

Status: Not yet enrolling

Conditions: Liver Transplantation; Hepatocellular Carcinoma

Treatments: Combination Product: Locoregional Therapy (LRT) + Systemic Drug Therapy

Study type: Observational

Funder types: Other

Identifiers

NCT07310836

KY2025PJ483

Details and patient eligibility

About

This is a single-center, retrospective cohort study aiming to evaluate the safety and efficacy of a combined treatment strategy for patients with hepatocellular carcinoma (HCC) who are beyond the UCSF criteria but intended for liver transplantation. The primary objective is to assess the outcomes of these "intention-to-treat" patients who received locoregional therapies (LRT, such as TACE or radiotherapy) in combination with systemic therapy (anti-VEGF/Tyrosine Kinase Inhibitors and Immune Checkpoint Inhibitors) as a "conversion therapy" prior to potential transplantation. This approach is distinct from traditional bridging or down-staging therapies by incorporating more aggressive systemic regimens. The study plans to enroll 300 subjects. The exposure group will include approximately 100 patients beyond UCSF criteria who received the combined conversion therapy between January 2020 and December 2024. A control group of about 200 patients who met the UCSF criteria and were directly listed for transplantation in the same period will be used for comparison. Data will be collected from medical records and the China Liver Transplant Registry (CLTR), with follow-up until December 2025.

Primary outcome measures include:

Objective Response Rate (ORR) of conversion therapy per mRECIST. Rate of successful down-staging to meet UCSF criteria.

1-, 2-, and 3-year overall survival (OS) rates of the intention-to-treat population.

Actual liver transplantation rate after successful conversion. Post-transplant 1-, 2-, and 3-year OS and recurrence-free survival (RFS) rates.

Secondary outcomes involve:

Safety profiles of both conversion therapy and subsequent transplantation. Analysis of factors influencing conversion success and treatment efficacy. Determination of an optimal washout period for Immune Checkpoint Inhibitors prior to transplantation.

The study seeks to provide high-level evidence for optimizing pre-transplant conversion strategies for advanced HCC patients currently outside standard transplant criteria.

Enrollment: 300 estimated patients

Sex: All

Ages: 16+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Age ≥16;
• Clinical diagnosis of HCC;
• CNLC Stage I-IIIA.

Exclusion criteria

• Extrahepatic metastasis;
• Type IV portal vein tumor thrombus.

Trial design

300 participants in 2 patient groups

Beyond UCSF Criteria Cohort

Description:

HCC patients receiving conversion therapy beyond UCSF criteria

Treatment:

Combination Product: Locoregional Therapy (LRT) + Systemic Drug Therapy

Within UCSF Criteria Cohort

Description:

HCC patients within UCSF Criteria Cohort