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

NCT07310836 | Not Yet Recruiting

• 1 other identifier: KY2025PJ483
• Study Type: observational
• Target: 300
• Locations: 0 countries
• Sites: N/A

Brief Summary

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.

Conditions

Hepatocellular Carcinoma; Liver Transplantation

Outcome Measures

Primary Outcomes (3)

• Objective Response Rate (ORR)

  Proportion of patients achieving a Complete Response (CR) or Partial Response (PR) according to mRECIST criteria during the conversion therapy period.

  From the start of conversion therapy until the completion of therapy (assessed up to 24 months).

• Successful Down-staging Rate to UCSF Criteria

  Proportion of patients whose tumor burden is reduced to meet the UCSF transplant criteria after conversion therapy, based on imaging and tumor marker assessment.

  From the start of conversion therapy until the date of meeting UCSF criteria or therapy termination (assessed up to 24 months).

• 1/2/3-year Overall Survival (OS) Rate

  The proportion of patients who received conversion therapy and are still alive at 1, 2, and 3 years after the transplantation surgery, analyzed using the Kaplan-Meier method.

  From the initiation of conversion therapy, assessed at 1, 2, and 3 years.

Study Arms (2)

Beyond UCSF Criteria Cohort

HCC patients receiving conversion therapy beyond UCSF criteria

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

Within UCSF Criteria Cohort

HCC patients within UCSF Criteria Cohort

Interventions

Locoregional Therapy (LRT) + Systemic Drug Therapy COMBINATION_PRODUCT

Treatment regimens, doses, and durations varied based on individual clinical decisions and are collected retrospectively.

Beyond UCSF Criteria Cohort

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

This retrospective, single-center cohort study plans to include 300 patients with HCC. All patients were managed at the study center between January 1, 2020, and December 31, 2024. 1. Beyond UCSF Criteria Cohort (n = 100): Patients with HCC tumors exceeding the UCSF transplant criteria but intended for liver transplantation. All patients in this cohort received multimodal conversion therapy, combining locoregional therapies with systemic therapy. 2. Within UCSF Criteria Cohort (n = 200): A control group of patients whose HCC tumors met the UCSF criteria at the time of listing and were directly registered for liver transplantation without receiving the aforementioned conversion therapy.

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

• Ningbo Medical Center Lihuili Hospital: lead

MeSH Terms

Conditions

Carcinoma, Hepatocellular

Condition Hierarchy (Ancestors)

Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Liver Neoplasms; Digestive System Neoplasms; Neoplasms by Site; Digestive System Diseases; Liver Diseases

Study Officials

• Caide Lu, Professor

  Ningbo Medical Centre Lihuili Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Caide Lu, Professor

CONTACT

+8613957800900; lucaide@nbu.edu.cn

Shuqi Mao, Professor

CONTACT

+8617855848257; mmmaoshuqi@163.com

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER GOV
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Chief Physician/Professor

Study Record Dates

• First Submitted: December 16, 2025
• First Posted: December 30, 2025
• Study Start: December 20, 2025
• Primary Completion: December 31, 2025
• Study Completion: March 31, 2026
• Last Updated: December 30, 2025

Record last verified: 2025-12