Hepatocellular Carcinoma, Liver Transplantation
Conditions
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.
Interventions
COMBINATION_PRODUCTLocoregional Therapy (LRT) + Systemic Drug Therapy
Treatment regimens, doses, and durations varied based on individual clinical decisions and are collected retrospectively.
Sponsors
Ningbo Medical Center Lihuili Hospital
Study design
Observational model
CASE_CONTROL
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
16 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Age ≥16; * Clinical diagnosis of HCC; * CNLC Stage I-IIIA.
Exclusion criteria
* Extrahepatic metastasis; * Type IV portal vein tumor thrombus.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Objective Response Rate (ORR) | From the start of conversion therapy until the completion of therapy (assessed up to 24 months). | Proportion of patients achieving a Complete Response (CR) or Partial Response (PR) according to mRECIST criteria during the conversion therapy period. |
| Successful Down-staging Rate to UCSF Criteria | From the start of conversion therapy until the date of meeting UCSF criteria or therapy termination (assessed up to 24 months). | Proportion of patients whose tumor burden is reduced to meet the UCSF transplant criteria after conversion therapy, based on imaging and tumor marker assessment. |
| 1/2/3-year Overall Survival (OS) Rate | From the initiation of conversion therapy, assessed at 1, 2, and 3 years. | 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. |
Contacts
Primary ContactCaide Lu, Professor
Backup ContactShuqi Mao, Professor
Outcome results
None listed