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

Обзор

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.

Вмешательства

• Комбинированный продукт Locoregional Therapy (LRT) + Systemic Drug Therapy
  Treatment regimens, doses, and durations varied based on individual clinical decisions and are collected retrospectively.

Первичные конечные точки

• Objective Response Rate (ORR) [Срок оценки: From the start of conversion therapy until the completion of therapy (assessed up to 24 months).]
• Successful Down-staging Rate to UCSF Criteria [Срок оценки: 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 [Срок оценки: From the initiation of conversion therapy, assessed at 1, 2, and 3 years.]

Критерии участия

Критерии включения

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

Критерии исключения

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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Случай-контроль

Центры проведения

Список центров уточняется — проверьте первичный протокол.

Идентификаторы

NCT: NCT07310836 · KY2025PJ483

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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