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Sirolimus-Based Immunosuppression Therapy in OLT for Patients With HCC Exceeding Milan Criteria

Phase 3
Conditions
Hepatocellular Carcinoma
Interventions
Registration Number
NCT00554125
Lead Sponsor
Fudan University
Brief Summary

The purpose of this study is to evaluate the influence of sirolimus on outcome after OLT for HCC exceeding Milan criteria.

Detailed Description

Hepatocellular carcinoma (HCC) is one of the most prevalent cancers in Asia and Africa. Although the first-line therapy for HCC is liver resection, the concomitant cirrhosis often leaves orthotopic liver transplantation (OLT) rather than liver resection as the only potentially curative option. The risk of recurrence is the major concern in patients transplanted for HCC. It has been demonstrated that utilizing more restrictive selection criteria before submitting cirrhotic patients with HCC to liver transplantation is associated with a better outcome. The Milan criteria (one nodule ≤5 cm or 2-3 nodules all \<3 cm, without macroscopic vascular invasion and extrahepatic spreading)provide a simple means of selecting patients with HCC for transplantation with a low risk (≈10%) for recurrence. However, the benefit of OLT for patients with HCC within the Milan criteria is opposed by a critical organ shortage, which lengthens the waiting time and thus allows tumor progression during the waiting period. Nearly one third of patients who have a transplant for HCC fall outside the Milan criteria on the basis of pathological findings in the explanted liver, and had a higher risk of tumor recurrence.This led to a dramatic decline in overall and disease-free survival, from 71-85% to 40-50%, and from 65-78% to 27-30%, respectively.

Although it can be hypothesized that the pharmacologic immunosuppression required after liver transplantation for HCC may be accelerated tumor recurrence and metastasis, recent reports have suggest that not all immunosuppressive drugs necessarily promote HCC recurrence in transplant recipients. Sirolimus has emerged as a new, potent immunosuppressive agent which unlike other immunosuppressants \[cyclosporine (CsA), tacrolimus (FK506), and azathioprine (AZA)\] has potent antitumor activity in vitro and in vivo. The immunosuppressive and antitumor effects of sirolimus share a common mechanism of action. Sirolimus inhibits the mammalian target of sirolimus (mTOR), which prevents acute graft rejection mediated by interleukin-2 and could block other cytokine signal transduction, thus directly inhibits tumor cell proliferation and angiogenesis. And the most important is that the antitumor activity of SRL has been shown at the same concentrations as maintenance target levels in posttransplant patients.

Thus, it seems reasonable to speculate that sirolimus could simultaneously contribute to inhibition of tumor recurrence and preventing of rejection in OLT for patients with HCC.

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
220
Inclusion Criteria
  • The major organ (liver,heart, lung and kidney) function after OLT was normal.
  • Pathologically proved HCC before randomisation .
  • Tumor exceeding the Milan criteria (one nodule ≤5 cm or 2-3 nodules all <3 cm, without macroscopic vascular invasion and extrahepatic spreading).
  • Signed, written informed consent.
Exclusion Criteria
  • Extrahepatic metastasis, nodal involvement, perioperative deaths (within 30 days after operation), and tumor thrombi in the proximal main trunk of the portal vein and / or vena cava
  • History of cardiac disease.
  • Active clinically serious infection (>grade 2 Nation Cancer Institute NCI-CTCAE version 3.0).
  • Known history of human immunodeficiency virus (HIV) infection.
  • Any condition that is unstable or which could jeopardize the safety of the patient and his / her compliance in the study.
  • Pregnant or breast-feeding patients.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
2, III ,interventionsirolimus-
Primary Outcome Measures
NameTimeMethod
Disease-free survival3-,5-year
Secondary Outcome Measures
NameTimeMethod
overall survival3-,5-year

Trial Locations

Locations (1)

Liver Cancer Institute and Zhongshan Hospital, Fudan University

🇨🇳

Shanghai, China

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