Abstract
BackgroundDisease progression of hepatocellular cancer (HCC) in patients eligible for liver transplantation (LTx) occurs in up to 50% of patients, resulting in withdrawal from the LTx waiting list. Transarterial chemoembolization (TACE) is used as bridging therapy with highly variable response rates. The oral multikinase inhibitor sorafenib significantly increases overall survival and time-to-progression in patients with advanced hepatocellular cancer.DesignThe HeiLivCa study is a double-blinded, controlled, prospective, randomized multi-centre phase III trial. Patients in study arm A will be treated with transarterial chemoembolization plus sorafenib 400 mg bid. Patients in study arm B will be treated with transarterial chemoembolization plus placebo. A total of 208 patients with histologically confirmed hepatocellular carcinoma or HCC diagnosed according to EASL criteria will be enrolled. An interim patients' analysis will be performed after 60 events. Evaluation of time-to-progression as primary endpoint (TTP) will be performed at 120 events. Secondary endpoints are number of patients reaching LTx, disease control rates, OS, progression free survival, quality of live, toxicity and safety.DiscussionAs TACE is the most widely used primary treatment of HCC before LTx and sorafenib is the only proven effective systemic treatment for advanced HCC there is a strong rational to combine both treatment modalities. This study is designed to reveal potential superiority of the combined TACE plus sorafenib treatment over TACE alone and explore a new neo-adjuvant treatment concept in HCC before LTx.
Highlights
Disease progression of hepatocellular cancer (HCC) in patients eligible for liver transplantation (LTx) occurs in up to 50% of patients, resulting in withdrawal from the LTx waiting list
Study objectives The primary objective is to evaluate the efficacy of Transarterial chemoembolization (TACE) plus Sorafenib compared to TACE plus placebo in patients with HCC before LTx
Estimates of the rates of patients reaching LTx in both arms, disease control rates, response rates, frequencies of TACE, overall survival, progression free survival and 95% confidence interval will be calculated for each treatment group
Summary
Tumor progress while waiting for LTx is common in HCC patients. Drop-out rates range between 30% to 50% [17]. AASLD: American Association for Study of Liver Diseases; AFP: α-Fetoprotein; ALAT: Alanine Amino Transferase, known as SGPT; ASAT: Aspartate Amino Transferase, known as SGOT; BCLC: Barcelona-clinic liver cancer bid twice a day; BUN: blood urea nitrogen; CBC: complete blood count; CTC: Common Toxicity Criteria; EASLD: European Association for Study of Liver Diseases; Ecrf: electronic case report form; EMEA : European Medicines Agency; FDA: Food and Drug Agency; GGT : Gamma Glutamyl Transpeptidase; HBV: Hepatitis B virus; HCV: Hepatitis C virus; HCC: hepatocellular carcinoma; ICHGCP: International Conference on Harmonization of Technical Requirements for Registration of Pharmaceuticals for Human Use – Good Clinical Practice; ISRCTN: International Standard Randomized Controlled Trial Number; LTx: liver transplantation; NCI: National Cancer Institute; NCT: National Center for Tumor Diseases; PT: Prothrombin Time; PTT: Partial Thromboplastin Time; PT/INR: Prothrombin time; TACE: transarterial chemoembolization; TTP: time-to-progression
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