Hepatocellular carcinoma (HCC) is a type of primary liver cancer that occurs in people with chronic liver diseases, like cirrhosis. The incidence of liver transplantation for HCC is increasing, but the expanded criteria remain controversial. This study aims to examine the efficacy of liver transplantation in patients with HCC after tumor downstaging.

This open-label, randomized, multicenter, controlled, phase 2b and 3 controlled trial included a total of 45 patients aged 18-65 years with HCC beyond Milan criteria. The patients were randomly assigned in a 1:1 ratio to receive liver transplantation (n=23) or non-transplantation therapies (control group, n=22). The primary outcomes of the study were 5-year tumor event-free survival for phase 2b and overall survival for phase 3.

The 5-year tumor event-free survival was 76.8% in the liver transplant group, compared with 18.3% in the control group. The 5-year overall survival was 77.5% in the liver transplant group and 31.2% in the control group. Hepatitis C virus recurrence (13%) and acute transplant rejection (9%) were the two registered grade 3 & 4 adverse events in the liver transplant group.

The research concluded that liver transplant could improve the rate of tumor event-free and overall survival after effective and sustained downstaging in patients with HCC.

Ref: https://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(20)30224-2/fulltext