Impact of neo-adjuvant Sorafenib treatment on liver transplantation in HCC patients – a prospective, randomized, double-blind, phase III trial

BMC Cancer 2015; 15(1): 392.

Aims
To determine whether a combination of Transarterial chemoembolization (TACE) and Sorafenib improves tumour control in hepatocellular carcinoma (HCC) patients on the waiting list for liver transplantation (LT).

Interventions
Patients were treated with either TACE plus Sorafenib (400 mg bid, orally) or TACE plus placebo.

Participants
Fifty HCC patients over 18 years of age who were candidates for liver transplantation.

Outcomes
The primary outcome of the study was the Time-to- Progression (TTP). Secondary outcomes were Tumour Response, Progression-free Survival (PFS), Time-to-LT and Adverse events (AEs).

Follow-up
33 months

CET Conclusions
This study investigates the addition of Sorafenib to transarterial chemoembolization (TACE) as a strategy to prevent progression in patients with hepatocellular carcinoma on the transplant waiting list. The control group received TACE and placebo. The original study power calculation required 208 patients – recruitment was halted after fifty patients due to perceived futility at demonstrating any difference in the primary outcome (time to tumour progression). The trial is well designed, and the results point to a lack of any benefit from Sorafenib. It is difficult to draw any conclusions about secondary outcomes (including safety) due to the lack of power, but no differences are seen in this small cohort.

Jadad score
4

Data analysis
Strict intention-to-treat analysis

Allocation concealment
No

Trial registration
EudraCT 2008 - 002269-29 / ISRCTN24081794

Funding source
Industry funded