Sorafenib use while waiting for liver transplant: We still need to wait

Abstract

With its approval in 2007 for advanced hepatocellular carcinoma (HCC), sorafenib became the first systemic agent with proven activity against the disease. To date, two large randomized studies in advanced stage HCC (BCLC C and a subset of BCLC B) have demonstrated its anti-cancer activity and ability to extend survival in patients with Childs A cirrhosis [1,2]. Since then, several studies have been launched to evaluate sorafenib’s potential role in less advanced HCC. Uniquely, sorafenib’s ability to improve survival is not mediated by inducing significant tumor shrinkage, but by its ability to slow progression and prolong the time to tumor progression (TTP). While some data has been presented [3], several ongoing studies including the SPACE (NCT00855218) and STORM (NCT0692770) studies are evaluating sorafenib as an adjuvant (after definitive therapy) to transarterial chemoembolization (TACE) and curative resection or radiofrequency ablation (RFA), respectively. The current study by Vitale and colleagues uses a cost–benefit analysis to determine the potential utility of using sorafenib in the neo-adjuvant setting, prior to liver transplant [4]. The clinical need to ask this question is real, as the authors highlight, drop-out from HCC progression beyond Milan criteria is one of the main reasons why patients awaiting transplant for HCC do not receive an organ. The Milan criteria was initially described by Mazzaferro and colleagues over a decade ago and has served as the benchmark for prioritizing patients with HCC and otherwise lower MELD scores [5]. However, even with priority, there is still a relative shortage of livers available and HCC patients are competing with those with decompensated liver disease for organs. Though there are regional differences for wait times, the optimal management