Sunitinib and the benefits of a negative study.

摘要

The efficacy of sorafenib for the treatment of hepatocellular carcinoma (HCC) has triggered major interest in molecular therapy in this setting and the search for agents or a combination of agents that exceed the benefits of sorafenib. An understanding of a new drug's mechanism of action is essential in determining whether the new agent has a unique therapeutic profile worth further testing, either alone or in combination. Additionally, proper description of patients and treatment effects in terms of response to therapy and of safety is essential. Cirrhosis underlies HCC in most patients and impairment of liver function-leading to life-threatening complications-is of major concern. It is important to note that molecular targeted agents do not induce a large number of objective tumour responses.3 Accordingly, capturing potential efficacy is based on time to tumour progression (TTP) data rather than in a reduction in tumour burden. New tools will be developed using imaging techniques to measure tumour perfusion and metabolic activity. However, until these are available, we must stick to definitions that can be used across studies.