While there is resonable theoretical basis for the use of Gleevec for HCC, there is littel supporting clicnial data. A recent pahse II study revealed that fifteen patients, median age 58 years, were enrolled and treated with imatinib. Most, or 7, patients had hepatitis B virus as a risk factor for HCC, followed by hepatitis C virus in 3 patients. Metastatic disease (American Joint Committee on Cancer stage IV) was noted in 13 patients and locally advanced (stage III) in the remainder. The median dose-level of imatinib was 500 mg/d. Two patients had stable disease lasting more than 2 months. The remainder progressed within 2 months of initiation of imatinib. No grade 3 or 4 hematologic toxicity was observed. Two patients had grade 3 elevated liver function tests during treatment; otherwise, there was no other grade 3 or 4 nonhematologic toxicity noted.
Although toxicities were tolerable, imatinib as a monotherapy for the treatment of unresectable HCC has little, if any, significant efficacy.
While EGFR analysis may allow selecting patients who will respond better, this remains to be proven.
Lin, Albert Y. MD et al, Phase II Study of Imatinib in Unresectable Hepatocellular Carcinoma. American Journal of Clinical Oncology. 31(1):84-88, February 2008.
S D Ryder Guidelines for the diagnosis and treatment of hepatocellular carcinoma (HCC) in adults Gut 2003;52:iii1