A phase II study of imatinib mesylate (IM) in patients (pts) with unresectable hepatocellular carcinoma (HCC)

4223 Background: HCC is a devastating disease with limited treatment options in advanced stage. Level of platelet-derived growth factor (PDGF), a potent mitogen, correlates with the severity of liver cirrhosis. Over-expression of the PDGF receptor (PDGF-R) has been demonstrated in human HCC tumors and cell lines. IM, a tyrosine kinase inhibitor, inhibits PDGF-R. To assess the efficacy and safety of IM in HCC pts, we performed a phase II trial. Methods: HCC patients with reasonable organ function, unresectable but measurable disease, not candidates for chemoinfusion, age above 18 years, performance status of 0–2, and able to give informed consent were eligible. IM was started at 300 mg/day orally with 100 mg/week dose escalation up to a maximum dose of 800 mg/d. Results: Between February 2003 and November 2004, 13 patients (M: F = 11:2), median age 49 years (range 22 - 76 years) were enrolled. Most (8) of them had HBV as a risk factor for HCC. Others include: HCV (1), alcohol (1), and non-identifiable (3)....