Lay Summary: Thalidomide can get an occasional response in hepatocellular cancer.
Thalidomide has poorly understood mechanisms of action, and it might exert its therapeutic properties through antiangiogenic activity and modulation of cytokines, including tumor necrosis factor-, interferon, interleukins 10 and 12, cyclooxygenase-2, and nuclear factor B. Several studies have examined the efficacy and toxicity of thalidomide in HCC . Hsu and colleagues conducted a study using thalidomide in 68 HCC patients. Of the 63 evaluable patients, one complete and three partial responses were seen, for a response rate of 6.3% (95% CI, 0%–12.5%). Ten patients had a dramatic decrease in their AFP levels. Interestingly, all responders received thalidomide at 300 mg or less per day. In another study, involving 99 patients with advanced HCC, responses were seen in six patients in response to single-agent thalidomide]. Several recent phase II studies from the U.S. have examined the use of thalidomide either as a single agent or in combination with epirubicin or interferon and have shown limited activity in HCC. Fatigue and somnolence were the most common side effects associated with thalidomide administration.
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