以低劑量thalidomide治療晚期肝細胞癌

Objective: To analyze the efficacy of oral thalidomide in the treatment of advanced hepatocellular carcinoma (HCC). Methods: Sixty-eight patients with unresectable and non- embolizable HCC were consecutively enrolled in a compassionate treatment program of oral thalidomide. Tumor response and treatment-related toxicity were prospectively followed. Thalidomide was given at a starting dose of 200 mg per day. The dose was gradually escalated in 100-mg steps up to 600 mg per day if no limiting toxicities developed. Results: Sixty-three patients were evaluable for response. One complete and 3 partial responses, defined by WHO criteria, were seen, with a response rate of 6.3 % (95% C.I. 0 to 12.5%). The duration of response was 50+, 24.6, 11.6+ and 8.7+ weeks, respectively. All 4 responders had dramatic decrease of alpha-fetoprotein (a-FP) levels. Another 6 of the 42 patients with elevated pre-treatment a-FP had more than 50% decrease of their a-FP levels. Totally 10 patients had an objective response to thalidomide. The median overall survival for all of the 68 patients was 18 .7 weeks (95% C.I . 11.8 to 25.6 weeks) with a 1-year survival rate of 27. 6%. The median overall survival of the 10 patients with an objective response to thalidomide was 62.4 weeks (95% C.I. 31.2 to 93.6 weeks). All responders responded at a dose equal to or less than 300 mg per day. Toxicities of thalidomide were generally manageable, and only 16, 6, and 0 patients developed grade 2, 3, and 4 toxicities, respectively. Conclusion : low-dose thalidomide is safe and induces unequivocal tumor response in a minority of patients with advanced HCC