Low-dose thalidomide treatment for advanced hepatocellular carcinoma.
OBJECTIVE
To analyze the efficacy of oral thalidomide in the treatment of advanced hepatocellular carcinoma (HCC).


METHODS
Sixty-eight patients with unresectable and nonembolizable 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 World Health Organization criteria, were seen, with a response rate of 6.3% (95% CI 0-12.5). The duration of response was 50+, 24.6, 11.6+ and 8.7+ weeks, respectively. All 4 responders had a dramatic decrease in alpha-fetoprotein (alpha-FP) levels. Another 6 of the 42 patients with elevated alpha-FP levels before treatment had a more than 50% decrease in their alpha-FP levels after thalidomide treatment. Totally 10 patients had an objective response to thalidomide. The median overall survival for all of the 68 patients was 18.7 weeks (95% CI 11.8- 25.6) 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% CI 31.2-93.6 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.


CONCLUSIONS
Low-dose thalidomide is safe and induces unequivocal tumor response in a minority of patients with advanced HCC.
Hsu
Chiun
C
Department of Oncology, National Taiwan University Hospital, Taipei, Taiwan, ROC.
Chen
Chiung-Nien
CN
Chen
Li-Tzong
LT
Wu
Chen-Yao
CY
Yang
Pei-Ming
PM
Lai
Ming-Yang
MY
Lee
Po-Huang
PH
Cheng
Ann-Lii
AL
D000284:Administration, Oral; D000328:Adult; D000368:Aged; D020533:Angiogenesis Inhibitors / Q000009:adverse effects / Q000627:therapeutic use*; D006528:Carcinoma, Hepatocellular / Q000188:drug therapy* / Q000378:metabolism / Q000473:pathology; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D005260:Female; D006801:Humans; D008113:Liver Neoplasms / Q000188:drug therapy* / Q000378:metabolism / Q000473:pathology; D008297:Male; D008875:Middle Aged; D009367:Neoplasm Staging; D015996:Survival Rate; D013792:Thalidomide / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome; D000509:alpha-Fetoproteins / Q000378:metabolism
D020533:Angiogenesis Inhibitors; D000509:alpha-Fetoproteins; D013792:Thalidomide