Efficacy and safety of thalidomide in patients with hepatocellular carcinoma.
OBJECTIVE
To evaluate which patients with hepatocellular carcinoma (HCC) are most likely to respond to thalidomide treatment.


METHODS
From July 2002 to July 2004, patients with HCC who received thalidomide treatment, were enrolled. We extracted relevant data from the patients' medical records, including history and type of hepatitis, comorbidity, serum alpha-fetoprotein (alpha-FP) level, volumetric changes in tumor, length of survival, and the dose, duration, side effects of thalidomide treatment. The tumor response was evaluated. On the basis of these data, the patients were divided into two groups: those with either partial response or stable disease (PR + SD group) and those with progressive disease (PD group).


RESULTS
Two of 42 (5%) patients had a partial tumor response after treatment with thalidomide, 200 mg/d, and 9 (21%) had stable disease. Patients in the PR + SD group all had cirrhosis. Comparing patients with and without cirrhosis, the former were more likely to respond to thalidomide therapy (PR + SD: 100% vs PD: 64.5%, P = 0.041 < 0.05). Thalidomide was significantly more likely to be effective in tumors smaller than 5 cm (PR + SD: 63.6% vs PD: 25.8%, P = 0.034 < 0.05). Compared with patients with progressive disease (PD), patients in the PR + SD group had a higher total dose of thalidomide (13669.4 +/- 8446.0 mg vs 22022.7 +/- 11461.4 mg, P = 0.023 < 0.05) and a longer survival (181.0 +/- 107.1 d vs 304.4 +/- 167.1 d, P = 0.047 < 0.05). Patients with comorbid disease had a significantly greater incidence of adverse reactions than those without (93.8% vs 60.0%, P = 0.021 < 0.05). The average number of adverse reactions in each person with a comorbid condition was twice as high as in those without other diseases (2.2 +/- 1.3 vs 1.1 +/- 1.2; P = 0.022 < 0.05).


CONCLUSIONS
Thalidomide therapy is most likely to be effective in patients with early stage small HCC, especially in those with other underlying diseases. A low dose (200 mg/d) of thalidomide is recommended to continue the treatment long enough to make it more effective.
Chiou
Hsueh-Erh
HE
Pharmacy Department, Mackay Memorial Hospital, Taipei 10449, Taiwan, China.
Wang
Tsang-En
TE
Wang
Ying-Yue
YY
Liu
Hui-Wen
HW
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D020533:Angiogenesis Inhibitors / Q000009:adverse effects* / Q000627:therapeutic use*; D006528:Carcinoma, Hepatocellular / Q000188:drug therapy*; D004305:Dose-Response Relationship, Drug; D005260:Female; D006801:Humans; D008113:Liver Neoplasms / Q000188:drug therapy*; D008297:Male; D008875:Middle Aged; D009367:Neoplasm Staging; D012189:Retrospective Studies; D013792:Thalidomide / Q000009:adverse effects* / Q000627:therapeutic use*; D016896:Treatment Outcome
D020533:Angiogenesis Inhibitors; D013792:Thalidomide