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Published on: May 15, 2019
Efficacy and safety of thalidomide in patients with hepatocellular carcinoma
Hsueh-Erh Chiou1, Tsang-En Wang, Ying-Yue Wang
1Pharmacy Department, Mackay Memorial Hospital, Taipei 10449, Taiwan, China.
Insights
Thalidomide treatment shows effectiveness in early-stage hepatocellular carcinoma (HCC), particularly in patients with cirrhosis or smaller tumors. Continued low-dose thalidomide (200 mg/d) may improve outcomes.
Area of Science:
- Hepatology
- Oncology
- Pharmacology
Background:
- Hepatocellular carcinoma (HCC) is a significant global health concern.
- Identifying predictive factors for treatment response in HCC is crucial for patient management.
- Thalidomide has emerged as a potential therapeutic agent for HCC.
Purpose of the Study:
- To determine patient characteristics associated with a positive response to thalidomide treatment in HCC.
- To identify predictors of thalidomide efficacy in hepatocellular carcinoma patients.
Main Methods:
- Retrospective analysis of 42 HCC patients treated with thalidomide (200 mg/d) from July 2002 to July 2004.
- Data collected included hepatitis history, comorbidities, alpha-fetoprotein levels, tumor volume, survival, and thalidomide dosage and side effects.
- Patients were categorized into partial response/stable disease (PR + SD) and progressive disease (PD) groups.
Main Results:
- Overall, 5% of patients achieved partial response and 21% had stable disease. Patients with cirrhosis (100% vs. 64.5%) and tumors smaller than 5 cm (63.6% vs. 25.8%) showed significantly higher response rates.
- The PR + SD group received higher total thalidomide doses and had longer survival.
- Comorbid conditions were associated with a higher incidence and number of adverse reactions to thalidomide.
Conclusions:
- Thalidomide therapy is most effective in patients with early-stage, small HCC, especially those with comorbidities.
- A low dose of 200 mg/d thalidomide, administered for a sufficient duration, is recommended for optimal efficacy.
- Further research into predictive biomarkers for thalidomide response in HCC is warranted.
Aim:
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).
Conclusion:
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.
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