Investigational New Drugs

, Volume 25, Issue 1, pp 77–84 | Cite as

Arsenic trioxide in patients with hepatocellular carcinoma: a phase II trial

  • Chia-Chi Lin
  • Chiun Hsu
  • Chih-Hung Hsu
  • Wei-Ling Hsu
  • Ann-Lii Cheng
  • Chih-Hsin YangEmail author
Phase II Studies


Background: Arsenic trioxide induces growth inhibition and apoptosis in human hepatocellular carcinoma (HCC) cell lines. A phase II trial was conducted to evaluate the efficacy and toxicity of single-agent arsenic trioxide in patients with HCC.

Methods: Inclusion criteria included advanced HCC patients to whom no standard palliative treatment can be offered, good organ function and liver function reserve. Patients received arsenic trioxide 0.16–0.24 mg/kg per day for 5–6 days per week for 3–4 weeks, followed by one-week rest. Tumor response was assessed every 2 cycles. Primary endpoint was the percentage of patients with 6-month disease stabilization.

Results: Twenty-nine patients (median age, 59) with locally advanced or metastatic HCC received a total of 61 cycles (median, 2; range, 1–6). One patient had partial response. Three patients had disease stabilization for at least six months. The 6-month tumor stabilization rate was 14% (95% CI, 1–27). The median overall survival was 4.8 months (95% CI, 1.4–8.2) and one-year survival was 30%.

Conclusion: Single-agent arsenic trioxide using this dose schedule is not active against advanced HCC.


Antineoplastic agents Arsenic Clinical trials Liver neoplasms 


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Copyright information

© Springer Science+Business Media, LLC 2006

Authors and Affiliations

  • Chia-Chi Lin
    • 1
    • 3
  • Chiun Hsu
    • 1
    • 3
  • Chih-Hung Hsu
    • 1
    • 3
  • Wei-Ling Hsu
    • 3
  • Ann-Lii Cheng
    • 1
    • 2
    • 3
  • Chih-Hsin Yang
    • 1
    • 3
    Email author
  1. 1.Department of OncologyNational Taiwan University HospitalTaipeiTaiwan
  2. 2.Department of Internal MedicineNational Taiwan University HospitalTaipeiTaiwan
  3. 3.Cancer Research CenterNational Taiwan University College of MedicineTaipeiTaiwan

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