A randomised phase II study of pegylated arginine deiminase (ADI-PEG 20) in Asian advanced hepatocellular carcinoma patients

Tsai Sheng Yang, Sheng Nan Lu, Yee Chao, I. Shyan Sheen, Chun Lin Chen, Tsang En Wang, Shinn Cherng Chen, Jing Houng Wang, Li Ying Liao, Alan J. Thomson, Jacqueline Whang-Peng, Pei Jer Chen, Li Tzong Chen

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135 Citations (Scopus)


Background:Human hepatocellular carcinoma (HCC) cells are largely deficient of argininosuccinate synthetase and thus auxotrophic for arginine. This study aims to investigate the efficacy and pharmacodynamics of pegylated arginine deiminase (ADI-PEG 20), a systemic arginine deprivation agent, in Asian HCC patients.Methods:Patients with advanced HCC who were not candidates for local therapy were eligible and randomly assigned to receive weekly intramuscular injections of ADI-PEG 20 at doses of 160 or 320 IU m 2. The primary end point was disease-control rate (DCR).Results:Of the 71 accruals, 43.6% had failed previous systemic treatment. There were no objective responders. The DCR and the median overall survival (OS) of the intent-to-treat population were 31.0% (95% confidence interval (CI): 20.5-43.1) and 7.3 (95% CI: 4.7-9.9) months respectively. Both efficacy parameters were comparable between the two study arms. The median OS of patients with undetectable circulating arginine for more than or equal to and 4 weeks was 10.0 (95% CI: 2.1-17.9) and 5.8 (95% CI: 1.4-10.1) months respectively (P0.251, log-rank test). The major treatment-related adverse events were grades 1-2 local and/or allergic reactions.Conclusions:ADI-PEG 20 is safe and efficacious in stabilising the progression of heavily pretreated advanced HCC in an Asian population, and deserves further exploration.

Original languageEnglish
Pages (from-to)954-960
Number of pages7
JournalBritish Journal of Cancer
Issue number7
Publication statusPublished - Sept 28 2010
Externally publishedYes


  • arginine
  • arginine deiminase
  • argininosuccinate synthetase
  • hepatocellular carcinoma
  • polyethylene glycol

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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