Phase II study of sirolimus in treatment-naive patients with advanced hepatocellular carcinoma

Digestive and Liver Diseases, 03/30/2012

The data suggest that first–line sirolimus shows antitumoural efficacy in advanced hepatocellular carcinoma.

Methods

  • In this phase II study, patients with advanced hepatocellular carcinoma and underlying cirrhosis received sirolimus (20mg/week for 1 month then 30mg/week).
  • Tumour response was assessed every 8 weeks.
  • The primary endpoint was the objective tumour response rate according to the Response Evaluation Criteria in Solid Tumours criteria.
  • Secondary endpoints included the objective response according to the modified Response Evaluation Criteria in Solid Tumours criteria, safety, and pharmacokinetic parameters.

Results

  • Twenty-five patients received sirolimus for a median of 20.6 weeks.
  • Two patients had an objective response (8%, 95CI: 0.98–26.03), including one complete response, and 8 patients had stable disease.
  • There were 2 cases of grade 5 toxicity (infections) and 5 cases of grade 3 toxicity.
  • The main grade 1/2 toxicity was mild transient fatigue (76%).
  • Median time to radiological progression and overall survival were 15.3 weeks (range: 8.2–173.9) and 26.4 weeks (range: 8.2–173.9) respectively.
  • Use of the modified Response Evaluation Criteria in Solid Tumours criteria did not identify any further responders.

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