Treatment of advanced hepatocellular carcinoma with long-acting octreotide: A phase III multicentre, randomised, double blind placebo-controlled study

Background: A previous study reported a significant survival benefit for octreotide compared with no treatment in patients with advanced hepatocellular carcinoma (HCC). This was investigated further in this multicentre study. Patients and methods: Two hundred and seventy two patients with HCC who were ineligible for curative treatments or had relapsed following potentially curative therapies were randomised to receive long-acting octreotide, 30 mg as an intramuscular injection once every 4 weeks for up to 2 years, or placebo. Results: At the time of the final analysis, median overall survival (OS) was 6.53 months (95% confidence interval [CI], 4.8-8.3) for octreotide versus 7.03 months (95% CI, 5.43-8.53) for placebo (p = 0.34). Progression-free survival (p = 0.26) also did not differ significantly between the two treatment groups. No objective responses were achieved in the octreotide group but 33% of patients achieved disease stabilisation for a mean time of 5.5 months (95% Cl, 1.1-9.9). The median time until definitive global health score deterioration (according to QLQ-C30) was 2.3 months (95% Cl, 1.4-3.7) in the octreotide and 4 months (95% Cl, 2.2-5.7) in the placebo group (p = 0.09). There were four objective responses in the placebo group. Octreotide was well tolerated; seven patients reported severe adverse events possibly related to octreotide and there were no cases of haematoma or cholecystitis. Conclusions: in patients with advanced HCC, octreotide has a favourable safety profile but does not improve OS and could have a negative impact on quality of life. (C) 2009 Elsevier Ltd. All rights reserved.