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

Abstract 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% CI, 1.1–9.9). The median time until definitive global health score deterioration (according to QLQ-C30) was 2.3 months (95% CI, 1.4–3.7) in the octreotide and 4 months (95% CI, 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.