(HealthDay News) — Everolimus does not improve overall survival in patients with advanced liver cancer, according to a study published in the issue of the Journal of the American Medical Association.

Andrew X. Zhu, MD, PhD, from Massachusetts General Hospital in Boston, and colleagues conducted a multi-national, phase 3 trial that included 546 adults with Barcelona Clinic Liver Cancer stage B or C hepatocellular carcinoma and Child-Pugh A liver function.

Either the patients’ disease had progressed during or after sorafenib or they were intolerant of sorafenib. Participants were randomly assigned (2:1) to receive everolimus (7.5 mg/day; 362 patients) or matching placebo (184 patients), both continued until disease progression or intolerable toxicity.

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The researchers observed no significant difference in overall survival between treatment groups (hazard ratio [HR], 1.05; 95% confidence interval [CI], 0.86 to 1.27; P = 0.68; median overall survival, 7.6 months with everolimus and 7.3 months with placebo).

With everolimus, the median time to progression was 3.0 months, and the median time to progression was 2.6 months with placebo (HR, 0.93; 95% CI, 0.75 to 1.15). The most common adverse events (grade 3/4) with everolimus were anemia, asthenia, and decreased appetite.

There were no hepatitis C viral flares; however, hepatitis B viral reactivation was seen in 39 patients (29 everolimus and 10 placebo).

“Everolimus did not improve overall survival in patients with advanced hepatocellular carcinoma whose disease progressed during or after receiving sorafenib or who were intolerant of sorafenib,” the researchers wrote.

Several authors disclosed financial ties to pharmaceutical companies, including Novartis, which funded the study and manufactures everolimus.


  1. Zhu AX, Kudo M, Assenat E, et al. Effect of Everolimus on Survival in Advanced Hepatocellular Carcinoma After Failure of Sorafenib: The EVOLVE-1 Randomized Clinical Trial. JAMA. 2014;312(1):57-67.