Background and goals: Several studies have demonstrated that sorafenib is effective in the treatment of unresectable hepatocellular carcinoma (HCC). We performed a systematic review of the efficacy and safety of sorafenib in Child-Pugh A patients with unresectable HCC. The value of sorafenib treatment in different subgroups was examined.
Materials and methods: A search of the literature published up to July 2012 was conducted. Pubmed, Embase, and the Cochrane library were searched and only randomized controlled trials were included.
Results: Five randomized controlled trials consisting of 1462 patients with unresectable HCC were included. Meta-analyses demonstrated that sorafenib improved the control rate of the disease [relative risk, 1.85; 95% confidence interval (CI), 1.55, 2.20; P<0.001], decreased the risk for tumor progression (hazard ratios, 0.61; 95% CI, 0.51, 0.73; P<0.001), and decreased mortality (hazard ratios, 0.71; 95% CI, 0.56, 0.89; P<0.001), relative to placebo. Subgroup analyses indicated that sorafenib-based treatments were effective in unresectable HCC regardless of the etiology, performance status, Barcelona Clinic Liver Cancer-stage, alanine transaminase/asparate transaminase, bilirubin, and α-feto protein level, except in the subgroup of prior local therapy. Sorafenib was associated with a higher risk of adverse effects than placebo. The risk for grade 3-4 hand-foot skin reactions, rash or desquamation, diarrhea, and hypertension was much higher in the sorafenib treatment group. These side effects could often be mitigated with appropriate treatment.
Conclusions: Sorafenib was a moderately effective and safe oral drug for use in Child-Pugh A patients with unresectable HCC. Sorafenib monotherapy is not recommended for treating intermediate-stage HCC. More research is needed on the efficacy of sorafenib treatment in patients with prior local therapy.