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. 2020 Aug 12;9(5):549–562. doi: 10.1159/000508485

Table 1.

Efficacy and toxicity of FDA-approved agents for advanced hepatocellular carcinoma included in the cost effectiveness analysis

Agent Line of Therapy Efficacy data
Toxicity data
OS probability
PFS probability
common AE probability
discontinuation probability for toxicity
4 mo 6 mo 12 mo 4 mo 6 mo 12 mo HFS fatigue diarrhea nausea emesis
Placebo [3] 1 0.75 0.62 0.33 0.42 n.e. n.e. 0.03 0.16 0.11 0.08 0.03 0.37
Sorafenib [3] 1 0.8 0.72 0.44 0.62 n.e. n.e. 0.21 0.22 0.39 0.11 0.05 0.38
Sorafenib [12] 1 0.9 0.75 0.52 0.45 0.35 0.18 0.52 0.25 0.46 0.14 0.08 0.07
Lenvatinib [12] 1 0.9 0.82 0.58 0.65 0.55 0.32 0.27 0.3 0.39 0.2 0.16 0.09
Placebo [11] 2 0.7 0.6 0.3 0.2 0.1 0.05 0.08 0.32 0.15 0.13 0.07 0.19
Regorafenib [11] 2 0.8 0.7 0.5 0.5 0.3 0.15 0.53 0.4 0.41 0.17 0.13 0.25
Placebo [13] 2/3 0.8 0.6 0.35 0.2 0.1 0.05 0.05 0.3 0.19 0.18 0.12 0.03
Cabozantinib [13] 2/3 0.8 0.75 0.5 0.55 0.4 0.2 0.46 0.45 0.54 0.31 0.26 0.16
Nivolumab [15] 2 n.e. 0.83 n.e. n.e. 0.37 n.e. 0.00 0.08 0.1 0.03 0.00 0.03
Pembrolizumab [14] 2 0.9 0.8 0.54 0.6 0.45 0.28 0.00 0.21 0.11 0.08 0.00 0.05

AE, adverse events; FDA, Food and Drug Administration; HFS, Hand-foot syndrome; mo, months; n.e., not evaluable; OS, overall survival; PFS, progression-free survival.