Lenalidomide is an immunomodulatory agent with antiangiogenic and antineoplastic properties, inhibiting fibroblast growth factor (FGF) and vascular endothelial growth factor (VEGF). Recent studies have found that FGF is an important growth factor in hepatocellular cancer (HCC).
Patients with HCC are initially treated with sorafenib, but there is no standard of care for patients who progress after the use of sorafenib. Investigators conducted a study to assess the activity and toxicity of lenalidomide for second-line HCC.
There were 40 patients in the study with advanced HCC who progressed on sorafenib and they were treated with lenalidomide on days 1-21 of a 28-day cycle until disease progression or unacceptable toxicities.
Patients were classified according to the Child-Pugh score: Child-Pugh A (n=19); Child-Pugh B (n= 16); Child-Pugh (n=5). Extrahepatic disease was seen in 17 patients, and grade 4 neutropenia was seen in one patient (2.5%).
The most common nonhematologic toxicity attributable to lenalidomide was grade 3 fatigue (n=3) and rash (n=4). Among the 40 patients, 6 patients (15%) had radiographic partial response and two patients (5%) had not progressed at 36 and 32 months. The median progression-free survival was 3.6 months and the median overall survival was 7.6 months.
Overall, the authors concluded that lenalidomide is an acceptable treatment option for patients with advanced HCC and hepatic dysfunction.
The study results showed a promising and durable activity of lenalidomide in HCC, but more studies need to be conducted to explore its mechanism of action.
Purpose of this study is to assess the activity and toxicity of lenalidomide for patients with advanced hepatocellular cancer (HCC) previously treated with sorafenib. Lenalidomide can be administered to patients with advanced HCC and hepatic dysfunction. Promising, and in a small percentage of patients, durable activity has been demonstrated.