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Angiopoietin-2/Tie2 inhibition by regorafenib associates with striking response in a patient with aggressive hepatocellular carcinoma.

Despite surveillance, hepatocellular carcinoma (HCC) often presents at such an advanced stage that only systemic therapy with sorafenib is feasible. However, aggressive HCCs bearing the neoangiogenic transcriptomic signature (TS)(1) are scarcely sensitive to sorafenib, which has no activity against the leading gene of the signature Angiopoietin-2 (Ang-2). The recently approved HCC drug regorafenib could instead possess some pharmacologic activity against it (2,3). We report a case of massive aggressive TS-positive HCC had a striking response to regorafenib. This article is protected by copyright. All rights reserved.

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