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Both in the United States and worldwide, the incidence of hepatocellular carcinoma (HCC) is on the rise, and, despite extensive research, sorafenib (Nexavar) remains the only FDA-approved systemic therapy available for advanced tumors.

The FDA has approved defibrotide sodium (Defitelio) as a treatment for severe hepatic veno-occlusive disease (VOD) with associated kidney or lung abnormalities following hematopoietic stem cell transplantation (HSCT), based on data from the collection of 3 studies.

Ghassan Abou-Alfa, MD, a medical oncologist at Memorial Sloan Kettering Cancer Center, discusses the significance of a recent trial and how the findings can impact the treatment paradigm of HCC.

Unresectable Hepatocellular carcinoma with Amit Singal, MD and Richard Finn, MD









Ghassan Abou-Alfa says serafnib is currently the only first-line treatment approved for HCC based on the SHARP trial, where the treatment showed improved survival in patients from 7.9 months to 10.7 months.

Unresectable Hepatocellular carcinoma with Amit Singal, MD and Richard Finn, MD







The FDA has granted the A3AR agonist CF102 a fast track designation as a second-line treatment for patients with hepatocellular carcinoma. The drug is also planned to be tested in patients with rheumatoid arthritis and psoriasis.

The presence of circulating tumor DNA in serum could predict microscopic vascular invasion of the portal vein and extrahepatic metastasis following surgery for patients with hepatocellular carcinoma.

Treatment with tivantinib has proven highly effective in patients with hepatocellular carcinoma and with high MET expression, suggesting a prognostic and predictive biomarker of response.

No established regimens exist for the treatment of patients with advanced hepatocellular carcinoma after failure of first-line treatment with the multikinase inhibitor sorafenib. Outcomes in the setting of sorafenib resistance or intolerance are poor, with a median expected overall survival for the placebo arms of second-line trials in the range of 7 to 8 months.

Hepatocellular carcinoma has traditionally been an aggressive malignancy, with diagnosis taking place at later stages of disease and a poor prognosis in most patients. However, recent advances in molecular diagnostics and treatment show promise in changing the HCC clinical landscape.


































