Abstract
Hepatocellular carcinoma (HCC) and biliary tract cancers (BTC) exhibit a poor prognosis with 5-year overall survival rates around 15%, all stages combined. Most of these primary liver malignancies are metastatic at diagnostic, with only limited therapeutic options, relying mainly on systemic therapies. Treatment modalities are different yet partially overlapping between HCC and BTC. The complex molecular profile of BTC yields to several actionable therapeutic targets, contrary to HCC that remains the field of antiangiogenic drugs in non-molecularly selected patients. Immunotherapy is now validated in the first line in HCC in combination with bevacizumab, while clinical activity of single agent immunotherapy appears limited to a subset of patients in BTC, still poorly characterized, and combinations are currently under investigation. In this review, we provide a critical evaluation and grading of clinical relevance on (i) the main prognostic biomarkers in HCC and BTC, (ii) the main theragnostic biomarkers in both tumors, and lastly (iii) what is recommended in clinical practice.
Highlights
Hepatocellular carcinoma (HCC) is the third leading cause of cancer death worldwide [1], and the most frequent primary liver cancer (65,000 and 42,000 new cases/per year in Europe and the United States, respectively)
We provide an overview of (i) the main prognostic biomarkers in HCC and biliary tract cancers (BTC), (ii) the main theragnostic biomarkers in both tumors, and lastly (iii) what is really useful in clinical practice
microsatellite instability (MSI) BTC tumors are expected to respond well to immune checkpoint inhibitors (ICIs) and pembrolizumab was recently approved by the Food and Drug Administration (FDA) for patients with metastatic and/or unresectable dMMR or MSI solid tumors that progressed after prior therapy regardless of tumor type [165]
Summary
Hepatocellular carcinoma (HCC) is the third leading cause of cancer death worldwide [1], and the most frequent primary liver cancer (65,000 and 42,000 new cases/per year in Europe and the United States, respectively). Regarding BTC, only one third of cases of them are resectable at diagnosis, and 5-year overall survival (OS) regardless the stage is as low as 5–15% [10,23]. Extensive research is ongoing to help in diagnosis and to predict prognosis and response to treatment of patients with HCC and BTC. These two tumors share common biological features. We provide an overview of (i) the main prognostic biomarkers in HCC and BTC, (ii) the main theragnostic biomarkers (predictive and diagnostic companion) in both tumors, and lastly (iii) what is really useful in clinical practice.
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