Predictive Biomarkers and Targeted Therapies in Hepatic, Pancreatic, and Biliary Cancers

  • Steven Alexander Mann
  • Romil SaxenaEmail author


Hepatic and pancreatobiliary cancers often present at advanced stages, and currently, there are very few approved therapeutic options. This chapter focuses on the predictive biomarkers for current therapies as well as selected alternative options that are currently being evaluated in Phase III trials for these malignancies. In a Phase III trial for advanced hepatocellular carcinoma (HCC), there was a significant improvement in overall survival for patients with elevated alpha-fetoprotein who received ramucirumab. A Phase II trial of glypican-3 (GPC3) peptide vaccines in HCC demonstrated lower recurrence rates in tumors that were GPC3-positive. Advanced HCC cases with high cellular mesenchymal-epithelial transcription factor (c-MET) expression have significantly improved overall survival on tivantinib. A study of adjuvant chemotherapy for pancreatic cancer reported improved survival in cases with low postoperative CA 19-9. High expression of human equilibrative nucleoside transporter 1 (hENT1) has been shown to predict significantly improved overall survival in both pancreatic cancer and cholangiocarcinoma after gemcitabine therapy. There are currently no predictive biomarker guidelines for hepatic, pancreatic, or biliary tumors. Additional clinical trials are required before consensus statements outlining the utility of biomarkers in the treatment of these cancers can be effectively formulated. Currently, there are many ongoing clinical trials designed to confirm and expand the roles of predictive biomarkers in hepatic and pancreatobiliary cancers. Incorporation of recent advances in molecular analysis and immunotherapy into clinical trials promises new hope for these dismal diagnoses.


Alpha-fetoprotein CA 19-9 Cellular mesenchymal-epithelial transcription factor Cholangiocarcinoma Gemcitabine Glypican-3 Hepatocellular carcinoma Human equilibrative nucleoside transporter 1 Pancreatic ductal adenocarcinoma Predictive biomarkers 


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© Springer Nature Switzerland AG 2019

Authors and Affiliations

  1. 1.Department of Pathology and Laboratory MedicineIndiana University School of MedicineIndianapolisUSA

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