Systemic and Targeted Therapy for Biliary Tract Tumors and Primary Liver Tumors

Key points

The challenges of developing an effective systemic therapy for advanced biliary tract and liver cancers

When evaluating systemic therapy for solid tumors, there are several factors that affect the design of clinical trials, selection of study end points, and the agents to be studied. The factors relevant to biliary tract cancer are summarized in this article.

Systemic therapy for biliary tract cancers: state of the clinical science

A large number of clinical trials of chemotherapy using single agents, doublets, and multidrug combinations have been published in recent years. Most trials were conducted to ascertain whether chemotherapy can provide clinical benefit to patients with advanced cancer of the biliary tract, in terms of palliation of tumor-related symptoms or increased survival. Most trials have been small, single-arm studies that commonly evaluate tumor response rate at the primary end point. For trials that

Advancing treatment of biliary tract cancer by incorporating targeted therapies into clinical trials

There remains a significant unmet medical need to develop effective and safe systemic therapy regimens for patients with advanced cholangiocarcinoma. The 2 main areas of unmet clinical need are to extend the benefit of gemcitabine and cisplatin, potentially by adding one or more targeted agents to the combination, and to develop effective regimens for patients who have failed first-line chemotherapy. However, the numerous single-arm clinical trials in advanced biliary tract cancer conducted

Systemic therapy for hepatocellular carcinoma: current management

Hepatocellular carcinoma (HCC) is a heterogeneous malignancy in terms of cause and molecular carcinogenesis. HCCs are clinically chemotherapy-resistant tumors, and this observation is supported by low response rates across a wide variety of chemotherapy agents. Cytotoxic chemotherapy agents have shown no clinical benefit to patients with HCC, and can be toxic in individuals with underlying liver dysfunction. Over the course of the previous decades, numerous clinical trials of a wide variety of

Summary

Tumors of the biliary tract and HCC are important malignancies worldwide; they are complex tumors with heterogeneous carcinogenic mechanisms that are steadily increasing in incidence in the United States and other Western countries. Most patients diagnosed with both biliary tract cancer and HCC have advanced disease, and these patients represent the highest priority for development of effective therapies. Advanced HCC remains a significant unmet medical need for which available research