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Systemic therapy for advanced cholangiocarcinoma

Author
Keith E Stuart, MD
Section Editor
Richard M Goldberg, MD
Deputy Editor
Diane MF Savarese, MD

INTRODUCTION

Cholangiocarcinomas are rare malignancies arising from the epithelial cells of the intrahepatic and extrahepatic bile ducts. Systemic therapy for advanced cholangiocarcinoma will be reviewed here. Surgical and other local treatments for cholangiocarcinoma and ampullary cancer; epidemiology, pathology, classification, clinical presentation, and diagnosis of cholangiocarcinomas and ampullary cancers; and systemic therapy for gallbladder cancer are all discussed elsewhere. (See "Treatment of localized cholangiocarcinoma: Adjuvant and neoadjuvant therapy and prognosis" and "Treatment options for locally advanced cholangiocarcinoma" and "Clinical manifestations and diagnosis of cholangiocarcinoma" and "Epidemiology, pathogenesis, and classification of cholangiocarcinoma" and "Ampullary carcinoma: Epidemiology, clinical manifestations, diagnosis and staging" and "Ampullary carcinoma: Treatment and prognosis" and "Gallbladder cancer: Epidemiology, risk factors, clinical features, and diagnosis".)

GENERAL PRINCIPLES OF CHEMOTHERAPY

Systemic chemotherapy is increasingly being applied in cases of advanced cholangiocarcinoma. A benefit for chemotherapy over best supportive care alone was suggested in a trial that randomly assigned 90 patients with advanced pancreatic or biliary cancer (37 with bile duct cancer) to fluorouracil (FU)-based systemic chemotherapy or best supportive care alone (median survival 6 versus 2.5 months, respectively) [1].

The literature regarding treatment results with specific regimens is limited because most series are small, and many reports consist of a mix of bile duct cancers, gallbladder cancer, ampullary cancer, and either pancreatic or hepatocellular cancers. Although they arise in similar locations, these cancers all have a unique natural history and response to chemotherapy. In general, no single drug or combination has consistently increased median survival beyond the expected six to eight months.

Prognostic factors — Patients with advanced biliary tract cancer are a heterogeneous group, comprising both locally advanced and metastatic disease, as well as a variety of primary disease sites (intrahepatic bile ducts, extrahepatic bile ducts, gallbladder, and ampulla). This heterogeneity complicates assessment of treatment efficacy.

The prognostic influence of these and other factors in patients with advanced biliary tract cancer can be illustrated by a Korean series of 213 patients with advanced biliary tract cancer who were either enrolled in one of two prospective phase II studies or included in a retrospective cohort study of patients receiving first-line chemotherapy [2]. In multivariate analysis, metastatic disease, intrahepatic primary site, the presence of liver metastases, poor Eastern Cooperative Oncology Group (ECOG) performance status (table 1), and elevated level of serum alkaline phosphatase were significant predictors of overall survival. Using these five variables, the authors developed a prognostic index to stratify patients into low-risk, intermediate-risk, and high-risk groups with different median (11.5, 7.3, and 3.6 months, respectively) and one-year survivals (48, 26, and 4 percent, respectively).

                             

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Literature review current through: Nov 2016. | This topic last updated: Fri May 06 00:00:00 GMT 2016.
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