Few randomized controlled trials (RCTs) with large numbers of patients have been conducted to date in patients with
biliary tract cancer, and standard
chemotherapy has not been established yet. In this article we review previous studies and clinical trials regarding
chemotherapy for unresectable
biliary tract cancer, and we present guidelines for the appropriate use of
chemotherapy in patients with
biliary tract cancer. According to an RCT comparing
chemotherapy and best supportive care for these patients, survival was significantly longer and quality of life was significantly better in the
chemotherapy group than in the control group. Thus,
chemotherapy for patients with
biliary tract cancer seems to be a significant treatment of choice. However,
chemotherapy for patients with
biliary tract cancer should be indicated for those with unresectable, locally advanced disease or distant
metastasis, or for those with recurrence after resection. That is why making the diagnosis of unresectable disease should be done with greatest care. As a rule, pathological diagnosis, including cytology or histopathological diagnosis, is preferable.
Chemotherapy is recommended in patients with a good general condition, because in patients with general deterioration, such as those with a performance status of 2 or 3 or those with insufficient biliary
decompression, the benefit of
chemotherapy is limited. As
chemotherapy for unresectable
biliary tract cancer, the use of
gemcitabine or
tegafur/gimeracil/oteracil potassium is recommended. As postoperative
adjuvant chemotherapy, no effective adjuvant
therapy has been established at the present time. It is recommended that further clinical trials, especially large multi-institutional RCTs (phase III studies) using novel agents such as
gemcitabine should be performed as soon as possible in order to establish a standard treatment.