BioScience Trends. 2022;16(3):189-197. (DOI: 10.5582/bst.2022.01267)

Treatment of biliary tract carcinoma over the last 30 years

Midorikawa Y


Surgical resection could offer the only chance of a long-term cure for biliary tract carcinoma. However, only a small percentage of these patients can undergo surgery based on the progression of the disease. Most patients with biliary tract carcinoma receive palliative chemotherapy. Until 2010, patients with unresectable biliary tract carcinoma received fluorouracil (5-FU), gemcitabine (GEM), and cisplatin (CDDP)-based chemotherapies. The ABC-02 study established GEM with CDDP as the first-line therapy for patients with unresectable biliary tract carcinoma, and phase III studies indicated that several combinations of anti-cancer drugs such as GEM with S-1 benefited patients. In contrast, clinical studies on targeted therapy dosages for biliary tract carcinoma in the 2010s failed to corroborate the advantages of administering cancer treatment with or without other anticancer drugs. Due to the easy access to cancer panels, precision medicines (such as ivosidenib for IDH1 mutations, pemigatinib for FGFR2 fusions, and entrectinib and larotrectinib for NTRK fusions) were recently found to be effective in the treatment of patients with these genetic alterations. Moreover, many clinical studies on immune checkpoint inhibitors for advanced biliary tract carcinoma are currently underway and could provide more effective treatment options in the near future.

KEYWORDS: biliary tract carcinoma, chemotherapy, anti-cancer drug, targeted therapy, precision medicine, immune checkpoint inhibitor

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