2021 Year in Review: Cholangiocarcinoma

Cholangiocarcinoma (CCA) is an aggressive cancer of the bile duct that forms inside the liver (ie, intrahepatic) or outside the liver (ie, extrahepatic, including perihilar and distal tumors). Individuals with colitis or certain liver diseases may have an increased risk for CCA. Although the precise incidence of CCA is unknown, an estimated 8000 new cases of CCA are diagnosed annually in the United States. It is likely, however, that this number is higher, because CCA is difficult to diagnose and may be misclassified at times.

Real-world retrospective evidence from Korean patients with advanced biliary tract cancer indicates that efficacy and safety with nab-paclitaxel plus gemcitabine/cisplatin combination were consistent with those previously reported in clinical trials.

Findings from a retrospective analysis support the use of both tissue and liquid biopsy biomarker testing to guide therapy selection in patients with advanced CCA, particularly when tissue may not be readily available.

Results of a pilot study demonstrate the feasibility of ctDNA testing and the prognostic value of ctDNA following potentially curative resection in patients with pancreatic/biliary tract malignancies.

Results of the secondary analysis of the SWOG S0809 trial indicate that nodal status did not impact survival outcomes achieved with adjuvant chemoradiation postresection in patients with extrahepatic CCA or gallbladder cancer.

The phase 2 NIFE study compared a liposomal irinotecan plus fluorouracil and leucovorin regimen with standard-of-care gemcitabine/cisplatin in patients with advanced biliary tract cancer.

Results of the NIFTY trial demonstrated that liposomal irinotecan plus 5-fluorouracil and leucovorin significantly improved efficacy outcomes compared with 5-fluorouracil plus leucovorin alone in patients with biliary tract cancer who progressed on prior gemcitabine/cisplatin, with a manageable adverse event profile.

Results of a phase 2 study indicate that toripalimab and lenvatinib in combination with GemOx chemotherapy provide antitumor activity and reasonable tolerability in patients with advanced intrahepatic CCA.

Results of an ongoing, phase 2a, multiple-basket study (MyPathway) showed that dual HER2-targeted therapy with pertuzumab plus trastuzumab was well-tolerated in patients with previously treated HER2-positive metastatic biliary tract cancer.