Identifying Indications for Neoadjuvant Therapy in Cholangiocarcinoma

The incidence of cholangiocarcinoma (CCA) is rising, and survival rates remain low. Recent randomized, controlled trials (RCTs) demonstrated improved survival with adjuvant chemotherapy and immunotherapy in the metastatic and postoperative settings. Neoadjuvant therapy is increasingly used for other cancers to achieve R0 resection and as an indicator of treatment response. Although there has not been an RCT for neoadjuvant therapy in CCA, there are multiple supportive retrospective studies. Data from the recent phase 2 NEO-GAP (NCT03579771) prospective trial for neoadjuvant chemotherapy in patients with resectable high-risk intrahepatic CCA demonstrated safety and the increased likelihood of R0 resection with a neoadjuvant approach.