Cholangiocarcinomas are epithelial cell tumors that originate within the biliary tree and comprise 3 subtypes: intrahepatic, perihilar, and distal.¹ Intrahepatic cholangiocarcinoma (ICC) accounts for approximately 20% of all cholangiocarcinomas diagnosed in the United States.^1,2 The incidence and mortality associated with ICC have been increasing over the past several decades related to an increase in known risk factors, such as nonalcoholic steatohepatitis, hepatitis C, and cirrhosis.³ Intrahepatic cholangiocarcinoma is an aggressive cancer often diagnosed in advanced stages owing to a lack of adequate early detection, its insidious clinical character, challenging anatomic access, and limitations of pathologic and cytologic diagnostic tools. Because of these factors, only approximately 35% of patients diagnosed with cholangiocarcinoma will have tumors amenable to curative intent resection at time of diagnosis.⁴ Prognosis is especially poor for the majority of patients diagnosed with unresectable biliary tract cancers, with median overall survival limited to less than 1 year.⁵ For most cases, surgical approaches are generally not indicated and recurrence rates remain high even in the minority of patients who respond to treatment and are able to undergo surgical resection. Approaches to improve survival outcomes for patients with unresectable ICC remain a grave unmet need.

胆管癌是起源于胆管树的上皮细胞肿瘤，包括 3 种亚型：肝内、肝门周围和远端。¹肝内胆管癌 (ICC) 约占美国诊断出的所有胆管癌的 20%。^{1,2与 ICC 相关的}发病率和死亡率在过去几十年中一直在增加，这与已知风险因素的增加有关，例如非酒精性脂肪性肝炎、丙型肝炎和肝硬化。³肝内胆管癌是一种侵袭性癌症，由于缺乏足够的早期检测、其隐匿的临床特征、具有挑战性的解剖通路以及病理学和细胞学诊断工具的局限性，通常在晚期诊断。由于这些因素，只有大约 35% 的被诊断患有胆管癌的患者在诊断时会出现适合根治性切除的肿瘤。⁴对于大多数被诊断患有不可切除胆道癌的患者来说，预后尤其差，中位总生存期限制在 1 年以下。⁵在大多数情况下，通常不需要手术方法，即使在少数对治疗有反应并能够接受手术切除的患者中，复发率仍然很高。改善不可切除 ICC 患者生存结果的方法仍然是一个严重的未满足需求。