Neoadjuvant Systemic Therapy for Resectable Intrahepatic Cholangiocarcinoma: From Retrospective Studies to Randomized Evidence

Abstract

Simple Summary Intrahepatic cholangiocarcinoma is an aggressive bile duct cancer that arises within the liver and frequently recurs after surgery. Administering anticancer treatment before surgery may treat occult disease earlier and help identify patients most likely to benefit from major liver surgery; however, this approach risks causing treatment-related toxicity or permitting disease progression prior to resection. This review examines the evidence for this approach in patients whose tumors are surgically resectable but exhibit features associated with a high risk of recurrence. Early studies were retrospective and difficult to interpret because they inherently included highly selected patient cohorts. A recent randomized trial demonstrated that a preoperative multidrug regimen prolonged the time to progression, recurrence, or death, although a definitive overall survival benefit has not yet been established. These findings support the selective use of this approach in medically fit patients and highlight the need for extended follow-up, international validation, and refined patient-selection criteria.