Neoadjuvant therapy for downstaging of locally advanced hilar cholangiocarcinoma: a systematic review




Hilar cholangiocarcinoma is a rare but highly lethal type of cancer. A minority of patients present with resectable disease. Surgery remains the only treatment modality offering a chance of long-term survival. Unresectable patients are typically offered palliative treatment. The aim of this systematic review was to summarize the evidence for neoadjuvant therapy followed by surgical resection in patients presenting with hilar cholangiocarcinoma.


Cochrane databases, Medline, PubMed and EMBASE were systematically searched to identify articles describing neoadjuvant therapy and surgical resection or re-assessment of resectability in patients with hilar cholangiocarcinoma. Included were all articles with original research. Study selection and data extraction were performed separately by two reviewers using a standardized protocol.


From 732 articles 8 full text articles and 2 abstracts met the inclusion criteria. The 2 abstracts and 1 full text article were case reports, 3 articles were retrospective and 4 were prospective studies (2 phase I and 2 phase II studies). Photodynamic therapy, chemotherapy and radiation therapy were used in various indications in populations that included patients with hilar cholangiocarcinoma, some of which were primarily unresectable. Overall quality of articles was limited.


Current evidence suggests that neoadjuvant therapy in patients with unresectable hilar cholangiocarcinoma can be performed safely and in a selected group of patients can lead to subsequent surgical R0 resection. Surgical resection of downstaged patients should be assessed in properly designed phase II studies.