Background: In 2007, a multicenter protocol was developed in Catalonia, Spain, combining neoadjuvant chemoradiotherapy and liver transplantation (LT) for those patients with unresectable hilar cholangiocarcinoma (hCCA).
Aim: To analyse the effectiveness of the neoadjuvant chemoradiotherapy and LT for those patients enrolled in the protocol based on intention-to-treat.
Methods: Observational prospective multicenter study which includes patients ≤ 68 years-old diagnosed with unresectable, solitary tumors ≤3cm in radial diameter, without evidence of lymph node metastases. The protocol was based on a strategy of neoadjuvant therapy with high-dose radiation (45Gy in total) plus intravenous fluorouracil (5-FU) given as a daily bolus for the first 3 days of radiation follow by oral capecitabine until transplantation.  The patient was included in waiting list for LT after operative staging if no evidence of disseminated disease was found.
Results: Between 2007 and 2018, 13 patients were enrolled in the transplant protocol. Three patients dropped out before LT: two cases due to disease progression and one death because of recurrent cholangitis. Two cases were excluded at the moment of LT based on previously undetected tumor progression. Finally, 61% (8/13) of the patients were transplanted. The average time spent on the waiting list was 122 days (range 5-192). Intent-to-treat survival was 62% and 39% at one and 5 years. Post-transplantation overall survival was 75% and 62% and 42% recurrence rate at 5 years.
Conclusion: The effectiveness of the neoadjuvant chemoradiotherapy and LT protocol was 61% in our series with a high rate of post-transplantation recurrence-free survival after 5 years and should be considered as an alternative to resection for patients with localized node-negative hCCA.