Liver Transplantation for Unresectable Perihilar Cholangiocarcinoma: Is Neo-Adjuvant Chemo-Radiation Therapy Really Necessary? An International, Multicenter Comparison

Abstract

Introduction: The Mayo Clinic protocol for patients with unresectable perihilar cholangiocarcinoma (pCCA) is based on strict selection and neo-adjuvant chemo-radiation therapy (NAT) followed by liver transplantation (LT). With this protocol, 5-year survival up to 68% has been reported. It remains unclear whether these favorable results are attributable to NAT or strict selection only. Our aim was to compare outcomes after LT for unresectable pCCA after strict selection between patients with or without NAT. Method: International, multicenter, comparison of two cohorts of patients transplanted between 2011-2021, for unresectable pCCA using the Mayo Clinic selection criteria and receiving NAT (NAT+) or not (NAT-). Primary endpoint was overall survival. Secondary outcomes included post-transplant morbidity (including vascular complications) and tumor recurrence evaluated by inverse-probability-weighting and competing risk analysis. Results: A total of 49 patients underwent LT for unresectable pCCA (NAT+ n=27, NAT- n=22). Median age was 55 years. Median follow-up was 25 months. In the NAT+ group, the 1-, 3- and 5-year survival rates were 65%, 51% and 41%, respectively, compared to 91%, 68% and 53% for NAT- patients (p=0.20; Figure). Tumor 5-year recurrence rate was similar between groups: 32% in the NAT+ group versus 53% in the NAT- group (p=0.55). Major vascular complications (Clavien-Dindo grade ≥3A) post-LT were significantly higher in the NAT+ cohort with 11 patients (41%) versus 3 patients (14%) (p=0.04). Conclusions: Survival after LT for pCCA in strictly selected patients did not differ between patients with or without NAT. Post-transplant vascular complications were significantly higher in patients after NAT.