Validating the PRETREAT Score: Risk Stratification Following Liver Transplantation for Perihilar Cholangiocarcinoma

Abstract

Introduction

Liver transplantation (LT) for early-stage unresectable perihilar cholangiocarcinoma (pCCA) following neoadjuvant therapy is potentially curative, with excellent long-term survival. Disease recurrence remains problematic, however, which negatively impacts survival. Recently the Mayo Clinic group proposed the Perihilar Cholangiocarcinoma Risk Estimation of Tumor REcurrence After Transplant (PRETREAT) score to attempt to risk-stratify patients following LT.

Aims

The aim of this study was to evaluate the performance of the PRETREAT score in our patient cohort.

Methods

All patients with unresectable perihilar cholangiocarcinoma who underwent neoadjuvant chemoradiotherapy followed by LT in our institution were identified retrospectively, and outcome data were collected. The PRETREAT score was applied, and patients were grouped into low-, medium-, and high-risk groups. Survival analysis was then performed.

Results

A total of 32 patients were identified with a median follow-up of 55 months. The 5-year recurrence-free survival (RFS) and overall survival (OS) were 57.1% and 45.6%, respectively. The 5-year RFS was as follows: low-risk (87.5%), medium-risk (17%), and high-risk (0%) groups (P < .001). A significant difference was seen in the 5-year OS among the low- (70.8%), medium- (14%), and high-risk (0%) groups (P = .011).

Conclusion

The PRETREAT score effectively stratified low-risk patients in our cohort who experienced superior long-term RFS and OS. It did not, however, discriminate significantly between those patients in the medium-risk and high-risk groups.