AT A GLANCE

A new study published in the Annals of Surgery documents the validation and application of a risk score for predicting post-transplant recurrence in perihilar cholangiocarcinoma (pCCA).¹

“PCCA is an aggressive malignancy with a poor prognosis,” explain study authors Li et al. “Although neoadjuvant chemoradiation followed by liver transplantation offers a potential cure for patients with unresectable, early-stage de novo or primary sclerosing cholangitis (PSC)-associated pCCA, post-transplant recurrence negatively impacts survival. Currently, no risk stratification tools exist.”

As part of a retrospective study, the authors therefore sought to develop and validate a risk score for predicting post-transplant recurrence in pCCA. A total 399 consecutive patients with unresectable de novo or PSC-associated pCCA who underwent neoadjuvant chemoradiation followed by liver transplantation at the Mayo Clinic (1993–2024) were enrolled and divided into (development (n = 301) and validation (n = 98) groups.

Multivariable Cox regression with LASSO variable selection was used to identify predictors of post-transplant recurrence; ultimately four independent predictors were identified, including macroscopic residual tumor on explant, vascular encasement, lymphovascular invasion, and radial tumor diameter. Next, the authors established the Perihilar Cholangiocarcinoma Risk Estimation of Tumor Recurrence after Transplant (PRETREAT) risk score (0–22 points) by assigning points from model coefficients. Based on the aforementioned four predictors, they explain, the PRETREAT score demonstrated excellent performance in both internal and external validation.

Study participants were subsequently stratified into low-risk (0–7 points), moderate-risk (8–15 points), and high-risk (16–22 points) groups according to their PRETREAT scores, and the authors found that 5-year recurrence-free survival rates significantly differed across these risk groups (89.0%, 38.3%, and 15.4%, respectively).

The authors add that overall survival improved significantly over the last decade only in moderate-risk patients (70.4% vs. 46.9%), without significant improvements in recurrence-free survival.

“The PRETREAT score provides a valuable tool for guiding post-transplant management of pCCA, enabling risk-stratified surveillance, and laying the foundation for future clinical trials,” they conclude.

Reference

1.     Li Z, Taner T, Eaton JE, et al. Development of the Perihilar Cholangiocarcinoma Risk Estimation of Tumor Recurrence After Transplant (PRETREAT) score. Ann Surg. 2025;282(3):503–514.