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

Area of Science:

• Hepatobiliary surgery
• Transplant oncology
• Surgical oncology

Background:

• Liver transplantation (LT) offers a potentially curative option for unresectable perihilar cholangiocarcinoma (pCCA) after neoadjuvant therapy.
• Disease recurrence post-LT remains a significant challenge impacting patient survival.
• The Perihilar Cholangiocarcinoma Risk Estimation of Tumor REcurrence After Transplant (PRETREAT) score was developed to stratify recurrence risk in LT patients.

Purpose of the Study:

• To evaluate the performance and clinical utility of the PRETREAT score in a cohort of patients with pCCA undergoing LT.
• To assess the score's ability to risk-stratify patients based on predicted recurrence and survival outcomes.

Main Methods:

• Retrospective analysis of patients with unresectable pCCA who received neoadjuvant chemoradiotherapy followed by LT.
• Application of the PRETREAT score to categorize patients into low-, medium-, and high-risk groups.
• Survival analysis, including recurrence-free survival (RFS) and overall survival (OS), was performed for each risk group.

Main Results:

• Thirty-two patients were analyzed with a median follow-up of 55 months.
• Overall 5-year RFS and OS were 57.1% and 45.6%, respectively.
• The PRETREAT score showed significant differences in 5-year RFS (low-risk: 87.5%, medium-risk: 17%, high-risk: 0%; P < .001) and OS (low-risk: 70.8%, medium-risk: 14%, high-risk: 0%; P = .011).

Conclusions:

• The PRETREAT score effectively identified low-risk patients with significantly better long-term RFS and OS after LT for pCCA.
• The score's ability to differentiate between medium- and high-risk groups was limited in this cohort.
• Further refinement or alternative risk stratification tools may be needed for precise risk assessment in higher-risk pCCA patients post-LT.