Neoadjuvant Multiagent Systemic Therapy Approach to Liver Transplantation for Perihilar Cholangiocarcinoma

Area of Science:

• Hepatobiliary Surgery
• Surgical Oncology
• Gastroenterology

Background:

• Perihilar cholangiocarcinoma (phCCA) demonstrates favorable outcomes after liver transplantation (LT).
• Neoadjuvant radiation-based locoregional therapy is the current standard of care for phCCA.
• The efficacy of neoadjuvant gemcitabine and cisplatin (gem/cis) systemic therapy (ST) prior to LT remains unstudied.

Purpose of the Study:

• To compare the efficacy of neoadjuvant gem/cis ST alone versus radiation-based approaches in patients with phCCA undergoing LT.
• To evaluate outcomes including posttransplant overall survival (OS), recurrence-free survival (RFS), waitlist time, and pathologic tumor response.

Main Methods:

• Retrospective review of 27 phCCA patients undergoing LT between January 2008 and February 2023.
• Patients were categorized based on neoadjuvant therapy: gem/cis ST alone or ST with radiotherapy (RT).
• Outcomes analyzed included OS, RFS, waitlist duration, and pathological tumor characteristics.

Main Results:

• Of 26 eligible patients, 12 received gem/cis ST and 14 received RT (6 with concurrent gem/cis ST).
• Median waitlist time was 199 days and did not differ between groups.
• No significant differences were observed in OS, RFS, or pathological tumor response between the gem/cis ST and RT groups.

Conclusions:

• Neoadjuvant gem/cis ST and radiation-based therapies yield similar post-LT outcomes for phCCA.
• Patients with phCCA not receiving RT may still be candidates for LT under specific institutional protocols.
• These findings support exploring non-radiation neoadjuvant therapies for phCCA prior to liver transplantation.