A new study covering Australia and New Zealand has found that liver transplantation can produce strong long-term survival for certain patients with bile duct cancer, especially those with very small tumors inside the liver.
The COMIT-ANZ research, published in Frontiers in Transplantation, analyzed 64 adult patients who received liver transplants despite having cholangiocarcinoma or related cancers.
The study used records from the Australia and New Zealand Liver and Intestinal Transplant Registry, which has tracked transplant activity since 1986.
Overall, the cohort's five-year overall survival rate was 47%, and recurrence-free survival reached 60%.
These figures are below the region's overall five-year liver transplant survival rate of 83% and the 79% reported for hepatocellular carcinoma.
Tumor Size and Location Drive Outcomes
Results varied substantially by tumor location and size. Patients with intrahepatic cholangiocarcinoma smaller than two centimeters achieved five-year overall and recurrence-free survival rates of 80%.
This supports international guidance that recognizes transplantation as an option for carefully selected patients with very early disease.
In contrast, patients with perihilar cholangiocarcinoma had less favorable outcomes, with five-year overall survival of 31% and recurrence-free survival of 40%.
The researchers also found that poorly differentiated tumors were independently associated with a higher risk of death, with a hazard ratio of 4.78 compared with better-differentiated cancers.
Three-quarters of the patients were male, and the median age at transplantation was 55 years.
Tumors were discovered incidentally during examination of the removed liver in 75% of cases, while only 16 patients had a known diagnosis of cholangiocarcinoma before transplantation.
The study also identified 17 mixed hepatocellular-cholangiocarcinomas, all discovered incidentally after surgery. Their five-year overall survival rate was 68%, compared with 51% for pure cholangiocarcinoma.
The researchers noted the challenges of identifying mixed tumors before transplantation.
Separately, a systematic review published in BMC Cancer examined clinical practice guidelines for precision oncology in biliary tract cancers.
Led by Xiang Pan and Ziting Su of the First Affiliated Hospital of Dali University in Yunnan, China, the review assessed 27 guideline families published or updated between January 2016 and June 2026.
The review found that guidelines generally communicated recommendations clearly but often lacked detailed instructions on molecular testing, patient involvement, external review, and updates as evidence changed.
Clarity of presentation received a median AGREE II score of 98.1%, while scope and purpose scored 88.9%.
Next-generation sequencing was supported by 18 of the 27 guideline families.
For biomarker-directed treatment, microsatellite instability-high or mismatch repair-deficient tumors received support from 20 guideline families, followed by FGFR2 alterations in 17 and IDH1 mutations in 16.
HER2-directed therapy was supported by 12 of 26 assessable families.
The review identified inconsistencies between recommendations for molecular testing and recommendations for the treatments that depend on test results.
Its authors emphasized the need for guidelines to connect testing indications, timing, and methods with subsequent treatment decisions or clinical trial options.
International clinical recommendations also identify surgery as the only established curative-intent approach for eligible cholangiocarcinoma patients, although only 20% to 30% of newly diagnosed cases are considered suitable for resection.
The guidance recommends six months of adjuvant capecitabine after surgery and multidisciplinary assessment for treatment planning.
For advanced disease, comprehensive molecular profiling can identify alterations such as IDH1 mutations, FGFR2 fusions, HER2 amplifications, and BRAF mutations that may guide targeted treatment.
First-line systemic therapy recommendations include cisplatin and gemcitabine combined with durvalumab or pembrolizumab, while FOLFOX remains an established second-line option when appropriate.
