Sept. 03, 2025
Results of a study led by Mayo Clinic experts in Hepatobiliary and Pancreas Surgery, published in Hepatology, offers important insights into the treatment of de novo perihilar cholangiocarcinoma (pCCA), a rare and aggressive cancer of the bile ducts near the liver. The research compared outcomes between liver resection — both with and without vascular reconstruction — and liver transplantation following neoadjuvant chemoradiation.
Personalized pathways to cure
Treatment choices for newly diagnosed pCCA depend on tumor location, size and vascular involvement. While liver transplantation after chemoradiation has shown excellent survival in select patients, not all candidates complete the rigorous pretransplant process. In this study, 41% of patients enrolled in the transplant protocol were unable to proceed to surgery due to disease progression or complications.
"Transplantation offers outstanding outcomes for unresectable cases," says Patrick P. Starlinger, M.D., Ph.D., a hepatobiliary and pancreas surgeon at Mayo Clinic in Rochester, Minnesota, and the study's senior author "But it's not a one-size-fits-all solution. Our data show that liver resection, even with vascular reconstruction, can be a curative option for patients who don't meet transplant criteria."
Surgical expertise expands possibilities
Over three decades of data revealed that patients who underwent liver resection with vascular reconstruction had significantly better survival than those who dropped out of the transplant protocol. While complex, liver resection with vascular reconstruction offers a viable path for patients with tumors involving major blood vessels.
"Historically, vascular involvement was considered a contraindication for surgery," says first author Yawen Dong, M.D., a research fellow at Mayo Clinic. "However, our findings support expanding resectability boundaries in high-volume centers with experienced surgical teams."
Patients who were eligible and underwent standard liver resection without vascular reconstruction had the most favorable recurrence-free survival and overall survival among all matched cohorts. In contrast, transplant patients had excellent outcomes only if they completed the full protocol.
Matching treatment to tumor biology
Treatment selection for pCCA is not solely about surgical feasibility. It's about the tumor's biology, the presence or absence of is underlying liver disease, and the patient's overall condition. The study found that for small tumors (less than 3 cm) without lymph node involvement, liver transplantation remains the preferred option. However, for resectable tumors, especially those with limited vascular encasement, liver resection — with or without vascular reconstruction — can yield comparable outcomes.
"Treatment decisions should be guided by tumor biology, resectability and institutional expertise," says Dr. Starlinger. "Our goal is to help patients live longer, healthier lives by choosing the right path for each individual."
Next steps: Optimizing outcomes through refined criteria
The authors call for refined selection criteria and improved pretransplant monitoring to reduce dropout rates. Researchers also suggest reevaluating transplant eligibility thresholds, such as the 3-cm tumor size cutoff, which may exclude patients who could benefit from liver transplants.
"Our findings reinforce the importance of careful patient selection and multidisciplinary collaboration," says Timucin Taner, M.D., Ph.D., a transplant surgeon at Mayo Clinic. "Liver transplantation offers excellent outcomes for unresectable perihilar cholangiocarcinoma, but it must be reserved for those who truly meet the criteria and can tolerate the protocol."
This study provides a framework for more personalized, multidisciplinary care. It's about expanding options, not just choosing between them.
For more information
Dong Y, et al. Liver resection with and without vascular resection versus transplantation for de novo perihilar cholangiocarcinoma. Hepatology. In press.
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