Clinical Trials and Outcome Studies

Perihilar cholangiocarcinoma (PHC) is a tumor of the bile duct extending above the junction of the cystic duct up to and including the second biliary branches of the right and left hepatic ducts. Highly aggressive in its nature, PHC evokes a 5y-survival of 20-45% if disease is resectable 30. Although resection remains the standard of care, many patients present with unresectable disease due to vascular encasement by the tumor, extension along the intra-hepatic bile ducts, or due to the underlying liver disease. In theory, replacement of the whole liver by transplantation could solve both issues, but has been recognized to also result in a poor outcome if done without additional treatment. This poor outcome is related to the high rates of tumor recurrence observed under post-transplant immunosuppressive therapy. The Mayo Clinic has developed a new protocol using neoadjuvant chemoradiation therapy followed by liver transplantation, leading to a 65% recurrence-free 5-year-survival31, 32. While this represents an excellent result, organ shortage and the associated waiting time may still impact negatively on this treatment approach in the absence of living donation.
On behalf of the European Hepato-Pancreato-Biliary Association, we recently developed - along with an international panel - a new staging system to assist decision in selecting patients for resection or transplantation33. Consequently, a new PHC International Registry ( was set up to prospectively record any data associated with the management of PHC34, facilitating the planning and interpretation of future trials.


Aim: To compare in select PHC patients the Mayo protocol of neoadjuvant chemoradiation plus liver transplantation with the ALPPS procedure in patients with unresectable PHC.

Study design for the evaluation of ALPPS and LT for PHC