Orthotopic
liver transplantation (OLT) alone for unresectable
cholangiocarcinoma is often associated with early disease relapse and limited survival. Because of these discouraging results, most programs have abandoned OLT for
cholangiocarcinoma. However, a small percentage of patients have achieved prolonged survival after OLT, suggesting that adjuvant approaches could perhaps improve the survival outcome. Based on these concepts, a protocol was developed at the Mayo Clinic using preoperative irradiation and
chemotherapy for patients with
cholangiocarcinoma. We report our initial results with this pilot experience. Patients with unresectable
cholangiocarcinoma above the cystic duct without intrahepatic or extrahepatic
metastases were eligible. Patients initially received external-beam irradiation plus bolus
fluorouracil (5-FU), followed by
brachytherapy with
iridium and concomitant protracted venous infusion of
5-FU.
5-FU was then administered continuously through an ambulatory
infusion pump until OLT. After irradiation, patients underwent an exploratory
laparotomy to exclude metastatic disease. To date, 19 patients have been enrolled onto the study and have been treated with irradiation. Eight patients did not go on to OLT because of the presence of
metastasis at the time of exploratory
laparotomy (n = 6), subsequent development of malignant
ascites (n = 1), or death from intrahepatic biliary
sepsis (n = 1). Eleven patients completed the protocol with successful OLT. Except for 1 patient, all had early-stage disease (stages I and II) in the explanted liver. All patients who underwent OLT are alive, 3 patients are at risk at 12 months or less, and the remaining 8 patients have a median follow-up of 44 months (range, 17 to 83 months; 7 of 9 patients > 36 months). Only 1 patient developed
tumor relapse. OLT in combination with preoperative irradiation and
chemotherapy is associated with prolonged disease-free and overall survival in highly selected patients with early-stage
cholangiocarcinoma.