Neoadjuvant therapy and liver transplantation for hilar cholangiocarcinoma: Is pretreatment pathological confirmation of diagnosis necessary?

BACKGROUND: Neoadjuvant chemoradiotherapy followed by operative staging and liver transplantation is an effective treatment for patients with unresectable hilar cholangiocarcinoma (CCA) and CCA arising in the setting of primary sclerosing cholangitis (PSC). Pathologic confirmation of CCA is notoriously difficult, and many patients have been treated based on clinical criteria without pathological confirmation. STUDY DESIGN: We reviewed our experience with the specific aim of determining the need for pathological confirmation of CCA before treatment. RESULTS: Two hundred and fifteen patients received neoadjuvant therapy between 1992 and 2011. One hundred and eighty-two patients underwent operative staging and 38 (21%) had findings that precluded transplantation. Pathological confirmation of CCA before therapy was achieved in 45 of 87 (52%) PSC patients and 22 of 49 (45%) de novo patients who underwent transplantation. Pretreatment pathological confirmation was associated with significantly worse 5-year survival after start of therapy for PSC patients (50% vs 80%; p = 0.001), but not for de novo patients (39% vs 48%; p = 0.27). Pretreatment pathological confirmation was associated with worse 5-year survival after transplantation for PSC patients (66% vs 92%; p = 0.01), but not for de novo patients (63% vs 65%; p = 0.71). The difference in the PSC patients was not due to recurrent cancer. Absence of pretreatment pathological confirmation did not result in less detection of residual CCA in the explanted livers or in less recurrence after transplantation. CONCLUSIONS: Rates of residual CCA in liver explants and recurrences after transplantation are comparable for patients with and without pretreatment pathological confirmation of CCA and attest to the accuracy of clinical diagnostic criteria. Pretreatment pathological confirmation of CCA is desirable but should not be a requirement for treatment.