Neoadjuvant Chemoradiotherapy and Liver Transplantation for Unresectable Hilar Cholangiocarcinoma: The Irish Experience of the Mayo Protocol

BACKGROUND: Pioneered by the Mayo Clinic, multimodal therapy with neoadjuvant chemoradiotherapy and orthotopic liver transplant has emerged as a promising option for unresectable hilar cholangiocarcinoma. This study reports the experience of the Irish National Liver Transplant Programme with the Mayo Protocol. METHODS: All patients diagnosed with unresectable hilar cholangiocarcinoma between 2004 and 2016 who were eligible for the treatment protocol were prospectively studied. RESULTS: Thirty-seven patients commenced chemoradiotherapy. Of those, 11 were excluded due to disease progression and 26 proceeded to liver transplantation. There were 24 males, the median age was 49 and 88% had underlying primary sclerosing cholangitis. R0 and pathologic complete response rates were 96% and 62% respectively. Overall median survival was 53 months and 1-, 3- and 5-year survival were 81%, 69% and 55%. The median survival of patients achieving a pathologic complete response was 83.8 months compared with 20.9 months in the group with residual disease (p=0.036). Six patients (23%) developed disease recurrence. Among the patients who developed metastatic disease during neoadjuvant treatment, median survival was 10.5 months compared to 53 months in patients who proceeded to transplant (p<0.001). CONCLUSION: Neoadjuvant chemoradiotherapy followed by liver transplantation substantially increases the survival of patients with unresectable hilar cholangiocarcinoma. Achieving a pathologic complete response confers a significant survival benefit.