Selected Patients with Unresectable Perihilar Cholangiocarcinoma (pCCA) Derive Long-Term Benefit from Liver Transplantation

Simple Summary: Cancers affecting the bile duct may occur in different anatomic regions of the bile duct and are called cholangiocarcinomas. A subset of these cancers involve the bile ducts just below the liver in a region named the hepatic hilum and are referred to as perihilar cholangiocarcinomas. These small cancers are often unresectable (cannot be taken apart by surgery), as they include crucial blood vessels and other bile ducts of the liver. Herein, we report on the role of liver transplant in these patients, which can lead to long term disease free survival. Selected patients with unresectable perihilar cholangiocarcinoma (pCCA) derive long-term benefits from liver transplantation. Between 1993-2019, our group at Mayo Clinic performed 237 transplants for pCCA. With this experience, we note that two distinct patient populations comprise this group of pCCA patients: those with underlying primary sclerosing cholangitis (PSC) and those without identifiable risk factors termed sporadic or de novo pCCA. Long-term survival after transplant is better in PSC patients (74% five-year survival) than in those with de novo pCCA (58% five-year survival). Herein, we review the likely clinical factors contributing to the divergence in outcomes for these two patient populations. We also offer our insights on how further advances may improve patient selection and survival, focusing on the de novo pCCA patient population.