Eligibility for Liver Transplantation in Patients with Perihilar Cholangiocarcinoma

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ORIGINAL ARTICLE – HEPATOBILIARY TUMORS

Eligibility for Liver Transplantation in Patients with Perihilar Cholangiocarcinoma Jaynee J. A. Vugts, MD1 , Marcia P. Gaspersz, MD1, Eva Roos, MD2, Lotte F. Franken, MD2, Pim B. Olthof, MD, PhD2, Robert J. S. Coelen, MD, PhD2, Jeroen L. A. van Vugt, MD, PhD1, Tim A. Labeur, MD2, Lieke Brouwer, MD, PhD3, Marc G. H. Besselink, MD, PhD2, Jan N. M. IJzermans, MD, PhD1, Sarwa Darwish Murad, MD, PhD4, Thomas M. van Gulik, MD, PhD2, Jeroen de Jonge, MD, PhD1, Wojciech G. Polak, MD, PhD1, Olivier R. C. Busch, MD2, Joris L. Erdmann, MD, PhD2, Bas Groot Koerkamp, MD, PhD1, and Stefan Buettner, PhD1 Department of Surgery, Erasmus MC University Medical Center, Rotterdam, The Netherlands; 2Department of Surgery, Cancer Center Amsterdam, Amsterdam UMC, University of Amsterdam, Amsterdam, The Netherlands; 3Department of Gastroenterology, Maasstad Ziekenhuis, Rotterdam, The Netherlands; 4Department of Gastroenterology and Hepatology, Erasmus MC University Medical Center, Rotterdam, The Netherlands 1

ABSTRACT Background. Liver transplantation (LT) has been performed in a select group of patients presenting with unresectable or primary sclerosing cholangitis (PSC)-associated perihilar cholangiocarcinoma (pCCA) in the Mayo Clinic with a reported 5-year overall survival (OS) of 53% on intention-to-treat analysis. The objective of this study was to estimate eligibility for LT in a cohort of pCCA patients in two tertiary referral centers. Methods. Patients diagnosed with pCCA between 2002 and 2014 were included from two tertiary referral centers in the Netherlands. The selection criteria used by the Mayo Clinic were retrospectively applied to determine the proportion of patients that would have been eligible for LT. Results. A total of 732 consecutive patients with pCCA were identified, of whom 24 (4%) had PSC-associated

Bas Groot Koerkamp and Stefan Buettner have contributed equally to this work. Ó The Author(s) 2020 First Received: 18 March 2020 Accepted: 22 July 2020 B. Groot Koerkamp, MD, PhD e-mail: [email protected] S. Buettner, PhD e-mail: [email protected]

pCCA. Overall, 154 patients had resectable disease on imaging and 335 patients were ineligible for LT because of lymph node or distant metastases. An age limit of 70 years led to the exclusion of 50 patients who would otherwise be eligible for LT. After applying the Mayo Clinic criteria, only 34 patients (5%) were potentially eligible for LT. Median survival from diagnosis for these 34 patients was 13 months (95% CI 3–23). Conclusion. Only 5% of all patients presenting with pCCA were potentially eligible for LT under the Mayo criteria. Without transplantation, a median OS of about 1 year was observed.

Perihilar cholangiocarcinoma (pCCA) is the most common malignancy of the biliary tract and arises from biliary epithelial cells at the liver hilum.1,2 The annual incidence in Western countries is about 1–2 per 100,000.3 The standard curative option for patients with pCCA is a radical surgical resection which is associ