ABSTRACT
Background and Aims: Primary sclerosing cholangitis (PSC) is a risk factor for cholangiocarcinoma (CCA) and gallbladder carcinoma (GBCa). Surveillance for GBCa is recommended, but the clinical utility of surveillance for other hepatobiliary cancers (HBCa) in PSC, namely CCA and hepatocellular carcinoma (HCC), is unclear. We aimed to determine whether surveillance is associated with better survival after diagnosis of HBCa in patients with PSC. Methods: Medical records of PSC patients seen at Mayo Clinic Rochester from 1995-2015 were reviewed. Patients were included if they had ≥1 year of follow-up and developed HBCa. Patients were categorized according to their surveillance status (abdominal imaging and carbohydrate antigen 19-9 and alpha-fetoprotein). The primary endpoints were HBCa recurrence, HBCa-related death, and all-cause mortality. Overall survival was assessed by the Kaplan-Meier survival method. HBCa-related survival was assessed using the competing risks regression model. Tests of significance were 2-tailed, and a p-value < 0.05 was considered statistically significant. Results: From 1995-2015, a total of 79 of 830 PSC patients were diagnosed with HBCa. Cumulative follow-up was 712 and 283 person-years pre- and post-HBCa diagnosis, respectively. Seventy-eight percent (54/79) developed CCA, 21% (17/79) HCC, 6% (5/79) GBCa, 3% (2/79) both CCA and HCC, and 1% (1/79) both HCC and GBCa. Fifty-one percent (40/79) were under surveillance and 49% (39/79) were not. Patients in the surveillance group had significantly higher 5-year overall survival (68% vs. 20%, respectively; p<0.001) and significantly lower 5-year probability of experiencing an HBCa-related adverse event (32% versus 75%, respectively; p<0.001), compared with the no-surveillance group. Conclusion: This study demonstrates that surveillance for HBCa significantly improves outcomes, including survival, in patients with PSC. This article is protected by copyright. All rights reserved.
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