Liver and Bile
J Hepatol. 2023;78(3):604–13
Prospective surveillance for cholangiocarcinoma in unselected individuals with primary sclerosing cholangitis
Background and aims: The evidence for hepatobiliary tumor surveillance in patients with primary sclerosing cholangitis (PSC) is scarce. In this study, the authors aimed to prospectively evaluate cholangiocarcinoma (CCA) surveillance with yearly magnetic resonance imaging (MRI) with cholangiopancreatography (MRI/MRCP) in a nationwide cohort.
Methods: In total, 512 patients with PSC from 11 Swedish hospitals were recruited. The study protocol included yearly clinical follow-ups, liver function tests and contrast-enhanced MRI/MRCP and carbohydrate antigen (CA)19-9. Patients with severe/progressive bile duct changes on MRI/MRCP were further investigated with endoscopic retrograde cholangiopancreatography (ERCP). Patients were followed for 5 years or until a diagnosis of CCA, liver transplantation (LT) and/or death. Risk factors associated with CCA were analyzed with Cox regression.
Results: 11 patients (2%) were diagnosed with CCA, and 2 (0.5%) with high-grade bile duct dysplasia. Severe/progressive bile duct changes on MRI/MRCP were detected in 122 patients (24%), of whom 10% had an underlying malignancy. The primary indication for LT (n = 54) was biliary dysplasia in 9 patients (17%) and end-stage liver disease in 45 patients (83%), of whom 3 patients (7%) had unexpected malignancy in the explants. The median survival for patients with CCA was 13 months (3–22 months). Time to diagnosis of high-grade dysplasia and/or hepatobiliary malignancy was significantly associated with severe/progressive bile duct changes on MRI/MRCP (hazard ratio [HR] = 10.50; 95% confidence interval [CI]: 2.49–44.31) and increased levels of CA19-9 (HR = 1.00; 95% CI: 1.00–1.01).
Conclusion: In an unselected cohort of patients with primary sclerosing cholangitis (PSC), yearly CA19-9 and MRI/MRCP surveillance followed by ERCP was ineffective in detecting cancer early enough to support long-term survival. Given the low occurrence of cholangiocarcinoma (CCA), studies on individualized strategies for follow-up and improved diagnostic methods for PSC-related CCA are warranted.