Background and Aims: Data on risk of hepatocellular carcinoma (HCC) in patients with Wilson disease are scarce. We determine HCC risk in a well-defined cohort of Wilson patients. Methods: All patients with a confirmed diagnosis of Wilson disease (Leipzig score≥4) in three Dutch university referral hospitals were included in this retrospective cohort study. End of follow-up was defined as date of diagnosis of HCC, liver transplantation, death, or last available hospital visit. Also, a meta-analysis was performed to determine incidence and mortality rate of HCC in Wilson disease based on all published cohorts. Results: In total, 130 patients with Wilson disease were followed during a median follow-up of 15 years (range 0.1-51.2). At baseline, cirrhosis was present in 74 patients (57% of total: 64% compensated, and 36% decompensated). At end of follow-up, liver disease severity was improved, stable or deteriorated in 20%, 46%, and 24% of all cases (10% unknown), respectively. Two patients developed HCC (one despite excellent decoppering after 50 years follow-up, the other with newly diagnosed Wilson disease). Estimated annual HCC risk for all patients was 0.09% (95% confidence interval [CI]: 0.01-0.28). Subgroup analysis in cirrhotic patients revealed an annual HCC risk of 0.14% (95% CI: 0.02-0.46). The meta-analysis showed an annual HCC risk of 0.04% (95% CI: 0.01-0.10) and HCC mortality rate of 2.6/10000 person-years (95% CI: 0.7-7.0). Conclusions: Even in case of cirrhosis, HCC risk is low in Wilson disease. Our data do not support regular HCC surveillance in Wilson disease.

Cirrhosis, Hepatocellular carcinoma, Wilson disease,
Journal of Gastroenterology and Hepatology
Department of Gastroenterology & Hepatology

van Meer, S, de Man, R.A, van den Berg, A.P, Houwen, R.H.J, Linn, F.H.H, van Oijen, M.G.H, … van Erpecum, K.J. (2015). No increased risk of hepatocellular carcinoma in cirrhosis due to Wilson disease during long-term follow-up. Journal of Gastroenterology and Hepatology, 30(3), 535–539. doi:10.1111/jgh.12716