No increased risk of hepatocellular carcinoma in cirrhosis due to Wilson disease during long-term follow-up

J Gastroenterol Hepatol. 2015 Mar;30(3):535-9. doi: 10.1111/jgh.12716.


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 2.6/10 000 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.

Keywords: Wilson disease; cirrhosis; hepatocellular carcinoma.

Publication types

  • Meta-Analysis

MeSH terms

  • Adolescent
  • Adult
  • Carcinoma, Hepatocellular / epidemiology
  • Carcinoma, Hepatocellular / etiology*
  • Child
  • Child, Preschool
  • Cohort Studies
  • Female
  • Follow-Up Studies
  • Hepatolenticular Degeneration / complications*
  • Humans
  • Infant
  • Liver Cirrhosis / etiology*
  • Liver Neoplasms / epidemiology
  • Liver Neoplasms / etiology*
  • Male
  • Retrospective Studies
  • Risk
  • Risk Assessment*
  • Severity of Illness Index
  • Time Factors
  • Young Adult