van Meer Suzanne, de Man Robert A, van den Berg Aad P, Houwen Roderick H J, Linn Francisca H H, van Oijen Martijn G H, Siersema Peter D, van Erpecum Karel J
Department of Gastroenterology and Hepatology, University Medical Center, Utrecht, The Netherlands.
J Gastroenterol Hepatol. 2015 Mar;30(3):535-9. doi: 10.1111/jgh.12716.
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.
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.
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).
Even in case of cirrhosis, HCC risk is low in Wilson disease. Our data do not support regular HCC surveillance in Wilson disease.
威尔逊病患者肝细胞癌（HCC）风险的数据稀缺。我们在一个明确界定的威尔逊病患者队列中确定HCC风险。
本回顾性队列研究纳入了荷兰三家大学转诊医院中所有确诊为威尔逊病（莱比锡评分≥4）的患者。随访结束定义为HCC诊断日期、肝移植、死亡或最后一次可获得的医院就诊日期。此外，进行了一项荟萃分析，以根据所有已发表的队列确定威尔逊病中HCC的发病率和死亡率。
总共对130例威尔逊病患者进行了随访，中位随访时间为15年（范围0.1 - 51.2年）。基线时，74例患者存在肝硬化（占总数的57%：64%为代偿期，36%为失代偿期）。随访结束时，所有病例中20%、46%和24%的肝病严重程度有所改善、稳定或恶化（10%情况不明）。两名患者发生了HCC（一名在随访50年后尽管进行了良好的排铜治疗仍发病，另一名为新诊断的威尔逊病患者）。所有患者的估计年度HCC风险为0.09%（95%置信区间[CI]：0.01 - 0.28）。肝硬化患者的亚组分析显示年度HCC风险为0.14%（95% CI：0.
