Histologic evolution and long-term outcome of Wilson's disease: results of a single-center experience

Eur J Gastroenterol Hepatol. 2013 Jan;25(1):111-7. doi: 10.1097/MEG.0b013e328358f7da.

Abstract

Background/aims: Wilson's disease (WD) is a rare inborn disease related to copper storage, leading to liver cirrhosis and neuropsychological deterioration. The aim of this study was to determine the clinical presentation and long-term outcome, and to examine the progression of hepatic histopathology in serial liver biopsies from WD patients.

Materials and methods: We carried out a retrospective analysis of 60 patients with WD treated with zinc and/or penicillamine. Demographic, clinical, and laboratory data were gathered and 40 patients underwent an initial biopsy and at least one repeat biopsy. Patients were divided into two groups: progressors (patients who presented worsening of at least one unit of fibrosis) and nonprogressors (patients who presented stable or improved fibrosis scores).

Results: A total of 33/40 (83%) patients (nonprogressors) showed stable hepatic histology or improvement. Seven of 40 (17%) patients (progressors) showed worsening of fibrosis. There was no significant correlation between the histological findings and clinical parameters or initial presentation.

Conclusion: In our study cohort, liver disease was stable or improving in most of the patients, and development of progressive hepatic symptoms while under treatment was a rare event. The development of new symptoms while under treatment or progression of pre-existing symptoms was more often recorded for neurological than for hepatic symptoms.

MeSH terms

  • Adolescent
  • Adult
  • Biopsy
  • Chelating Agents / therapeutic use
  • Chi-Square Distribution
  • Child
  • Child, Preschool
  • Disease Progression
  • Female
  • Hepatolenticular Degeneration / complications
  • Hepatolenticular Degeneration / drug therapy
  • Hepatolenticular Degeneration / pathology*
  • Humans
  • Italy
  • Liver / drug effects
  • Liver / pathology*
  • Liver Cirrhosis / drug therapy
  • Liver Cirrhosis / etiology
  • Liver Cirrhosis / pathology*
  • Male
  • Middle Aged
  • Penicillamine / therapeutic use
  • Retrospective Studies
  • Time Factors
  • Treatment Outcome
  • Trientine / therapeutic use
  • Young Adult
  • Zinc Sulfate / therapeutic use

Substances

  • Chelating Agents
  • Zinc Sulfate
  • Penicillamine
  • Trientine