Zinc Therapy for Wilson Disease in Children in French Pediatric Centers

J Pediatr Gastroenterol Nutr. 2015 Dec;61(6):613-8. doi: 10.1097/MPG.0000000000000926.


Background and aims: Zinc therapy is considered a good option in Wilson disease (WD), as a first-line treatment in presymptomatic children and a maintenance therapy after the initial chelator therapy. The aim of the study was to determine the practical use of zinc treatment in French pediatric centers.

Methods: A national survey was conducted in the 6 French centers using zinc acetate to treat WD. Clinical and biological parameters, dosage, and outcome were recorded.

Results: A total of 26 children were reported to be treated with zinc acetate, alone or in association with chelators. Of the 9 children (35%) who received zinc alone as a first-line therapy, 2 were switched to D-penicillamine because of inefficacy and 7 remained on zinc alone, but serum transaminase levels normalized in only 4 of them. Five children (19%) were initially treated with zinc in association with D-penicillamine (n = 4) or Trientine (n = 1) with good efficacy. Among the 12 children (46%) who received zinc as a maintenance therapy after D-penicillamine, no relapse of hepatic cytolysis occurred during a median follow-up of 5.2 years, but 2 of them were switched to Trientine because of zinc-related adverse effects. Epigastric pain was observed in 4 children, and a gastric perforation occurred in 1 child.

Conclusions: The present study demonstrates poor efficacy of zinc as first-line therapy to control liver disease in half presymptomatic children and a high incidence of related gastrointestinal adverse effects in children with WD.

MeSH terms

  • Abdominal Pain / etiology
  • Adolescent
  • Chelating Agents / therapeutic use*
  • Child
  • Child, Preschool
  • Copper / metabolism
  • Female
  • France
  • Health Care Surveys
  • Health Facilities
  • Hepatolenticular Degeneration / blood
  • Hepatolenticular Degeneration / complications
  • Hepatolenticular Degeneration / drug therapy*
  • Hepatolenticular Degeneration / pathology
  • Humans
  • Infant
  • Liver / drug effects*
  • Liver / metabolism
  • Liver / pathology
  • Male
  • Pediatrics
  • Penicillamine / therapeutic use*
  • Retrospective Studies
  • Stomach / drug effects
  • Trace Elements / adverse effects
  • Trace Elements / metabolism
  • Trace Elements / therapeutic use*
  • Transaminases / blood
  • Treatment Outcome
  • Trientine
  • Zinc / adverse effects
  • Zinc / therapeutic use*
  • Zinc Acetate / adverse effects
  • Zinc Acetate / therapeutic use


  • Chelating Agents
  • Trace Elements
  • Copper
  • Transaminases
  • Zinc Acetate
  • Penicillamine
  • Zinc
  • Trientine