Zinc Therapy for Wilson Disease in Children in French Pediatric Centers.

Abstract

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.

• Authors: Raoul Santiago, Frédéric Gottrand, Dominique Debray, Laure Bridoux, Alain Lachaux, Alain Morali, Delphine Lapeyre, Thierry Lamireau
• Journal: Journal of pediatric gastroenterology and nutrition (J Pediatr Gastroenterol Nutr) Vol. 61 Issue 6 Pg. 613-8 (Dec 2015) ISSN: 1536-4801 [Electronic] United States
• PMID: 26230903 (Publication Type: Journal Article)

Chemical References

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

Topics

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