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Published on: May 16, 2019
Gastrointestinal side effects in children with Wilson's disease treated with zinc sulphate
Anna Wiernicka1, Wojciech Jańczyk, Maciej Dądalski
1Department of Gastroenterology, Hepatology and Malnutrition, the Children's Memorial Health Institute, 04-730 Warsaw, Poland.
Insights
Zinc sulphate therapy for pediatric Wilson
Area of Science:
- Hepatology and Gastroenterology
- Pediatric Rare Diseases
- Pharmacovigilance
Background:
- Wilson's disease is a rare genetic disorder of copper metabolism.
- Zinc sulphate is a therapeutic option for Wilson's disease, particularly in pediatric cases.
- Understanding treatment-related side effects is crucial for patient management.
Purpose of the Study:
- To evaluate the gastrointestinal side effects of zinc sulphate therapy in Polish pediatric Wilson's disease patients.
- To assess the incidence and nature of adverse events associated with zinc sulphate treatment.
- To inform clinical monitoring protocols for children undergoing zinc sulphate therapy.
Main Methods:
- Retrospective analysis of 53 pediatric patients diagnosed with Wilson's disease.
- Patients treated with zinc sulphate between 1996-2011 at a tertiary pediatric center.
- Data collected on diagnosis, treatment, side effects, and efficacy, including esophagogastroduodenoscopy in symptomatic cases.
Main Results:
- Gastrointestinal side effects were reported in 40% of pediatric patients.
- Common side effects included abdominal pain, leading to esophagogastroduodenoscopy revealing ulcerations/erosions.
- Proton pump inhibitors were partially effective; some patients required a switch to D-penicillamine.
Conclusions:
- Zinc sulphate therapy is associated with significant gastrointestinal adverse events in pediatric Wilson's disease.
- Close monitoring for gastrointestinal symptoms is essential during zinc sulphate treatment.
- Alternative therapies may be necessary for patients experiencing severe side effects.
Aim:
To investigate the side effects of a zinc sulphate therapy in a cohort of Polish pediatric patients with Wilson's disease.
Methods:
We retrospectively analyzed a cohort of 53 pediatric patients with Wilson's disease treated at the Children's Memorial Health Institute in Warsaw, Poland between the years 1996 and 2011 with zinc sulphate. Patients were diagnosed with Wilson's disease according to the scoring system of Ferenci, with 49 cases confirmed by mutation analysis. Data about the dosage scheme of zinc sulphate, side effects and efficacy and toxicity of the treatment were collected and recorded in the patient's medical chart at each visit to the hospital.
Results:
Mean age of diagnosis for the entire cohort was 10 years (range, 2.5-17 years). Duration of treatment with zinc sulfate was 83.3 wk (range, 8-344 wk). Side effects, all of gastrointestinal origin, were observed in 21 patients (40%--9 males and 12 females), irrespective of the duration of therapy. Thirteen out of 21 patients were over the age of 10 years. The most common ATP7B mutation was p.H1069Q. Esophagogastroduodenoscopy, performed in 7 patients (33.3%) suffering from persistent and severe abdominal pain, revealed gastrointestinal ulcerations or erosions with negative Helicobacter pylori tests in all subjects investigated. The above mentioned 7 patients were treated with proton pump inhibitors. Three of those experienced resolution of symptoms, whereas proton-pump inhibitors failed to alleviate symptoms of the remaining four children and conversion of therapy to D-penicillamine was needed.
Conclusion:
Zinc sulphate appears to cause significant gastrointestinal side effects, which children on therapy for Wilson's disease should be closely monitored for.
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