Zinc mono-therapy in pre-symptomatic Chinese children with Wilson disease: a single center, retrospective study
Kuerbanjiang Abuduxikuer1, Jian-She Wang2
1Liver Center, Children's Hospital of Fudan University, Shanghai, China.
Insights
Zinc gluconate effectively treated pre-symptomatic Wilson Disease (WD) in Chinese children, improving liver enzymes and copper excretion. Higher doses showed similar safety and efficacy to standard doses, with mild gastrointestinal side effects.
Area of Science:
- Pediatric Hepatology
- Clinical Pharmacology
- Genetics and Rare Diseases
Background:
- Lack of consensus on zinc therapy for pre-symptomatic Wilson Disease (WD) necessitates further research.
- Zinc is a potential therapeutic agent for WD, aiming to induce metallothionein synthesis and reduce copper absorption.
Purpose of the Study:
- To evaluate the safety and efficacy of zinc gluconate in Chinese children diagnosed with pre-symptomatic Wilson Disease.
- To compare the outcomes of different elemental zinc dosages in this pediatric cohort.
Main Methods:
- Retrospective analysis of 30 pre-symptomatic children with Wilson Disease treated with zinc gluconate at a specialized Chinese pediatric hepatology center.
- Short-term follow-up data collected to assess safety, efficacy, and effects of varying zinc dosages.
Main Results:
- Zinc gluconate significantly reduced ALT, AST, GGT, and direct bilirubin levels within 1-6 months.
- Urinary copper excretion decreased significantly after 6 months, with serum zinc levels increasing, indicating good compliance.
- Higher elemental zinc doses demonstrated comparable efficacy and safety to conventional doses, with only mild, transient gastrointestinal side effects observed in 8 children.
Conclusions:
- Zinc gluconate is an effective treatment for pre-symptomatic Wilson Disease in Chinese children.
- Higher initial doses of elemental zinc appear to be as effective and safe as conventional doses, warranting further investigation.
Background:
There is no official consensus regarding zinc therapy in pre-symptomatic children with Wilson Disease (WD); more data is needed.
Objective:
To investigate the safety and efficacy of zinc gluconate therapy for Chinese children with pre-symptomatic WD.
Methods:
We retrospectively analyzed pre-symptomatic children receiving zinc gluconate in a single Chinese center specialized in pediatric hepatology. Short-term follow-up data on safety and efficacy were presented, and effects of different zinc dosages were compared.
Results:
30 children (21 males) aged 2.7 to 16.8 years were followed for up to 4.4 years; 26 (87%) children had abnormal ALT at baseline. Most patients (73%) received higher than the currently recommended dose of elemental zinc. Zinc gluconate significantly reduced mean ALT (p<0.0001), AST (p<0.0001), GGT (p<0.0001) levels after 1 month, and urinary copper excretion after 6 months (p<0.0054). Mean direct bilirubin levels dropped significantly at 1 month (p = 0.0175), 3 months (p = 0.0010), and 6 months (p = 0.0036). Serum zinc levels gradually increased and reached a significantly higher level after 6 months (p<0.0026), reflecting good compliance with the therapy. Complete blood count parameters did not change throughout the analysis period. 8 children experienced mild and transient gastrointestinal side effects. The higher zinc dose did not affect treatment response and was not associated with different or increased side effects when compared to conventional zinc dose.
Conclusion:
In our cohort, zinc gluconate therapy for Chinese children with pre-symptomatic WD was effective, and higher initial dose of elemental zinc had the same level of efficacy as the conventional dose.
