Zinc mono-therapy in pre-symptomatic Chinese children with Wilson disease: a single center, retrospective study

Abstract

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.

Figure 1. Parameters of Liver function.

Linear plot of mean values of liver function parameters at each visit, with number of observations, and p value from paired t-test results with the 1st visit. A: Serum ALT and AST levels. B: Serum ALP and GGT levels. C: Serum total and direct bilirubin levels. D: Serum albumin and total bile acid levels.

Figure 2. Parameters of copper/zinc metabolism and complete blood count.

Linear plot of mean values at each visit with number of observations and p value from paired t-test results with the 1st visit. A: Serum copper and zinc Levels. B: Urinary copper levels. C: White/red blood cell counts. D: Hemoglobin level and platelet counts.