The study’s goal was to look at the efficacy and safety of trientine-dihydrochloride (TD) in paediatric Wilson disease (WD) patients, as well as the influence of different weight-based doses on their clinical and biochemical outcomes. Researchers evaluated the clinical data of 31 children with WD who were under the age of 18 at the time of diagnosis and were undergoing TD treatment. Two dose subgroups were studied to assess the influence of different weight-based dosages. Group 1 received less than 20 mg/kg TD per day, while Group 2 received more than 20 mg kg1 day1. During TD treatment, nonceruloplasmin-bound copper decreased from a mean of 1.53 mol/l at baseline to 0.62 mol/l, and 24h urinary copper excretion decreased to 1.85 mol/day, close to the therapeutic target of 1.6 mol/day. Seven of the 31 patients required TD medication termination, four of which were due to adverse effects. Weight-based dose studies revealed no significant differences in any laboratory parameter between the two cohorts. However, in terms of clinical safety, adverse effects due to TD were detected in just 6.7 percent of children in the group, whereas it was 63.6 percent in group 2.
For children with WD, TD proved to be an effective alternative chelating agent. Weight-based doses more than the recommended 20 mg kg1 day1 may increase the risk of adverse effects in young patients.