Wilson disease in children: serum aminotransferases and urinary copper on triethylene tetramine dihydrochloride (trientine) treatment.

Objectives: To evaluate the efficacy of and adherence to trientine and/or zinc therapy in children with Wilson disease (WD).

Materials And Methods: We retrospectively reviewed the clinical records of all children with WD in the pediatric liver/liver transplant program at our institution between 1998 and 2006.

Results: A total of 22 children with WD were evaluated and treated. Seven with fulminant disease required liver transplantation and 15 were treated with trientine and/or zinc. Ten of those 15 had follow-up for 12 to 60 months and 6 of the latter 10 were followed for 12 to 18 months. All 10 patients were started on a trientine treatment regimen. Mean alanine aminotransferase (ALT) levels decreased from 183 +/- 103 IU at presentation (n = 10) to 80 +/- 46 IU at 12 months (n = 10) and 66 +/- 40 IU at 18 months (n = 7). Mean 24-hour urinary copper levels increased from 156 microg at presentation to 494 microg at 1 to 2 months, then decreased to 71 microg after 21 to 24 months of treatment. Three of 10 patients had normalized ALT levels and 1 patient with cirrhosis continued with normal ALT levels since presentation. Four of 10 patients were documented to be nonadherent, as manifested by increased ALT levels (99 +/- 31 IU); 1 patient had previously normalized ALT levels. In 3 of 10 patients, ALT level decreased but remained at an abnormal level (93 +/- 53 IU).

Conclusions: Trientine and/or zinc therapy is effective for children with WD. Nonadherence is a common cause of increased aminotransferase levels in patients with WD.