Wilson disease in children: serum aminotransferases and urinary copper on triethylene tetramine dihydrochloride (trientine) treatment.
OBJECTIVE
To evaluate the efficacy of and adherence to trientine and/or zinc therapy in children with Wilson disease (WD).


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.
Arnon
Ronen
R
Department of Pediatrics, Mount Sinai Medical Center, Mount Sinai School of Medicine, New York, New York, USA.
Calderon
Judith Flores
JF
Schilsky
Michael
M
Emre
Sukru
S
Shneider
Benjamin L
BL
D000465:Algorithms; D002614:Chelating Agents / Q000627:therapeutic use*; D002648:Child; D003300:Copper / Q000652:urine*; D006527:Hepatolenticular Degeneration / Q000097:blood / Q000188:drug therapy* / Q000652:urine; D006801:Humans; D010349:Patient Compliance; D012189:Retrospective Studies; D000637:Transaminases / Q000097:blood*; D016896:Treatment Outcome; D014266:Trientine / Q000627:therapeutic use; D015032:Zinc / Q000627:therapeutic use
D002614:Chelating Agents; D003300:Copper; D000637:Transaminases; D015032:Zinc; D014266:Trientine