TY - JOUR
T1 - Prospective Pilot Study of a Single Daily Dosage of Trientine for the Treatment of Wilson Disease
AU - Ala, Aftab
AU - Aliu, Ermal
AU - Schilsky, Michael L.
N1 - Publisher Copyright:
© 2015, The Author(s).
PY - 2015/5/1
Y1 - 2015/5/1
N2 - Background: Wilson disease requires lifelong therapy, currently given daily in multiple divided dosages. Aim: To prospectively evaluate once-daily trientine as therapy for Wilson disease. Methods: Study group: eight patients (seven males) aged 22–71 years with stable Wilson disease treated from 4 to 50 years. Patients were monitored for 3 months then for 12 months on a single daily dose of trientine (15 mg/kg). Results: All patients remained clinically well. ALT and AST fluctuated in some, but none required treatment stoppages or side effects. Liver synthetic function was unchanged. Mean 24-h urine copper and zinc excretions at end of treatment were 313.4 ± 191.7 and 2,214 ± 1,346 μg, respectively. Conclusions: Once-daily trientine should be explored further for possible maintenance therapy for WD. Single daily dose may improve adherence to therapy. Larger trials and longer-term follow-up will establish the safety and treatment efficacy of this once-daily treatment regimen for WD (registration: NCT01472874).
AB - Background: Wilson disease requires lifelong therapy, currently given daily in multiple divided dosages. Aim: To prospectively evaluate once-daily trientine as therapy for Wilson disease. Methods: Study group: eight patients (seven males) aged 22–71 years with stable Wilson disease treated from 4 to 50 years. Patients were monitored for 3 months then for 12 months on a single daily dose of trientine (15 mg/kg). Results: All patients remained clinically well. ALT and AST fluctuated in some, but none required treatment stoppages or side effects. Liver synthetic function was unchanged. Mean 24-h urine copper and zinc excretions at end of treatment were 313.4 ± 191.7 and 2,214 ± 1,346 μg, respectively. Conclusions: Once-daily trientine should be explored further for possible maintenance therapy for WD. Single daily dose may improve adherence to therapy. Larger trials and longer-term follow-up will establish the safety and treatment efficacy of this once-daily treatment regimen for WD (registration: NCT01472874).
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U2 - 10.1007/s10620-014-3495-6
DO - 10.1007/s10620-014-3495-6
M3 - Article
C2 - 25605552
AN - SCOPUS:84940001519
SN - 0163-2116
VL - 60
SP - 1433
EP - 1439
JO - Digestive Diseases and Sciences
JF - Digestive Diseases and Sciences
IS - 5
ER -