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 - Funding Information:
Aftab Ala was supported by the Royal College of Physicians, London Sheila Sherlock Travel Fellowship in Hepatology and the Surrey and Sussex National Institute for Health and Research (NIHR). The project was funded by Aton and Valeant Pharmaceuticals with recruitment assistance from the Wilson Disease Association, USA.
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 -