Prospective Pilot Study of a Single Daily Dosage of Trientine for the Treatment of Wilson Disease

Aftab Ala, Ermal Aliu, Michael L. Schilsky

Research output: Contribution to journalArticle

17 Citations (Scopus)

Abstract

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).

Original languageEnglish (US)
Pages (from-to)1433-1439
Number of pages7
JournalDigestive Diseases and Sciences
Volume60
Issue number5
DOIs
StatePublished - May 1 2015

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Trientine
Hepatolenticular Degeneration
Prospective Studies
Therapeutics
Zinc
Copper
Urine
Safety
Liver

All Science Journal Classification (ASJC) codes

  • Physiology
  • Gastroenterology

Cite this

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title = "Prospective Pilot Study of a Single Daily Dosage of Trientine for the Treatment of Wilson Disease",
abstract = "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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Prospective Pilot Study of a Single Daily Dosage of Trientine for the Treatment of Wilson Disease. / Ala, Aftab; Aliu, Ermal; Schilsky, Michael L.

In: Digestive Diseases and Sciences, Vol. 60, No. 5, 01.05.2015, p. 1433-1439.

Research output: Contribution to journalArticle

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