Prospective pilot study of a single daily dosage of trientine for the treatment of Wilson disease

Dig Dis Sci. 2015 May;60(5):1433-9. doi: 10.1007/s10620-014-3495-6. Epub 2015 Jan 21.


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.

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).

Publication types

  • Clinical Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Administration, Oral
  • Adult
  • Aged
  • Chelating Agents / administration & dosage*
  • Chelating Agents / adverse effects
  • Drug Administration Schedule
  • Female
  • Hepatolenticular Degeneration / diagnosis
  • Hepatolenticular Degeneration / drug therapy*
  • Humans
  • Male
  • Medication Adherence
  • Middle Aged
  • Patient Dropouts
  • Patient Satisfaction
  • Pilot Projects
  • Prospective Studies
  • Surveys and Questionnaires
  • Time Factors
  • Treatment Outcome
  • Trientine / administration & dosage*
  • Trientine / adverse effects
  • Young Adult


  • Chelating Agents
  • Trientine

Associated data