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

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 mu g, 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).