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

Aftab Ala, Ermal Aliu, Michael L Schilsky, Aftab Ala, Ermal Aliu, Michael L Schilsky

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.

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).

Figures

Fig. 1
Fig. 1
The graph demonstrates the variation of ALT during the period of the experimental study. Visits 1–4 were the pre-treatment and baseline visits. With upper limit of normal at 40, only 3 were above at the start. All the patients had end values below the 0 time point at the end of the study except series 3 but that patient had a value below 100 a year later

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