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Web End = Dig Dis Sci (2015) 60:14331439 DOI 10.1007/s10620-014-3495-6
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Web End = Prospective Pilot Study of a Single Daily Dosage of Trientine for the Treatment of Wilson Disease
Aftab Ala Ermal Aliu Michael L. Schilsky
Received: 23 April 2014 / Accepted: 15 December 2014 / Published online: 21 January 2015 The Author(s) 2015. This article is published with open access at Springerlink.com
AbstractBackground 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 2271 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 uctuated 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 lg, 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 efcacy of this once-daily treatment regimen for WD (registration: NCT01472874).
Keywords Wilson disease Treatment Trientine
Adherence
AbbreviationsALT Alananine aminotransferase AST Aspartate aminotransferase INR International normalized ratio WD Wilson disease
Introduction
Wilson disease (WD) is an autosomal recessive disorder of copper metabolism affecting *1:30,000 individuals [1, 2].
Liver and neurological injury may be prevented or treated by lifelong medical therapy with D-penicillamine or trientine, or zinc salts that inhibit copper absorption. Up to 50 % of WD patients have medication non-adherence [3]. Failure to adhere to treatment inevitably leads to neurological injury and psychiatric symptoms, hepatic failure, and ultimately death [4].
Trientine was approved for WD patients intolerant of D-
penicillamine [5, 6]. Trientine is gaining popularity as a rst-line agent due to its favorable safety prole. Trientine is conventionally dosed at 7501,500 mg/day in 24 divided...