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.
  
  
  OBJECTIVE
  To prospectively evaluate once-daily trientine as therapy for Wilson disease.
  
  
  METHODS
  
  
  
  METHODS
  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).
authors.affiliation: Faculty of Health and Medical Sciences, Faculty of Health Management and Strategy, University of Surrey, Guildford, Surrey, GU2 7JP, UK.
authors.forename: Aftab
authors.initials: A
authors.lastname: Ala
authors.forename: Ermal
authors.initials: E
authors.lastname: Aliu
authors.forename: Michael L
authors.initials: ML
authors.lastname: Schilsky
mesh_terms: D000284:Administration, Oral; D000328:Adult; D000368:Aged; D002614:Chelating Agents / Q000008:administration & dosage* / Q000009:adverse effects; D004334:Drug Administration Schedule; D005260:Female; D006527:Hepatolenticular Degeneration / Q000175:diagnosis / Q000188:drug therapy*; D006801:Humans; D008297:Male; D055118:Medication Adherence; D008875:Middle Aged; D010352:Patient Dropouts; D017060:Patient Satisfaction; D010865:Pilot Projects; D011446:Prospective Studies; D011795:Surveys and Questionnaires; D013997:Time Factors; D016896:Treatment Outcome; D014266:Trientine / Q000008:administration & dosage* / Q000009:adverse effects; D055815:Young Adult
chemical_list: D002614:Chelating Agents; D014266:Trientine