title: Wilson's disease treatment by triethylene tetramine dihydrochloride (trientine, 2HCl): long-term observations.
abstract: Wilson's disease is an autosomal recessive disorder characterized by an accumulation of a toxic amount of copper in the body. Triethylene tetramine dihydrochloride (trientine, 2HCl) is a new chelating agent that may be effective in the removal of excess copper but long-term efficacy has not yet been investigated. Here we report the use of trientine over more than 8 years in 2 patients with Wilson's disease who could not tolerate D-penicillamine. We found no significant side effect, except a decreased serum iron concentration without clinical symptoms of anemia. In annual examinations at a steady state, the serum copper levels remained below 20 micrograms/100 ml. The 24-hour urinary copper excretion was less than that found using D-penicillamine, while the basal copper excretion, after 5 days abstinence from trientine, was maintained below 100 micrograms/day. Both hepatic and neurological manifestations except bulbar symptoms were recovered without any initial deterioration.
authors.affiliation: Department of Pediatrics and Child Health, Kurume University School of Medicine, Fukuoka, Japan.
authors.forename: J
authors.initials: J
authors.lastname: Morita
authors.forename: M
authors.initials: M
authors.lastname: Yoshino
authors.forename: H
authors.initials: H
authors.lastname: Watari
authors.forename: I
authors.initials: I
authors.lastname: Yoshida
authors.forename: T
authors.initials: T
authors.lastname: Motohiro
authors.forename: F
authors.initials: F
authors.lastname: Yamashita
authors.forename: Y
authors.initials: Y
authors.lastname: Okano
authors.forename: T
authors.initials: T
authors.lastname: Hashimoto
mesh_terms: D000293:Adolescent; D000328:Adult; D000410:Alanine Transaminase / Q000097:blood; D001219:Aspartate Aminotransferases / Q000097:blood; D002614:Chelating Agents / Q000009:adverse effects / Q000627:therapeutic use*; D003300:Copper / Q000097:blood / Q000652:urine; D005260:Female; D006527:Hepatolenticular Degeneration / Q000188:drug therapy*; D006801:Humans; D007501:Iron / Q000097:blood; D008297:Male; D010396:Penicillamine / Q000009:adverse effects / Q000627:therapeutic use; D014266:Trientine / Q000009:adverse effects / Q000627:therapeutic use*
chemical_list: D002614:Chelating Agents; D003300:Copper; D007501:Iron; D001219:Aspartate Aminotransferases; D000410:Alanine Transaminase; D010396:Penicillamine; D014266:Trientine