Novel therapeutic approaches to the treatment of Wilson's disease.
The treatment of Wilson's disease has changed considerably in recent times, from the use of penicillamine (Cuprimine, Merck) for all stages and types of disease, to the use of three other anticopper drugs at appropriate times for appropriate patients. Each type and stage of the disease can be considered as a therapeutic target, for which specialised therapy is appropriate. This paper systematically reviews the various types and stages of Wilson's disease presentation, and provides opinion on the appropriate therapy for each. For patients presenting with neurological disease, the use of tetrathiomolybdate is optimum; for patients presenting with mild-to-moderate hepatic failure, a combination of trientine (Syprine, Merck) and zinc is recommended, whereas liver transplantation is necessary for those with severe failure; zinc therapy alone or trientine alone as second choice is recommended for patients presenting with hepatitis or cirrhosis without liver failure, for maintenance therapy, for treatment of presymptomatic patients and for treatment of paediatric and pregnant patients.
Brewer
George J
GJ
Department of Human Genetics, University of Michigan Medical School, Ann Arbor, MI 48109-0534, USA. brewergj@umich.edu
D002614:Chelating Agents / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D002986:Clinical Trials as Topic; D003300:Copper; D005260:Female; D006527:Hepatolenticular Degeneration / Q000175:diagnosis / Q000188:drug therapy*; D006801:Humans; D008982:Molybdenum / Q000627:therapeutic use*; D010396:Penicillamine / Q000627:therapeutic use*; D011247:Pregnancy; D011248:Pregnancy Complications / Q000175:diagnosis / Q000188:drug therapy*; D015032:Zinc / Q000627:therapeutic use*
D002614:Chelating Agents; D003300:Copper; D008982:Molybdenum; C020809:tetrathiomolybdate; D010396:Penicillamine; D015032:Zinc