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  • Cited by 3
  • Print publication year: 2008
  • Online publication date: May 2010

Tetrathiomolybdate versus Trientine in the Initial Treatment of Neurologic Wilson's Disease

    • By George J. Brewer, Department of Human Genetics, Department of Internal Medicine, University of Michigan, Ann Arbor, MI, USA, Fred Askari, Department of Internal Medicine, University of Michigan, Ann Arbor, MI, USA, Matthew T. Lorincz, Department of Neurology, University of Michigan, Ann Arbor, MI, USA, Martha Carlson, Department of Pediatrics-Neurology, University of Michigan, Ann Arbor, MI, USA, Michael Schilsky, Department of Internal Medicine, Cornell University, New York, NY, USA, Karen J. Kluin, Department of Neurology, Department of Speech Pathology, University of Michigan, Ann Arbor, MI, USA, Peter Hedera, Department of Neurology, Vanderbilt University, Nashville, TN, USA, Paolo Moretti, Departments of Neurology and Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA, John K. Fink, Department of Neurology, University of Michigan, Ann Arbor, MI, USA, Roberta Tankanow, College of Pharmacy, University of Michigan, Ann Arbor, MI, USA, Robert B. Dick, Department of Human Genetics, University of Michigan, Ann Arbor, MI, USA, Julia Sitterly, Department of Human Genetics, University of Michigan, Ann Arbor, MI, USA
  • Edited by Jeffrey L. Cummings
  • Publisher: Cambridge University Press
  • DOI: https://doi.org/10.1017/CBO9780511666971.010
  • pp 153-166

Summary

ABSTRACT

Background: The initial treatment of the neurologic presentation of Wilson's disease is problematic. Penicillamine, used for years on most patients, causes neurologic worsening in up to half of such patients, and half of those who worsen never recover. Zinc, ideal for maintenance therapy, is too slow for these acutely ill patients. We have developed tetrathiomolybdate (TM) for this type of patient, and it has worked well in open label studies. Trientine, another anticopper drug on the market approved for penicillamine intolerant patients, had not been tried in this type of patient. Here, we report on a double blind trial of TM versus trientine in the neurologically presenting Wilson's disease patient. Design and Methods: The study was a double blind design in which patients received either TM plus zinc, or trientine plus zinc, for 8 weeks. Patients were accepted if they presented with neurologic symptoms from Wilson's disease, if they had not been treated longer than 4 weeks with penicillamine or trientine. Patients were followed in the hospital for the 8 weeks of treatment with weekly semiquantitative neurologic and speech examinations, to evaluate possible neurologic worsening. They also had blood and urine studies done weekly. At discharge from hospital they were continued on zinc maintenance therapy, and returned at yearly intervals for 3 years for further evaluation. Results: Twenty-three patients were entered into the trientine arm and 6 reached criteria for neurologic deterioration, while 25 patients were entered into the TM arm and only 1 deteriorated (p < 0.05).