Format

Send to

Choose Destination
Arch Neurol. 2006 Apr;63(4):521-7.

Treatment of Wilson disease with ammonium tetrathiomolybdate: IV. Comparison of tetrathiomolybdate and trientine in a double-blind study of treatment of the neurologic presentation of Wilson disease.

Author information

1
Department of Human Genetics, University of Michigan Medical School, 5024 Kresge Bldg. II, Ann Arbor, MI 48109-0534, USA. brewergj@umich.edu

Abstract

OBJECTIVE:

To compare tetrathiomolybdate and trientine in treating patients with the neurologic presentation of Wilson disease for the frequency of neurologic worsening, adverse effects, and degree of neurologic recovery.

DESIGN:

A randomized, double-blind, controlled, 2-arm study of 48 patients with the neurologic presentation of Wilson disease. Patients either received 500 mg of trientine hydrochloride 2 times per day or 20 mg of tetrathiomolybdate 3 times per day with meals and 20 mg 3 times per day between meals for 8 weeks. All patients received 50 mg of zinc 2 times per day. Patients were hospitalized for 8 weeks, with neurologic and speech function assessed weekly; discharged taking 50 mg of zinc 3 times per day, and returned annually for follow-up.

SETTING:

A university hospital referral setting.

PATIENTS:

Primarily newly diagnosed patients with Wilson disease presenting with neurologic symptoms who had not been treated longer than 4 weeks with an anticopper drug.

INTERVENTION:

Treatment with either trientine plus zinc or tetrathiomolybdate plus zinc.

MAIN OUTCOME MEASURES:

Neurologic function was assessed by semiquantitative neurologic and speech examinations. Drug adverse events were evaluated by blood cell counts and biochemical measures.

RESULTS:

Six of 23 patients in the trientine arm and 1 of 25 patients in the tetrathiomolybdate arm underwent neurologic deterioration (P<.05). Three patients receiving tetrathiomolybdate had adverse effects of anemia and/or leukopenia, and 4 had further transaminase elevations. One patient receiving trientine had an adverse effect of anemia. Four patients receiving trientine died during follow-up, 3 having shown initial neurologic deterioration. Neurologic and speech recovery during a 3-year follow-up period were quite good.

CONCLUSION:

Tetrathiomolybdate is a better choice than trientine for preserving neurologic function in patients who present with neurologic disease.

TRIAL REGISTRATION:

ClinicalTrials.gov NCT00004339.

PMID:
16606763
DOI:
10.1001/archneur.63.4.521
[Indexed for MEDLINE]

Supplemental Content

Full text links

Icon for Silverchair Information Systems
Loading ...
Support Center