Ammonium tetrathiomolybdate in the decoppering phase treatment of Wilson's disease with neurological symptoms: A case series.

OBJECTIVES: To present our experience with ammonium tetrathiomolybdate (ATTM) in the decoppering phase treatment of Wilson's disease (WD) with neurological symptoms. METHODS: An uncontrolled longitudinal study was carried out to describe a case series of five patients diagnosed of WD with neurological symptoms in our hospital over the last 5 years and receiving ATTM for 8 (or 16) weeks. Unified Wilson's Disease Rating Scale (UWDRS), Global Assessment Scale (GAS) for WD and the Brewer-adapted Unified Huntington's Disease Rating Scale (UHDRS) for WD, magnetic resonance imaging, and monitoring for potential adverse effects were carried out in all patients before starting ATTM and 3 months later when ATTM was stopped and zinc treatment was initiated. RESULTS: All five patients presented neurological clinical improvement in UWDRS, GAS, and Brewer-adapted UHDRS for WD. Neuroimaging improvement was present in 2/5 patients with brain edema reduction. Mild anemia, leukopenia, and elevation of transaminases were detected in 1 patient, with complete remission after stopping ATTM for 1 week and then restarting at a half dose. CONCLUSION: ATTM could be a good treatment for the initial treatment of WD with neurological symptoms due to its high efficacy, with a lower rate of neurological deterioration than the drugs currently available, despite the potential adverse effects.