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Case Reports
. 2020 May;10(5):e01596.
doi: 10.1002/brb3.1596. Epub 2020 Mar 22.

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

Oriol De Fabregues  1 , Jaume Viñas  1 , Antoni Palasí  1 , Manuel Quintana  1 , Ignasi Cardona  2 , Cristina Auger  3 , Víctor Vargas  4
Affiliations
Case Reports

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

Oriol De Fabregues et al. Brain Behav. 2020 May.

Abstract

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.

Keywords: Wilson's disease; ammonium tetrathiomolybdate; efficacy; neurological symptoms; safety.

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Conflict of interest statement

On behalf of all authors, the corresponding author states that there is no conflict of interest.

Figures

Figure 1
Figure 1
Neuroimaging improvement with brain edema reduction 3 months after starting treatment with ATTM and persistence of chronic necrotic lesions of basal ganglia. Patient 2. (a). Pre‐ATTM brain MRI. Increase in signal intensity on T2‐weighted images involving diffusely the pons (arrowhead), and the external border of lenticular nuclei (arrows). (b). Post‐ATTM brain MRI. Observe the almost complete resolution of the diffuse pontine signal abnormalities, but persistent T2 signal abnormalities associated with atrophy, likely related to necrosis, of both putamina
See this image and copyright information in PMC

References

    1. Aggarwal, A. , Aggarwal, N. , Nagral, A. , Jankharia, G. , & Bhatt, M. (2009). A novel Global Assessment Scale for Wilson's Disease (GAS for WD). Movement Disorders, 24, 509–518. 10.1002/mds.22231 - DOI - PubMed
    1. Aggarwal, A. , & Bhatt, M. (2018). Advances in treatment of Wilson disease. Tremor and Other Hyperkinetic Movements, 8, 525 10.7916/D841881D - DOI - PMC - PubMed
    1. Ala, A. , Walker, A. P. , Ashkan, K. , Dooley, J. S. , & Schilsky, M. L. (2007). Wilson's disease. The Lancet, 369, 397–408. 10.1016/S0140-6736(07)60196-2 - DOI - PubMed
    1. Appenzeller‐Herzog, C. , Mathes, T. , Heeres, M. L. S. , Weiss, K. H. , Houwen, R. H. J. , & Ewald, H. (2019). Comparative effectiveness of common therapies for Wilson disease: A systematic review and meta‐analysis of controlled studies. Liver International, 39(11), 2136–2152. 10.1111/liv.14179 - DOI - PubMed
    1. Brewer, G. J. , & Askari, F. K. (2005). Wilson disease: Clinical management and therapy. Journal of Hepatology, 42, S13–S21. 10.1016/j.jhep.2004.11.013 - DOI - PubMed

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