Authors: Laurencin, Poujois, Bonjour, Demily, Klinger, Roze, Leclert, Danaila, Langlois-Jacques, Couchonnal, Woimant, Obadia, Perez, Pernon, Blanchet, Broussolle, Vidailhet, Kassai, Moro, Karachi, Polo, Grabli, Portefaix & Thobois (2025).
This study provides a rigorous evaluation of deep brain stimulation (DBS) for severe dystonia in Wilson disease (WD) through a prospective, multicentre N-of-1 trial. Conducted across French WD reference centres, the trial enrolled three patients with medically refractory dystonia, each undergoing alternating four-month "on" and "off" stimulation phases targeting either the subthalamic nucleus (STN) or the internal globus pallidus (GPi). Primary outcomes, assessed via the Canadian Occupational Performance Measure Performance (COPM-P) and Satisfaction scores, alongside secondary measures including the Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) and Unified Wilson's Disease Rating Scale (UWDRS), revealed no significant improvement in dystonia severity or quality of life. These findings challenge the therapeutic potential of DBS in WD-related dystonia, underscoring the need for further exploration of alternative treatment strategies. Notably, the study reported no serious adverse events, reinforcing the safety profile of DBS in this patient population.
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Full reference: Laurencin, C., Poujois, A., Bonjour, M., Demily, C., Klinger, H., Roze, E., Leclert, V., Danaila, T., Langlois-Jacques, C., Couchonnal, E., Woimant, F., Obadia, M. A., Perez, G., Pernon, M., Blanchet, L., Broussolle, E., Vidailhet, M., Kassai, B., Moro, E., Karachi, C., Polo, G., Grabli, D., Portefaix, A., & Thobois, S. (2025). Deep brain stimulation for severe dystonia associated with Wilson disease: A prospective multicenter meta-analysis of an N-of-1 trial. European Journal of Neurology, 32(1), e16524.
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