Wojtecki, L., Groiss, S. J., Ferrea, S., Elben, S., Hartmann, C. J., Dunnett, Stephen Bruce ORCID: https://orcid.org/0000-0003-1826-1578, Rosser, Anne Elizabeth ORCID: https://orcid.org/0000-0002-4716-4753, Saft, C., Sudmeyer, M., Ohmann, C., Schnitzler, A. and Vesper, J. 2015. A prospective pilot trial for pallidal deep brain stimulation in Huntington's Disease. Frontiers in Neurology 6 , 177. 10.3389/fneur.2015.00177 |
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Abstract
BACKGROUND: Movement disorders in Huntington's disease are often medically refractive. The aim of the trial was assessment of procedure safety of deep brain stimulation, equality of internal- and external-pallidal stimulation and efficacy followed-up for 6 months in a prospective pilot trial. METHODS: In a controlled double-blind phase six patients (four chorea-dominant, two Westphal-variant) with predominant movement disorder were randomly assigned to either the sequence of 6-week internal- or 6-week external-pallidal stimulation, or vice versa, followed by further 3 months chronic pallidal stimulation at the target with best effect-side-effect ratio. Primary endpoints were changes in the Unified Huntington's Disease Rating Scale motor-score, chorea subscore, and total motor-score 4 (blinded-video ratings), comparing internal- versus external-pallidal stimulation, and 6 months versus baseline. Secondary endpoints assessed scores on dystonia, hypokinesia, cognition, mood, functionality/disability, and quality-of-life. RESULTS: Intention-to-treat analysis of all patients (n = 3 in each treatment sequence): Both targets were equal in terms of efficacy. Chorea subscores decreased significantly over 6 months (-5.3 (60.2%), p = 0.037). Effects on dystonia were not significant over the group due to it consisting of three responders (>50% improvement) and three non-responders. Westphal patients did not improve. Cognition was stable. Mood and some functionality/disability and quality-of-life scores improved significantly. Eight adverse events and two additional serious adverse events - mostly internal-pallidal stimulation-related - resolved without sequalae. No procedure-related complications occurred. CONCLUSION: Pallidal deep brain stimulation was demonstrated to be a safe treatment option for the reduction of chorea in Huntington's disease. Their effects on chorea and dystonia and on quality-of-life should be examined in larger controlled trials
Item Type: | Article |
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Date Type: | Publication |
Status: | Published |
Schools: | Biosciences MRC Centre for Neuropsychiatric Genetics and Genomics (CNGG) Medicine Neuroscience and Mental Health Research Institute (NMHRI) |
Subjects: | R Medicine > RC Internal medicine > RC0321 Neuroscience. Biological psychiatry. Neuropsychiatry |
Publisher: | Frontiers Research Foundation |
ISSN: | 1664-2295 |
Date of First Compliant Deposit: | 30 March 2016 |
Date of Acceptance: | 27 July 2015 |
Last Modified: | 05 May 2023 12:39 |
URI: | https://orca.cardiff.ac.uk/id/eprint/81564 |
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