Chloé Laurencin, Aurelia Poujois, Maxime Bonjour, Caroline Demily, Hélène Klinger, Emmanuel Roze, Victoire Leclert, Teodor Danaila, Carole Langlois-Jacques, Eduardo Couchonnal, France Woimant, Mickael Alexandre Obadia, Gwennaelle Perez, Michaela Pernon, Laurianne Blanchet, Emmanuel Broussolle, Marie Vidailhet, Behrouz Kassai, Elena Moro, Carine Karachi, Gustavo Polo, David Grabli, Aurélie Portefaix, Stéphane Thobois
{"title":"深部脑刺激治疗与威尔逊氏病相关的严重肌张力障碍：一项N-of-1试验的前瞻性多中心荟萃分析。","authors":"Chloé Laurencin, Aurelia Poujois, Maxime Bonjour, Caroline Demily, Hélène Klinger, Emmanuel Roze, Victoire Leclert, Teodor Danaila, Carole Langlois-Jacques, Eduardo Couchonnal, France Woimant, Mickael Alexandre Obadia, Gwennaelle Perez, Michaela Pernon, Laurianne Blanchet, Emmanuel Broussolle, Marie Vidailhet, Behrouz Kassai, Elena Moro, Carine Karachi, Gustavo Polo, David Grabli, Aurélie Portefaix, Stéphane Thobois","doi":"10.1111/ene.16524","DOIUrl":null,"url":null,"abstract":"<p><strong>Background and purpose: </strong>Disabling dystonia despite optimal medical treatment is common in Wilson disease (WD). No controlled study has evaluated the effect of deep brain stimulation (DBS) on dystonia related to WD. This study was undertaken to evaluate the efficacy of DBS on dystonia related to WD.</p><p><strong>Methods: </strong>A meta-analysis of an N-of-1 prospective, randomized, double-blind, multicenter DBS study was conducted at two French WD reference centers. Main inclusion criteria were patients with WD, stabilized for at least 6 months with significant disability due to dystonia despite optimized medical treatment. The subthalamic nucleus (STN) was targeted for bradykinetic patients with tonic dystonia, and the internal globus pallidus (GPi) was chosen for patients with hyperkinetic dystonia. Each patient underwent two periods of DBS \"on\" and two periods of DBS \"off,\" each lasting 4 months. The order of stimulation conditions was randomized. The primary outcome was the change in the Canadian Occupational Performance Measure Performance (COPM-P) and Satisfaction scores after each 4-month period. Secondary outcomes were changes in the Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) severity and disability scores and Unified Wilson's Disease Rating Scale (UWDRS) scores.</p><p><strong>Results: </strong>Between 12 May 2016 and 7 October 2022, three patients were included. Two patients received bilateral GPi DBS, and one received bilateral STN DBS. There was no change of COPM-P (p = 0.956), BFMDRS, and UWDRS scores. No serious adverse events were reported.</p><p><strong>Conclusions: </strong>STN or GPi DBS are ineffective on dystonia related to WD.</p>","PeriodicalId":11954,"journal":{"name":"European Journal of Neurology","volume":" ","pages":"e16524"},"PeriodicalIF":4.5000,"publicationDate":"2024-10-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Deep brain stimulation for severe dystonia associated with Wilson disease: A prospective multicenter meta-analysis of an N-of-1 trial.\",\"authors\":\"Chloé Laurencin, Aurelia Poujois, Maxime Bonjour, Caroline Demily, Hélène Klinger, Emmanuel Roze, Victoire Leclert, Teodor Danaila, Carole Langlois-Jacques, Eduardo Couchonnal, France Woimant, Mickael Alexandre Obadia, Gwennaelle Perez, Michaela Pernon, Laurianne Blanchet, Emmanuel Broussolle, Marie Vidailhet, Behrouz Kassai, Elena Moro, Carine Karachi, Gustavo Polo, David Grabli, Aurélie Portefaix, Stéphane Thobois\",\"doi\":\"10.1111/ene.16524\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background and purpose: </strong>Disabling dystonia despite optimal medical treatment is common in Wilson disease (WD). No controlled study has evaluated the effect of deep brain stimulation (DBS) on dystonia related to WD. This study was undertaken to evaluate the efficacy of DBS on dystonia related to WD.</p><p><strong>Methods: </strong>A meta-analysis of an N-of-1 prospective, randomized, double-blind, multicenter DBS study was conducted at two French WD reference centers. Main inclusion criteria were patients with WD, stabilized for at least 6 months with significant disability due to dystonia despite optimized medical treatment. The subthalamic nucleus (STN) was targeted for bradykinetic patients with tonic dystonia, and the internal globus pallidus (GPi) was chosen for patients with hyperkinetic dystonia. Each patient underwent two periods of DBS \\\"on\\\" and two periods of DBS \\\"off,\\\" each lasting 4 months. The order of stimulation conditions was randomized. The primary outcome was the change in the Canadian Occupational Performance Measure Performance (COPM-P) and Satisfaction scores after each 4-month period. Secondary outcomes were changes in the Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) severity and disability scores and Unified Wilson's Disease Rating Scale (UWDRS) scores.</p><p><strong>Results: </strong>Between 12 May 2016 and 7 October 2022, three patients were included. Two patients received bilateral GPi DBS, and one received bilateral STN DBS. There was no change of COPM-P (p = 0.956), BFMDRS, and UWDRS scores. No serious adverse events were reported.</p><p><strong>Conclusions: </strong>STN or GPi DBS are ineffective on dystonia related to WD.</p>\",\"PeriodicalId\":11954,\"journal\":{\"name\":\"European Journal of Neurology\",\"volume\":\" \",\"pages\":\"e16524\"},\"PeriodicalIF\":4.5000,\"publicationDate\":\"2024-10-29\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"European Journal of Neurology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1111/ene.16524\",\"RegionNum\":2,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"CLINICAL NEUROLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"European Journal of Neurology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1111/ene.16524","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"CLINICAL NEUROLOGY","Score":null,"Total":0}
Deep brain stimulation for severe dystonia associated with Wilson disease: A prospective multicenter meta-analysis of an N-of-1 trial.
Background and purpose: Disabling dystonia despite optimal medical treatment is common in Wilson disease (WD). No controlled study has evaluated the effect of deep brain stimulation (DBS) on dystonia related to WD. This study was undertaken to evaluate the efficacy of DBS on dystonia related to WD.
Methods: A meta-analysis of an N-of-1 prospective, randomized, double-blind, multicenter DBS study was conducted at two French WD reference centers. Main inclusion criteria were patients with WD, stabilized for at least 6 months with significant disability due to dystonia despite optimized medical treatment. The subthalamic nucleus (STN) was targeted for bradykinetic patients with tonic dystonia, and the internal globus pallidus (GPi) was chosen for patients with hyperkinetic dystonia. Each patient underwent two periods of DBS "on" and two periods of DBS "off," each lasting 4 months. The order of stimulation conditions was randomized. The primary outcome was the change in the Canadian Occupational Performance Measure Performance (COPM-P) and Satisfaction scores after each 4-month period. Secondary outcomes were changes in the Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) severity and disability scores and Unified Wilson's Disease Rating Scale (UWDRS) scores.
Results: Between 12 May 2016 and 7 October 2022, three patients were included. Two patients received bilateral GPi DBS, and one received bilateral STN DBS. There was no change of COPM-P (p = 0.956), BFMDRS, and UWDRS scores. No serious adverse events were reported.
Conclusions: STN or GPi DBS are ineffective on dystonia related to WD.
期刊介绍：
The European Journal of Neurology is the official journal of the European Academy of Neurology and covers all areas of clinical and basic research in neurology, including pre-clinical research of immediate translational value for new potential treatments. Emphasis is placed on major diseases of large clinical and socio-economic importance (dementia, stroke, epilepsy, headache, multiple sclerosis, movement disorders, and infectious diseases).
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