Long-Term Outcomes on Pallidal Neurostimulation for Dystonia: A Controlled, Prospective 10-Year Follow-Up

IF 7.6 1区医学 Q1 CLINICAL NEUROLOGY Movement Disorders Pub Date : 2025-02-05 DOI:10.1002/mds.30130

Patricia Krause MD, Philipp Mahlknecht MD, PhD, Inger Marie Skogseid MD, PhD, Frank Steigerwald MD, Günther Deuschl MD, PhD, Richard Erasmi MD, Alfons Schnitzler MD, Tobias Warnecke MD, Jörg Müller MD, Werner Poewe MD, Gerd-Helge Schneider MD, Jan Vesper MD, Nils Warneke MD, Wilhelm Eisner MD, Thomas Prokop MD, Jan-Uwe Müller MD, Jens Volkmann MD, PhD, Andrea A. Kühn MD, PhD, for the Deep-Brain Stimulation for Dystonia Study Group

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Abstract

Background

Pallidal neurostimulation is an effective treatment for severe isolated dystonia, but long-term data from clinical trials are lacking.

Objectives

To evaluate long-term efficacy and safety of pallidal neurostimulation in patients with isolated generalized or segmental dystonia.

Methods

Extension study of the prospective multicenter trial (n = 40; July 2002 to May 2004), all patients received effective stimulation and underwent regular follow-up. The 10-year follow-up (n = 31) included Burke–Fahn–Marsden Dystonia Rating Scale (BFMDRS) motor and disability score, Beck Depression Inventory, Beck Anxiety Inventory, and Mattis Dementia Rating Scale. Primary and secondary endpoints compared motor symptoms, disability scores, mood, and cognition changes.

Results

Thirty-one patients (12 female), aged 23–72 years, completed the 10-year study extension. Per protocol analysis showed sustained significant improvement in BFMDRS motor scores at 10 years compared with baseline, without significant change from the 6-month or 5-year follow-up. On average, motor scores decreased by 25.3 ± 5.2 points at 10 years (P < 0.0001; 56% improvement). Individual outcomes varied, with 27 responders (≥25% improvement; mean improvement 65.2 ± 21.4%) and 13 non-responders compared with baseline. Sustained improvements were seen in disability, mood, and anxiety scores. Cognition remained stable.

Conclusions

This study presents the longest prospective, multicenter follow-up of pallidal neurostimulation in generalized and segmental dystonia. Two-thirds of patients showed strong and stable long-term improvements of dystonia, confirming sustained efficacy and safety over 10 years in treatment-refractory dystonic patients. However, one-third experienced primary (3/40) or secondary (10/40) treatment failure. Diagnostic advances, including genetic testing, and technological progress in pallidal neurostimulation may help to reduce the non-responder rates in the future. © 2025 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.

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苍白球神经刺激治疗肌张力障碍的长期疗效：一项控制性、前瞻性 10 年随访。

背景：苍白球神经刺激是一种治疗严重孤立性肌张力障碍的有效方法，但缺乏临床试验的长期数据：评估苍白球神经刺激对孤立性全身性或节段性肌张力障碍患者的长期疗效和安全性：前瞻性多中心试验的扩展研究（n = 40；2002 年 7 月至 2004 年 5 月），所有患者均接受了有效的刺激并进行了定期随访。10 年随访（n = 31）包括 Burke-Fahn-Marsden 肌张力障碍评定量表（BFMDRS）运动和残疾评分、贝克抑郁量表、贝克焦虑量表和马蒂斯痴呆评定量表。主要和次要终点比较运动症状、残疾评分、情绪和认知变化：31 名患者（12 名女性）完成了为期 10 年的延长研究，年龄在 23-72 岁之间。按方案进行的分析表明，与基线相比，10 年的 BFMDRS 运动评分有持续的明显改善，但与 6 个月或 5 年的随访结果相比没有显著变化。10年后，运动评分平均下降了25.3±5.2分（P 结论：BFMDRS运动评分与基线相比有明显改善，但与6个月或5年随访相比无明显变化：这项研究是对苍白球神经刺激治疗全身性和节段性肌张力障碍进行的时间最长的前瞻性多中心随访。三分之二的患者的肌张力障碍得到了长期、有力和稳定的改善，证实了对难治性肌张力障碍患者进行 10 年治疗的持续有效性和安全性。然而，三分之一的患者经历了原发性（3/40）或继发性（10/40）治疗失败。包括基因检测在内的诊断方法的进步以及苍白球神经刺激技术的进步可能有助于降低未来的无应答率。© 2025 The Author(s).运动障碍》由 Wiley Periodicals LLC 代表国际帕金森和运动障碍协会出版。

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来源期刊

Movement Disorders 医学-临床神经学

CiteScore

13.30

自引率

8.10%

发文量

371

审稿时长

12 months

期刊介绍： Movement Disorders publishes a variety of content types including Reviews, Viewpoints, Full Length Articles, Historical Reports, Brief Reports, and Letters. The journal considers original manuscripts on topics related to the diagnosis, therapeutics, pharmacology, biochemistry, physiology, etiology, genetics, and epidemiology of movement disorders. Appropriate topics include Parkinsonism, Chorea, Tremors, Dystonia, Myoclonus, Tics, Tardive Dyskinesia, Spasticity, and Ataxia.