Patient Selection for Deep Brain Stimulation for Pantothenate Kinase-Associated Neurodegeneration.

IF 2.5 Q2 CLINICAL NEUROLOGY Tremor and Other Hyperkinetic Movements Pub Date : 2024-10-17 eCollection Date: 2024-01-01 DOI:10.5334/tohm.929

Jason L Chan, Ashley E Rawls, Joshua K Wong, Penelope Hogarth, Justin D Hilliard, Michael S Okun

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Abstract

Clinical vignette: A 23-year-old woman with pantothenate kinase-associated neurodegeneration (PKAN) presented with medication-refractory generalized dystonia and an associated gait impairment.

Clinical dilemma: Bilateral globus pallidus internus (GPi) deep brain stimulation (DBS) can be an effective treatment for dystonia. However, outcomes for PKAN DBS have been variable and there are no standardized criteria for patient selection.

Clinical solution: Bilateral GPi DBS implantation resulted in improvement in dystonia and gait. The benefit has persisted over one year after implantation.

Gap in knowledge: PKAN is a rare neurodegenerative disorder and evidence supporting the use of PKAN DBS has been largely limited to case reports and case series. Consequently, there is a paucity of long-term data, especially on gait-related outcomes.

Expert commentary: The clinical characteristics of dystonia that respond to DBS tend to respond in PKAN. Clinicians counselling patients about the effects of DBS for PKAN should thoughtfully discuss gait and postural instability as important aspects to consider, especially as the disease will progress post-DBS.

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选择脑深部刺激治疗泛酸激酶相关神经变性的患者。

临床小故事临床困境：双侧苍白球内肌（GPi）深部脑刺激（DBS）可以有效治疗肌张力障碍。然而，PKAN DBS 的疗效参差不齐，而且在患者选择方面也没有统一的标准：双侧 GPi DBS 植入术改善了肌张力障碍和步态。临床解决方案：双侧 GPi DBS 植入术后，肌张力障碍和步态得到改善，植入一年后疗效仍在：PKAN 是一种罕见的神经退行性疾病，支持使用 PKAN DBS 的证据主要局限于病例报告和系列病例。因此，长期数据很少，尤其是步态相关的结果：专家评论：对 DBS 有反应的肌张力障碍的临床特征往往在 PKAN 中也有反应。临床医生在向患者提供有关 DBS 对 PKAN 的影响的咨询时，应深思熟虑地讨论步态和姿势不稳定性，因为这是需要考虑的重要方面，尤其是在 DBS 治疗后疾病会进展的情况下。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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