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代谢、发育和遗传障碍中的严重急性运动恶化(SAME)。

Severe Acute Motor Exacerbations (SAME) across Metabolic, Developmental and Genetic Disorders.

机构信息

Edmond J. Safra Program in Parkinson's Disease, Rossy PSP Centre and the Morton and Gloria Shulman Movement Disorders Clinic, Toronto Western Hospital, Toronto, Ontario, Canada.

Instituto de Neurociencia Cognitiva y Traslacional, INECO-Favaloro-CONICET, Buenos Aires, Argentina.

出版信息

Mov Disord. 2024 Sep;39(9):1446-1467. doi: 10.1002/mds.29905. Epub 2024 Aug 9.

Abstract

Acute presentation of severe motor disorders is a diagnostic and management challenge. We define severe acute motor exacerbations (SAME) as acute/subacute motor symptoms that persist for hours-to-days with a severity that compromise vital signs (temperature, breath, and heart rate) and bulbar function (swallowing/dysphagia). Phenomenology includes dystonia, choreoathetosis, combined movement disorders, weakness, and hemiplegic attacks. SAME can develop in diverse diseases and can be preceded by triggers or catabolic states. Recent descriptions of SAME in complex neurodevelopmental and epileptic encephalopathies have broadened appreciation of this presentation beyond inborn errors of metabolism. A high degree of clinical suspicion is required to identify appropriately targeted investigations and management. We conducted a comprehensive literature analysis of etiologies. Reported triggers are described and classified as per pathophysiological mechanism. A video of six cases displaying multiple SAME with diverse outcomes is provided. We identified 50 different conditions that manifest SAME, some associated with developmental regression. Etiologies include disorders of metabolism: energy substrate, amino acids, complex molecules, vitamins/cofactors, minerals, and neurotransmitters/synaptic vesicle cycling. Non-metabolic neurodegenerative and genetic disorders that present with movement disorders and epilepsy can additionally manifest SAME. A limited number of triggers are grouped here, together with an approach to investigations and general management strategies. Several neurogenetic and neurometabolic disorders manifest SAME. Identifying triggers can help in certain cases narrow the differential diagnosis and guide the expeditious application of targeted therapies to minimize adverse developmental and neurological consequences. This process may inform pathogenesis and eventually improve our understanding of the mechanisms that lead to the development of SAME. © 2024 International Parkinson and Movement Disorder Society.

摘要

严重运动障碍的急性发作是一个诊断和管理方面的挑战。我们将严重急性运动恶化(SAME)定义为持续数小时至数天的急性/亚急性运动症状,其严重程度会影响生命体征(体温、呼吸和心率)和延髓功能(吞咽/吞咽困难)。表现形式包括肌张力障碍、舞蹈手足徐动症、运动障碍合并、无力和偏瘫发作。SAME 可在多种疾病中发生,并且可能有诱因或分解代谢状态。最近在复杂的神经发育和癫痫性脑病中对 SAME 的描述拓宽了对这种表现的认识,超出了代谢性遗传病的范围。需要高度的临床怀疑才能识别出适当的靶向检查和管理。我们对病因进行了全面的文献分析。报告的诱因按病理生理机制进行描述和分类。提供了六个显示多种 SAME 和不同结局的病例的视频。我们确定了 50 种不同的疾病表现为 SAME,其中一些与发育性倒退有关。病因包括代谢紊乱:能量底物、氨基酸、复杂分子、维生素/辅助因子、矿物质和神经递质/突触小泡循环。伴有运动障碍和癫痫的非代谢性神经退行性和遗传疾病也可能表现为 SAME。这里将一些有限的诱因分组,并提供了一种针对检查和一般管理策略的方法。有几种神经遗传和神经代谢疾病表现为 SAME。识别诱因可以帮助某些情况下缩小鉴别诊断范围,并指导迅速应用靶向治疗以最大程度减少不良的发育和神经后果。这个过程可能会为发病机制提供信息,并最终提高我们对导致 SAME 发展的机制的理解。

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