肌萎缩综合征治疗指南。

IF 4.7 2区 医学 Q1 CLINICAL NEUROLOGY
Therapeutic Advances in Neurological Disorders Pub Date : 2023-12-26 eCollection Date: 2023-01-01 DOI:10.1177/17562864231213240
Heinz Wiendl, Angela Abicht, Andrew Chan, Adela Della Marina, Tim Hagenacker, Khosro Hekmat, Sarah Hoffmann, Hans-Stefan Hoffmann, Sebastian Jander, Christian Keller, Alexander Marx, Arthur Melms, Nico Melzer, Wolfgang Müller-Felber, Marc Pawlitzki, Jens-Carsten Rückert, Christiane Schneider-Gold, Benedikt Schoser, Bettina Schreiner, Michael Schroeter, Bettina Schubert, Jörn-Peter Sieb, Fritz Zimprich, Andreas Meisel
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引用次数: 0

摘要

重症肌无力(MG)、兰伯特-伊顿肌无力综合征(LEMS)和先天性肌无力综合征(CMS)是一组病因异构的(非常)罕见慢性疾病。MG 和 LEMS 的病因是自身免疫介导,而 CMS 则是遗传性疾病。神经肌肉传递障碍导致的(应变依赖性)肌无力是其共同特征。全身性 MG 需要越来越多的差异化治疗策略,并考虑到近年来治疗方面的巨大发展。为了纳入最新的治疗建议,德国神经病学协会在跨学科专家小组的协助下,对现有的德语指南《肌无力综合征的诊断和治疗》进行了全面更新。本文是对更新版和部分新制定的治疗指南的改编翻译。该指南将肌无力患者迅速实现疾病完全控制作为治疗的核心目标。标准疗法和现代免疫疗法的使用应根据自身抗体状态和疾病活动情况分阶段进行。随着现代速效免疫调节剂的出现,疾病活动性评估变得至关重要,需要对临床病程(包括严重程度和所需疗法)进行评估。应用MG专用评分和分类,如日常生活活动肌无力、定量肌无力和美国肌无力基金会,可以区分轻度/中度和(高度)活动性(包括难治性)疾病。治疗决策必须考虑年龄、胸腺病理、抗体状态和疾病活动性。糖皮质激素和经典免疫抑制剂(主要是硫唑嘌呤)是治疗轻度/中度至(高度)活动性全身性 MG/幼年 MG 和眼部 MG 的基本免疫疗法。胸腺切除术适用于治疗胸腺肿瘤相关性 MG 和乙酰胆碱受体抗体(AChR-Ab)阳性的全身性 MG。对于乙酰胆碱受体抗体(AChR-Ab)阳性的(高度)活动性全身性 MG,建议使用补体抑制剂(目前为 eculizumab 和 ravulizumab)或新生儿 Fc 受体调节剂(目前为 efgartigimod);对于肌肉特异性受体酪氨酸激酶(MuSK)-Ab 阳性的 MG,建议使用利妥昔单抗。肌无力危象的特殊治疗需要进行浆细胞分离、免疫吸附或静脉注射免疫球蛋白。本指南还强调了眼肌型、幼年型和先天性肌无力症的具体治疗方法。该指南将根据其他免疫调节剂和有助于准确测量疾病活动性的生物标志物的新研究结果进一步完善。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Guideline for the management of myasthenic syndromes.

Myasthenia gravis (MG), Lambert-Eaton myasthenic syndrome (LEMS), and congenital myasthenic syndromes (CMS) represent an etiologically heterogeneous group of (very) rare chronic diseases. MG and LEMS have an autoimmune-mediated etiology, while CMS are genetic disorders. A (strain dependent) muscle weakness due to neuromuscular transmission disorder is a common feature. Generalized MG requires increasingly differentiated therapeutic strategies that consider the enormous therapeutic developments of recent years. To include the newest therapy recommendations, a comprehensive update of the available German-language guideline 'Diagnostics and therapy of myasthenic syndromes' has been published by the German Neurological society with the aid of an interdisciplinary expert panel. This paper is an adapted translation of the updated and partly newly developed treatment guideline. It defines the rapid achievement of complete disease control in myasthenic patients as a central treatment goal. The use of standard therapies, as well as modern immunotherapeutics, is subject to a staged regimen that takes into account autoantibody status and disease activity. With the advent of modern, fast-acting immunomodulators, disease activity assessment has become pivotal and requires evaluation of the clinical course, including severity and required therapies. Applying MG-specific scores and classifications such as Myasthenia Gravis Activities of Daily Living, Quantitative Myasthenia Gravis, and Myasthenia Gravis Foundation of America allows differentiation between mild/moderate and (highly) active (including refractory) disease. Therapy decisions must consider age, thymic pathology, antibody status, and disease activity. Glucocorticosteroids and the classical immunosuppressants (primarily azathioprine) are the basic immunotherapeutics to treat mild/moderate to (highly) active generalized MG/young MG and ocular MG. Thymectomy is indicated as a treatment for thymoma-associated MG and generalized MG with acetylcholine receptor antibody (AChR-Ab)-positive status. In (highly) active generalized MG, complement inhibitors (currently eculizumab and ravulizumab) or neonatal Fc receptor modulators (currently efgartigimod) are recommended for AChR-Ab-positive status and rituximab for muscle-specific receptor tyrosine kinase (MuSK)-Ab-positive status. Specific treatment for myasthenic crises requires plasmapheresis, immunoadsorption, or IVIG. Specific aspects of ocular, juvenile, and congenital myasthenia are highlighted. The guideline will be further developed based on new study results for other immunomodulators and biomarkers that aid the accurate measurement of disease activity.

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来源期刊
CiteScore
8.30
自引率
1.70%
发文量
62
审稿时长
15 weeks
期刊介绍: Therapeutic Advances in Neurological Disorders is a peer-reviewed, open access journal delivering the highest quality articles, reviews, and scholarly comment on pioneering efforts and innovative studies across all areas of neurology. The journal has a strong clinical and pharmacological focus and is aimed at clinicians and researchers in neurology, providing a forum in print and online for publishing the highest quality articles in this area.
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