Management challenges in muscle-specific tyrosine kinase myasthenia gravis
Management challenges in muscle-specific tyrosine kinase myasthenia gravis
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DOI：
10.1111/j.1749-6632.2012.06781.x
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发表时间：
2012-01-01
期刊：
影响因子：
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通讯作者：
Bartocccioni, Emanuela
中科院分区：
文献类型：
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作者：
Evoli, Amelia;Alboini, Paolo E.;Bartocccioni, Emanuela
伴有肌肉特异性酪氨酸激酶（MuSK-MG）抗体的重症肌无力通常被认为是一种严重的疾病，因为其相关的无力分布与球肌的普遍受累以及快速进展的病程和早期呼吸危象有关。由于大多数患者对乙酰胆碱酯酶抑制剂的反应较差，尽管使用了高剂量的免疫抑制剂，但疾病仍会复发，并出现永久性延髓无力，因此治疗效果不佳。大剂量泼尼松加血浆置换是治疗快速进展性球无力的推荐方法。在疾病管理方面，口服类固醇被证明是有效的，血浆置换产生了显著的改善，尽管是短期的，而传统的免疫抑制剂相对而言效果较差。利妥昔单抗是治疗难治性MuSK-MG的一种有希望的治疗方法;在非对照研究中，几乎所有接受治疗的患者都取得了显着改善，同时大幅减少了药物治疗。目前尚不清楚早期使用利妥昔单抗是否可以预防永久性延髓无力，这构成了这些患者的相关残疾。
Myasthenia gravis with antibodies to muscle-specific tyrosine kinase (MuSK-MG) is generally considered a severe disease because of the associated weakness distribution with prevalent involvement of bulbar muscles and a rapidly progressive course and early respiratory crises. Its treatment can be unrewarding, owing to poor response to acetylcholinesterase inhibitors in most patients, disease relapses in spite of high-dose immunosuppression, and development of permanent bulbar weakness. High-dose prednisone plus plasma exchange is the recommended approach for treating rapidly progressive bulbar weakness. In the disease management, oral steroids proved effective, plasma exchange produced marked, albeit short-term, improvement, while conventional immunosuppressants were comparatively less effective. Rituximab is a promising treatment for refractory MuSK-MG; in uncontrolled studies, nearly all treated patients achieved significant improvement with substantial decrease of medication. It is yet to be clarified whether the early use of rituximab could prevent the permanent bulbar weakness, which constitutes a relevant disability in these patients.