全身性肌无力的新治疗方案

Antanas Vaitkus, J. Čiauškaitė, M. Malakauskaitė, M. Baublytė
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引用次数: 0

摘要

重症肌无力是一种自身免疫性疾病,患者体内针对神经肌肉接头突触后膜蛋白的自身抗体会破坏神经冲动的传递,从而引起病理性肌肉无力和疲劳,并在一天中不断加重。虽然这种疾病尚无法治愈,但大多数患者通过适当的治疗可以完全控制症状,改善生活质量。临床上采用四种治疗策略:对症治疗、免疫抑制治疗、免疫调节治疗和手术治疗,这四种治疗方法有助于控制病情,但并非对所有患者都同样有效。使用乙酰胆碱酯酶(AChE)抑制剂进行对症治疗往往不够有效,因此需要额外使用免疫抑制剂进行治疗。这些药物虽然有效,但如果长期服用,会引起全身副作用。即使是多种疗法,也往往不足以治疗重症肌无力患者。治疗这种疾病所面临的挑战促使人们寻求替代疗法。针对乙酰胆碱受体(AChRs)和神经肌肉接头处其他结构的抗体正受到越来越多的关注,这些抗体对重症肌无力的发病机制非常重要。目前正在开发针对免疫系统特定环节的药物,以降低全身不良反应的风险。目前,只有两种药物被批准用于治疗全身性肌萎缩症--依库珠单抗和依加替莫德。这两种药物都能安全有效地治疗普遍存在抗ACHR抗体的全身性肌无力症。目前,还有10种药物正在接受临床试验,以确定其治疗重症肌无力患者的安全性和有效性。在本文中,我们将回顾分析生物疗法及其在重症肌无力治疗中的新颖性的出版物。我们更关注已获批准的生物药物。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
New treatment options for generalized myasthenia gravis
Myasthenia gravis is an autoimmune disease in which autoantibodies against the postsynaptic membrane proteins of the neuromuscular junction disrupt impulse transmission thus causing pathological muscle weakness and fatigue that worsens throughout the day. Although the disease is not yet curable, most patients can achieve complete symptom control and improved quality of life with appropriate treatment. Four treatment strategies are used in clinical practice: symptomatic, immunosuppressive, immunomodulatory, and surgical treatment, which can help control the disease but are not equally effective for all patients. Symptomatic treatment with acetylcholinesterase (AChE) inhibitors is often not effective enough, so additional treatment with immunosuppressants is indicated. These are effective, but can cause systemic side effects if taken for long periods. Even polytherapy is often not sufficient enough to treat patients with myasthenia gravis. The challenges of treating this disease are encouraging to seek alternatives. Increasing attention is being paid to antibodies against acetylcholine receptors (AChRs) and other structures of the neuromuscular junction that are important in pathogenesis of myasthenia gravis. Drugs are being developed that target specific links in the immune system to reduce the risk of systemic adverse effects. Currently, only two drugs are approved for the treatment of generalized myasthenia gravis – eculizumab and efgartigimod. Both of them are safe and effective in treating generalized myasthenia gravis with prevalent anti-AChR antibodies. Currently, 10 other drugs are clinically tested for their safety and efficacy in treating patients with myasthenia gravis. In this article, we review publications that analyze biological therapy and its novelty in the treatment of myasthenia gravis. We focus more on already approved biological drugs.
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