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Emerging Therapies for Autoimmune Myasthenia Gravis: towards treatment without corticosteroids.
Neuromuscular Disorders ( IF 2.7 ) Pub Date : 2020-02-01 , DOI: 10.1016/j.nmd.2019.12.003
Martijn R Tannemaat 1 , Jan J G M Verschuuren 1
Affiliation  

Myasthenia gravis is an autoimmune disease characterized by dysfunction of the neuromuscular junction. Current treatment is based on lifestyle advice, symptomatic treatment, immunosuppressive drugs and thymectomy. Corticosteroids remain the cornerstone of treatment beside symptomatic medication due to their low cost, wide availability and fast mode of action. However, long term steroid use carries substantial risks of severe adverse side effects. Therefore, non-steroidal immunosuppressive drugs are commonly added to the treatment. Unfortunately, they have a delayed-onset effect and evidence of their efficacy appears to be difficult to obtain. Several trials using drugs that have had clear positive results in other immunological disorders have failed in myasthenia. This failure may in part be related to difficulties in the design of clinical trial for myasthenia, which has a fluctuating disease course involving weakness that may be difficult to assess quantitively. This problem is exacerbated by the tendency of most clinical trials to select patients with a stable, but severe disease. Future trials should: select patients with weakness and fatigability that is completely explained by their myasthenia gravis, use a design that avoids the exclusion of patients with recent changes in medication, and explore the possibilities to completely avoid the use of corticosteroids.

中文翻译:

自身免疫性重症肌无力的新兴疗法:无皮质类固醇治疗。

重症肌无力是一种以神经肌肉接头功能障碍为特征的自身免疫性疾病。目前的治疗基于生活方式建议、对症治疗、免疫抑制药物和胸腺切除术。皮质类固醇仍然是除对症药物之外的治疗基石,因为它们成本低、可得性广泛和起效快。然而,长期使用类固醇会带来严重不良副作用的巨大风险。因此,通常在治疗中加入非甾体免疫抑制药物。不幸的是,它们具有延迟作用,并且似乎难以获得其功效的证据。使用在其他免疫系统疾病中具有明显阳性结果的药物的几项试验在肌无力中失败了。这种失败可能部分与肌无力临床试验设计的困难有关,肌无力的病程波动较大,可能难以定量评估。大多数临床试验倾向于选择病情稳定但病情严重的患者,从而加剧了这个问题。未来的试验应该:选择完全由重症肌无力解释的虚弱和易疲劳的患者,使用避免排除最近药物改变的患者的设计,并探索完全避免使用皮质类固醇的可能性。
更新日期:2020-02-01
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