重症肌无力的诊断与治疗。
Diagnosis and treatment of myasthenia gravis.
机构信息
Neurology IV, Neuroimmunology and Neuromuscular Diseases Unit, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, Milan, Italy.
出版信息
Curr Opin Rheumatol. 2019 Nov;31(6):623-633. doi: 10.1097/BOR.0000000000000647.
PURPOSE OF REVIEW
This article provides an update on the most recent advances in diagnostic procedures and therapeutic approaches for myasthenia gravis, spanning from autoantibody and neuroelectrophysiological tests as diagnostic tools, to innovative and promising treatments based on biological drugs.
RECENT FINDINGS
Novel studies performed by cell-based assays (CBAs) indicate an improvement in the chance of identifying serum autoantibodies in myasthenic patients. Clinical trials on the use of biological drugs were recently concluded, providing important data on safety and efficacy of eculizumab, efgartigimod and amifampridine phosphate: the first, a complement blocker, showed long-term safety and efficacy in acetylcholine receptor (AChR)-positive myasthenic patients with refractory generalized disease; the second, the neonatal Fc receptor blocker, was well tolerated and clinically effective in both AChR-specific and muscle-specific kinase receptor (MuSK)-positive patients; the third, a blocker of presynaptic potassium channels, was found to be well tolerated and effective in MuSK-positive patients.
SUMMARY
CBAs can lead to a significant reduction of seronegative patients, improving myasthenia gravis diagnostic process. New biological drugs offer innovative approaches to treat myasthenic patients with generalized disease, promising to change the paradigm of treatment and to significantly enhance therapeutic success within a precision medicine framework.
目的综述
本文就重症肌无力的最新诊断程序和治疗方法进展进行综述,从用于诊断的自身抗体和神经电生理检测,到基于生物药物的创新且有前途的治疗方法。
最近的发现
通过基于细胞的检测(CBAs)进行的新研究表明,提高了在肌无力患者中识别血清自身抗体的可能性。最近完成了生物药物的临床试验,提供了关于依库珠单抗、efgartimod 和磷氨霉素的安全性和疗效的重要数据:第一种是补体抑制剂,在难治性全身型疾病的乙酰胆碱受体(AChR)阳性肌无力患者中显示出长期安全性和疗效;第二种是新生儿 Fc 受体抑制剂,在 AChR 特异性和肌肉特异性激酶受体(MuSK)阳性患者中具有良好的耐受性和临床疗效;第三种是一种抑制突触前钾通道的药物,在 MuSK 阳性患者中具有良好的耐受性和疗效。
总结
CBAs 可显著减少血清阴性患者,改善重症肌无力的诊断过程。新型生物药物为治疗全身性疾病的肌无力患者提供了创新方法,有望改变治疗模式,并在精准医学框架内显著提高治疗成功率。