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Old and new breakthroughs in neuromyelitis optica.
The Lancet Neurology ( IF 46.5 ) Pub Date : 2020-04-01 , DOI: 10.1016/s1474-4422(20)30062-4
Hans-Peter Hartung 1 , Orhan Aktas 1
Affiliation  

More than a decade ago, the identification of a disease-specific antibody targeting the aquaporin 4 (AQP4) water channel on astrocytes opened a new chapter in neuroimmunology: neuromyelitis optica, up to then classified as a rare clinical variant of multiple sclerosis, emerged as an independent entity in clinical practice. The AQP4 antibody transformed our understanding of the underlying disease mechanisms, of possible treatment targets, and disease phenomenology. The biomarker enabled better characterisation of patient cohorts and paved the way for the definition of neuromyelitis optica spectrum disorder (NMOSD), with the AQP4 antibody at the core of a new set of diagnostic criteria. The AQP4 antibody also facilitated clinical trials in NMOSD. Indeed, specific treatments for neuromyelitis optica were needed, because well established and highly efficient immunotherapies for multiple sclerosis were judged insufficient (if not deleterious) in the management of patients with neuromyelitis optica.

中文翻译:

视神经脊髓炎的新旧突破。

十多年前,针对星形胶质细胞上水通道蛋白4(AQP4)水通道的疾病特异性抗体的鉴定开启了神经免疫学的新篇章:视神经性脊髓炎,直到现在被归类为罕见的多发性硬化症的临床变异,临床实践中的独立实体。AQP4抗体改变了我们对潜在疾病机制,可能的治疗靶标和疾病现象学的理解。生物标志物使患者队列更好地表征,并为视神经脊髓炎光谱疾病(NMOSD)的定义铺平了道路,而AQP4抗体则是新的诊断标准的核心。AQP4抗体也促进了NMOSD的临床试验。实际上,需要针对视神经脊髓炎的具体治疗方法,
更新日期:2020-03-19
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