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髓鞘少突胶质细胞糖蛋白抗体相关疾病:难治性和严重形式的病理生理学、临床模式及治疗挑战

Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease: Pathophysiology, Clinical Patterns, and Therapeutic Challenges of Intractable and Severe Forms.

作者信息

Misu Tatsuro

机构信息

Department of Neurology, Tohoku University Hospital, 1-1 Seiryomachi, Aobaku, Sendai 980-8574, Japan.

出版信息

Int J Mol Sci. 2025 Sep 2;26(17):8538. doi: 10.3390/ijms26178538.

Abstract

Myelin oligodendrocyte glycoprotein (MOG) antibody-associated disease (MOGAD) is characterized by the predominance of optic neuritis, myelitis, acute disseminated encephalomyelitis (ADEM), and cortical encephalitis, and can be diagnosed by the presence of pathogenic immunoglobulin G (IgG) antibodies targeting the extracellular domain of MOG in the serum and cerebrospinal fluid (CSF). Initially considered a variant of multiple sclerosis (MS) or neuromyelitis optica spectrum disorder (NMOSD), it is now widely recognized as a separate entity, supported by converging evidence from serological, pathological, and clinical studies. Patients with MOGAD often exhibit better recovery from acute attacks; however, their clinical and pathological features vary based on the immunological role of MOG-IgG via antibody- or complement-mediated perivenous demyelinating pathology, in addition to MOG-specific cellular immunity, resulting in heterogeneous demyelinated lesions from vanishing benign forms to tissue necrosis, even though MOGAD is not a mild disease. The key is the immunological mechanism of devastating lesion coalescence and long-term degenerating mechanisms, which may still accrue, particularly in the relapsing, progressing, and aggressive clinical course of encephalomyelitis. The warning features of the severe clinical forms are: (1) fulminant acute multifocal lesions or multiphasic ADEM transitioning to diffuse (Schilder-type) or tumefactive lesions; (2) cortical or subcortical lesions related to brain atrophy and/or refractory epilepsy (Rasmussen-type); (3) longitudinally extended spinal cord lesions severely affected with residual symptoms. In addition, it is cautious for patients refractory to acute stage early 1st treatment including intravenous methylprednisolone treatment and apheresis with residual symptoms and relapse activity with immunoglobulin and other 2nd line treatments including B cell depletion therapy. Persistent MOG-IgG high titration, intrathecal production of MOG-IgG, and suggestive markers of higher disease activity, such as cerebrospinal fluid interleukin-6 and complement C5b-9, could be identified as promising markers of higher disease activity, worsening of disability, and poor prognosis, and used to identify signs of escalating treatment strategies. It is promising of currently ongoing investigational antibodies against anti-interleukin-6 receptor and the neonatal Fc receptor. Moreover, due to possible refractory issues such as the intrathecal production of autoantibody and the involvement of complement in the worsening of the lesion, further developments of other mechanisms of action such as chimeric antigen receptor T-cell (CAR-T) and anti-complement therapies are warranted in the future.

摘要

髓鞘少突胶质细胞糖蛋白(MOG)抗体相关疾病(MOGAD)的特征是视神经炎、脊髓炎、急性播散性脑脊髓炎(ADEM)和皮质脑炎占主导,可通过血清和脑脊液(CSF)中存在靶向MOG细胞外结构域的致病性免疫球蛋白G(IgG)抗体来诊断。MOGAD最初被认为是多发性硬化症(MS)或视神经脊髓炎谱系障碍(NMOSD)的一种变体,现在已被广泛认可为一个独立的疾病实体,血清学、病理学和临床研究的一致证据支持这一点。MOGAD患者急性发作后通常恢复较好;然而,他们的临床和病理特征因MOG-IgG通过抗体或补体介导的静脉周围脱髓鞘病理作用以及MOG特异性细胞免疫的免疫作用而异,导致脱髓鞘病变从消失的良性形式到组织坏死不等,尽管MOGAD并非轻症疾病。关键在于破坏性病变融合和长期退变机制的免疫机制,这些机制可能仍会出现,尤其是在脑脊髓炎的复发、进展和侵袭性临床病程中。严重临床形式的警示特征包括:(1)暴发性急性多灶性病变或多相性ADEM转变为弥漫性(希尔德型)或瘤样病变;(2)与脑萎缩和/或难治性癫痫(拉斯穆森型)相关的皮质或皮质下病变;(3)严重受累且有残留症状的纵向延伸脊髓病变。此外,对于急性期早期包括静脉注射甲泼尼龙治疗和血浆置换在内的首次治疗难治且有残留症状以及对包括B细胞清除疗法在内的免疫球蛋白和其他二线治疗有复发活动的患者要谨慎。持续的MOG-IgG高滴度、MOG-IgG的鞘内产生以及较高疾病活动度的提示性标志物,如脑脊液白细胞介素-6和补体C5b-9,可被确定为较高疾病活动度、残疾恶化和预后不良的有前景标志物,并用于确定治疗策略升级的迹象。目前正在研究的抗白细胞介素-6受体和新生儿Fc受体抗体很有前景。此外,由于可能存在自身抗体鞘内产生和补体参与病变恶化等难治性问题,未来有必要进一步开发其他作用机制,如嵌合抗原受体T细胞(CAR-T)和抗补体疗法。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/eaee/12429651/c488712bebf3/ijms-26-08538-g001.jpg

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