Chatterton Sophie, Parratt John Douglas Edward, Ng Karl
Department of Neurology, Royal North Shore Hospital, Sydney, NSW, Australia.
School of Medicine, University of Sydney, Sydney, NSW, Australia.
Front Neurol. 2022 Aug 8;13:951423. doi: 10.3389/fneur.2022.951423. eCollection 2022.
Eculizumab has been shown to be an effective and typically well-tolerated medication in the treatment of neuromyelitis optica spectrum disorder (NMOSD) in maintaining disease remission in patients who are aquaporin-4 water channel autoantibody (AQP4-IgG) seropositive. The efficacy of eculizumab in an acute relapse of NMOSD however is still under review.
We describe a 46 year-old female who presented with acute left monocular vision loss on a background of bilateral optic neuritis treated 15 years prior as suspected NMOSD. She had very poor vision from the right eye (6/60). On presentation she was not on any long-term immunosuppressive agents. Her serum was positive for AQP4-IgG and MRI brain and spine demonstrated areas of demyelination in the corpus callosum and thoracic spine. She was treated with high dose intravenous methylprednisolone and underwent plasmapheresis for five consecutive days, but continued to clinically deteriorate with ongoing blindness in her left eye (light perception only). She was subsequently administered eculizumab with weaning oral corticosteroids. Clinically her vision improved to counting fingers and she remains on maintenance eculizumab infusions in the community. At 3 months, there is a steady improvement but still significant loss of central vision from that eye.
The utility of eculizumab in NMOSD may assist with treating acute episodes. This theoretically accords with the mode of action in inhibiting conversion of C5-C5a/b, perhaps arresting the acute inflammatory process in this disease. Given that disease burden and mortality in NMOSD is almost entirely related to relapses, increased use of eculizumab acutely could potentially aid recovery from an attack in very severe attacks, and therefore minimize immediate stepwise accrual of disability.
在治疗视神经脊髓炎谱系障碍（NMOSD）方面，依库珠单抗已被证明是一种有效且通常耐受性良好的药物，可使水通道蛋白4水通道自身抗体（AQP4-IgG）血清阳性患者维持疾病缓解状态。然而，依库珠单抗在NMOSD急性复发中的疗效仍在研究中。
我们描述了一名46岁女性，她在15年前因疑似NMOSD接受治疗的双侧视神经炎背景下，出现急性左眼视力丧失。她右眼视力极差（6/60）。就诊时，她未服用任何长期免疫抑制剂。她的血清AQP4-IgG呈阳性，脑部和脊柱MRI显示胼胝体和胸椎有脱髓鞘区域。她接受了大剂量静脉注射甲基强的松龙治疗，并连续五天进行了血浆置换，但临床症状仍持续恶化，左眼持续失明（仅存光感）。随后她接受了依库珠单抗治疗，并逐渐停用口服皮质类固醇。临床上，她的视力改善到能数手指，目前仍在社区接受依库珠单抗维持输注治疗。3个月时，视力有稳步改善，但该眼中心视力仍有明显丧失。
依库珠单抗在NMOSD中的应用可能有助于治疗急性发作。从理论上讲，这与抑制C5-C5a/b转化的作用模式相符，可能会阻止该疾病的急性炎症过程。鉴于NMOSD的疾病负担和死亡率几乎完全与复发相关，在急性发作时增加依库珠单抗的使用可能有助于在非常严重的发作中从发作中恢复，从而尽量减少残疾的立即逐步累积。
