Xu Xintong, Wang Yuhang, Sun Mingming, Li Yuyu, Chen Biyue, Chen Xiyun, Xu Quangang, Wei Shihui, Zhou Huanfen
Medical School of Chinese PLA, Beijing, China.
Department of Ophthalmology, The Third Medical Center of Chinese PLA General Hospital, Beijing, China.
Ther Adv Neurol Disord. 2024 Dec 16;17:17562864241306685. doi: 10.1177/17562864241306685. eCollection 2024.
Myelin oligodendrocyte glycoprotein (MOG) IgG related optic neuritis (ON) which manifests as recurrent episodes and severe visual impairment remains a challenging issue in relapse prevention. Tocilizumab (TCZ), a human monoclonal antibody against IL-6R, may be an alternative treatment for the prevention of relapse in refractory MOG-ON patients.
To investigate the efficacy and safety of Tocilizumab (TCZ) in patients with recurrent myelin oligodendrocyte glycoprotein IgG related optic neuritis (MOG-ON).
We conducted an open-label, single-arm, nonrandomized, uncontrolled clinical trial at a tertiary neuro-ophthalmology center between April 1, 2021, and April 1, 2022.
Participants with relapsed MOG-ON, whose disease had been resistant to previous immunotherapies, received tocilizumab as monotherapy or as an add-on therapy and were followed up for at least 12 months. Annual recurrence rate (ARR), best corrected visual acuity (BCVA), and adverse events were recorded for analyses.
Ten patients (7 females and 3 males) with relapsed MOG-ON were included with a mean (SD) ages of 28.6 (20.5) years old at disease onset and 30.9 (19.7) years at first TCZ administration, with a mean disease duration of 26.6 (11.3) months. Seven (70%) patients remained relapse-free, and the median (range) ARR dropped significantly from 1.9 (0.4-3.5) to 0.0 (0-4.0) during TCZ treatment ( = 0.006). Three patients experienced a relapse of ON at 2, 3, and 7 months after TCZ therapy. The median BCVA improved from 2.7 (2.0-3.0) logMAR at the nadir to 0.2 (0-2.0) logMAR at the last follow-up. Adverse effects included transient diarrhea ( = 1) and upper respiratory infection ( = 1).
This study supports that Tocilizumab therapy, with or without concomitant immunosuppression, is safe and effective in reducing relapses in MOG-ON patients who have failed immunosuppressive therapy or targeted B-cell therapy.
This trial is registered with the Chinese Clinical Trial Registry, number ChiCTR2100045273. (URL: https://www.chictr.org.cn/showproj.html?proj=124810).
髓鞘少突胶质细胞糖蛋白（MOG）IgG相关视神经炎（ON）表现为反复发作和严重视力损害，在预防复发方面仍然是一个具有挑战性的问题。托珠单抗（TCZ）是一种抗IL-6R的人单克隆抗体，可能是预防难治性MOG-ON患者复发的替代治疗方法。
探讨托珠单抗（TCZ）治疗复发性髓鞘少突胶质细胞糖蛋白IgG相关视神经炎（MOG-ON）患者的疗效和安全性。
我们于2021年4月1日至2022年4月1日在一家三级神经眼科中心进行了一项开放标签、单臂、非随机、无对照的临床试验。
复发的MOG-ON患者，其疾病对先前的免疫治疗耐药，接受托珠单抗作为单一疗法或附加疗法，并随访至少12个月。记录年复发率（ARR）、最佳矫正视力（BCVA）和不良事件进行分析。
纳入10例复发的MOG-ON患者（7例女性和3例男性），疾病发作时平均（标准差）年龄为28.6（20.5）岁，首次使用TCZ时平均年龄为30.9（19.7）岁，平均病程为26.6（11.3）个月。7例（70%）患者无复发，在TCZ治疗期间，ARR中位数（范围）从1.9（0.4-3.5）显著降至0.0（0-4.0）（P = 0.006）。3例患者在TCZ治疗后2、3和7个月出现ON复发。BCVA中位数从最低点时的2.7（2.0-3.0）logMAR提高到最后一次随访时的0.2（0-2.0）logMAR。不良反应包括短暂性腹泻（n = 1）和上呼吸道感染（n = 1）。
本研究支持托珠单抗治疗，无论是否联合免疫抑制，对于免疫抑制治疗或靶向B细胞治疗失败的MOG-ON患者，在减少复发方面是安全有效的。
本试验已在中国临床试验注册中心注册，注册号为ChiCTR2100045273。（网址：https://www.chictr.org.cn/showproj.html?proj=124810）
