Objective: To analyze the clinical features of children with refractory N-methyl-D-aspartate (NMDA) receptor antibody encephalitis treated with tocilizumab. Methods: Demographic and clinical manifeatations, immunotherapy and prognosis data of 9 children with refractory NMDA receptor antibody encephalitis who received tocilizumab in the Department of Pediatrics Neurology, XiangYa Hospital of Central South University from August 2021 to September 2023 were collected retrospectively. Prognosis was evaluated using the modified Rankin scale at initial diagnosis, at the initiation of tocilizumab treatment, and at the last follow-up. Treatment related complications, neuroimaging, and electroencephalography data were analyzed. Results: Among the 9 children, 6 were male and 3 were female, with an onset age of 4.2 (2.8, 8.7) years. At the onset of the disease, 9 children had a modified Rankin scale score of 5. When tocilizumab treatment was initiated, 7 children had a score of 5, and 2 children had a score of 4. The interval between the onset and initiation of tocilizumab treatment was 12 (5, 27) months, and the treatment frequency was 8 (5, 13) times. The follow-up time was 2.8 (1.5, 3.7) years. At the last follow-up, the symptoms of 9 children, including movement disorder, sleep disorder, consciousness disorder, silence and autonomic dysfunction, were improved to varying degrees, and none of them had seizures. At the last follow-up, 4 cases with a modified Rankin scale score of 0, 1 case with a score of 1, 2 cases with a score of 3, 1 case with a score of 4 and 1 case with a score of 5. The modified Rankin scale at the last follow-up was significantly different from that at the start of tocilizumab (Z=-2.56, P=0.014). All children had no serious adverse reactions during the treatment. Conclusions: After treatment with tocilizumab, the symptoms in patients with refractory NMDA receptor antibody encephalitis, including movement disorder, sleep disorder, consciousness disorder, silence and autonomic dysfunction were improved, and none of them had seizures. The modified Rankin scale were improved, and the safety was good.
目的： 分析使用托珠单抗治疗的难治性N-甲基-D-天冬氨酸（NMDA）受体抗体脑炎患儿的临床特征。 方法： 回顾性分析2021年8月至2023年9月中南大学湘雅医院儿科接受托珠单抗治疗的9例难治性NMDA受体抗体脑炎患儿的人口学数据、临床表现、辅助检查、免疫治疗和预后资料。采用改良Rankin评分进行预后评估，分析治疗相关并发症及神经影像学和脑电图等资料。 结果： 9例患儿中男6例、女3例，起病年龄4.2（2.8，8.7）岁。起病时改良Rankin评分均为5分，开始托珠单抗治疗时改良Rankin评分5分7例，4分2例。起病与开始托珠单抗治疗间隔12（5，27）个月，治疗次数8（5，13）次。随访时间2.8（1.5，3.7）年。末次随访时9例患儿运动障碍、睡眠障碍、意识障碍、语言缄默、自主神经功能紊乱等临床症状均不同程度好转，且均无癫痫发作。末次随访时改良Rankin评分0分4例，1分1例，3分2例，4分1例，5分1例，末次随访时改良Rankin评分较开始托珠单抗治疗时，差异有统计学意义（Z=-2.56，P=0.014）。治疗过程中9例患儿均未见严重不良反应。 结论： 儿童难治性NMDA受体抗体脑炎使用托珠单抗治疗后运动障碍、睡眠障碍、意识障碍、语言缄默、自主神经功能紊乱等症状均不同程度好转，且均无癫痫发作，改良Rankin评分改善，治疗过程中安全性良好。.