Han Y, Peng J, He F, Zhang C L, Yang L F, Mao L L
Department of Pediatrics Neurology, XiangYa Hospital of Central South University, Changsha 410008, China.
Zhonghua Er Ke Za Zhi. 2024 Jun 2;62(6):559-564. doi: 10.3760/cma.j.cn112140-20231009-00265.
To analyze the clinical features of children with refractory N-methyl-D-aspartate (NMDA) receptor antibody encephalitis treated with tocilizumab. 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. 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 (=-2.56, =0.014). All children had no serious adverse reactions during the treatment. 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.
分析托珠单抗治疗难治性N-甲基-D-天冬氨酸（NMDA）受体抗体脑炎患儿的临床特征。回顾性收集2021年8月至2023年9月在中南大学湘雅医院儿科神经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统计学和临床表现、免疫治疗及预后数据。在初始诊断时、开始托珠单抗治疗时及末次随访时，使用改良Rankin量表评估预后。分析治疗相关并发症、神经影像学和脑电图数据。9例患儿中，男6例，女3例，发病年龄为4.2（2.8，8.7）岁。疾病发作时，9例患儿改良Rankin量表评分为5分。开始托珠单抗治疗时，7例患儿评分为5分，2例患儿评分为4分。从发病到开始托珠单抗治疗的间隔时间为12（5，27）个月，治疗次数为8（5，13）次。随访时间为2.8（1.5，3.7）年。末次随访时，9例患儿的运动障碍、睡眠障碍、意识障碍、缄默和自主神经功能障碍等症状均有不同程度改善，且均无癫痫发作。末次随访时，改良Rankin量表评分为0分者4例，1分者1例，3分者2例，4分者1例，5分者1例。末次随访时的改良Rankin量表评分与开始托珠单抗治疗时相比有显著差异（=-2.56，=0.014）。所有患儿在治疗期间均无严重不良反应。托珠单抗治疗后，难治性NMDA受体抗体脑炎患儿的运动障碍、睡眠障碍、意识障碍、缄默和自主神经功能障碍等症状得到改善，且均无癫痫发作。改良Rankin量表评分得到改善，安全性良好。
