20例儿童抗N-甲基-D-门冬氨酸受体脑炎的前瞻性研究

目的 探讨儿童抗N-甲基-D-门冬氨酸(anti-N-methyl-D-aspartate receptor encephalitis,NMDA)受体脑炎的临床特点、最佳治疗方案和预后.方法 对20例抗NMDA受体脑炎的患者进行临床队列观察,总结其临床表现、对治疗的反应和临床预后特点.结果 入组20例患儿中,男患儿8例,女患儿12例,年龄1岁11个月～13岁4个月.观察时间8个月～3年6个月,至病情稳定,6个月内无改善/进展.患者均以神经系统症状为首发表现,其中以癫癇发作最为突出,90％的患者均有癫癇发作;其次为语言障碍、认知行为异常和运动异常.偏瘫和小脑共济失调在6岁以下患者中出现频率较高.50％患者一线免疫治疗(IVIG、皮质激素)有效,一线治疗失败者予二线免疫(利妥昔单抗)治疗后,80％患儿有明显好转.部分患儿恢复缓慢,在8 ～12月的治疗中逐渐好转.结论 抗NMDA受体脑炎在儿童脑炎患者中并不少见,免疫治疗有效.一线免疫治疗失败后应给予二线免疫治疗.

Prospective study of 20 children with anti-NMDA receptor encephalitis

Objective To assess the presentation of Anti-NMDA receptor (NMDAR)encephalitis,the spectrum of symptoms,immunotherapies used,timing of improvement,and long-term outcome.Methods In this observational study,we tested the presence of NMDAR antibodies in serum or CSF samples of patients with encephalitis from Dec 1,2011,to Dec 1,2014.All patients who tested positive for NMDAR antibodies were included in the study;patients were assessed at symptom onset and at months 4,8,12,18,24,and 36 by use of the modified Rankin scale(mRS).Treatment included first-line immunotherapy(steroids,intravenous immunoglobulin),second-line immunotherapy(rituximab).Results We enrolled 20 patients(range 1 year 11 months to 13 years 4 months),observation time range 8 months to 3 years 6 months.All Patients presented with acute neurology symptoms:90％ with seizures.Speech problems,memory deficit,dyskinesias are quite popular in these children.Cerebellar ataxia and hemiparesis were not uncommon in children younger than 6 years.Twenty patients underwent first-line immunotherapy,resulting in improvement within 4 weeks in 10(50％).10 patients who did not improve with first-line treatment received second-line immunotherapy,8 of them resulted in a better outcome.Outcomes may continued to improve for up to 8 months to 1 year after symptom onset.Condusion Anti-NMDAR encephalitis are not rare in children.Most patients with anti-NMDAR encephalitis respond to immunotherapy.Second-line immunotherapy is usually effective when first-line treatments fail.