Takahashi Yukitoshi, Yamazaki Etsuko, Mine Jun, Kubota Yuko, Imai Katsumi, Mogami Yuki, Baba Koichi, Matsuda Kazumi, Oguni Hirokazu, Sugai Kenji, Ohtsuka Yoko, Fujiwara Tateki, Inoue Yushi
National Epilepsy Center, Shizuoka Institute of Epilepsy and Neurological Disorder, Shizuoka, Japan.
Brain Dev. 2013 Sep;35(8):778-85. doi: 10.1016/j.braindev.2013.01.010. Epub 2013 Feb 19.
We examined seizure, cognitive, and motor outcomes in patients with Rasmussen syndrome or Rasmussen encephalitis (RS), after recent initiation of immunomodulatory therapies. Among 53 patients with a diagnosis of RS referred from all over Japan, 49 patients (male 22, female 27) with symptoms and findings characteristic of RS were evaluated. Regular intravenous immunoglobulin (IVIg) therapy was administered at a dose of 100mg/kg/day, etc. Regular steroid pulse therapy was conducted with methylprednisolone at a dose of 30mg/kg/day (children) or 1000mg/day (adults) for 3days. Tacrolimus was given at an initial dose of 0.1mg/kg/day (children). Mean onset age was 8.7±10.5years. Seizure-free rate was 71% after treatment by functional hemispherectomy (FH), and response rate for seizures was 81% by regular steroid pulse therapy, 42% by tacrolimus therapy, and 23% by regular IVIg therapy. Rate of patients with IQ higher than 80 (R80) was 50% by regular steroid pulse therapy, 43% by regular IVIg therapy, 29% by tacrolimus therapy, and 0% by FH. R80 after regular steroid pulse therapy was 100% in patients without MRI lesions, and 37% in those with advanced MRI lesions. Improvement of motor function (paresis) was observed only by immunomodulatory therapy. Motor function was aggravated in 100% of patients treated by FH, 62% by regular IVIg, and 10% by regular steroid pulse therapy. We suggest a new treatment strategy for RS using early immunomodulatory therapy: initiation of regular steroid pulse therapy after early diagnosis indicated by biomarkers, then switching to tacrolimus therapy after several months.
我们研究了近期开始免疫调节治疗的拉斯穆森综合征或拉斯穆森脑炎（RS）患者的癫痫发作、认知和运动结局。在从日本各地转诊的53例诊断为RS的患者中，对49例（男性22例，女性27例）具有RS特征性症状和表现的患者进行了评估。定期静脉注射免疫球蛋白（IVIg）治疗的剂量为100mg/kg/天等。采用甲泼尼龙进行定期类固醇冲击治疗，剂量为30mg/kg/天（儿童）或1000mg/天（成人），持续3天。他克莫司的初始剂量为0.1mg/kg/天（儿童）。平均发病年龄为8.7±10.5岁。功能性半球切除术（FH）治疗后无癫痫发作率为71%，常规类固醇冲击治疗的癫痫发作缓解率为81%，他克莫司治疗为42%，常规IVIg治疗为23%。智商高于80（R80）的患者比例，常规类固醇冲击治疗为50%，常规IVIg治疗为43%，他克莫司治疗为29%，FH治疗为0%。常规类固醇冲击治疗后，无MRI病变的患者R80为100%，有晚期MRI病变的患者为37%。仅免疫调节治疗观察到运动功能（轻瘫）改善。FH治疗的患者中100%运动功能加重，常规IVIg治疗的患者中62%，常规类固醇冲击治疗的患者中10%。我们建议采用早期免疫调节治疗的RS新治疗策略：在生物标志物提示早期诊断后开始常规类固醇冲击治疗，数月后改用他克莫司治疗。
