Bi Zhuajin, Kang Wenzhong, Liu Ruihan, Jiang Yan, Chen Min
Department of Neurology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, China.
Xiangya School of Medicine, Central South University, Changsha, Hunan, China.
Eur J Neurol. 2025 Aug;32(8):e70280. doi: 10.1111/ene.70280.
Autoimmune encephalitis (AE) is a severe neurological disorder that requires effective and safe treatment options. This study aimed to compare the efficacy and safety of intravenous methylprednisolone (IVMP) plus efgartigimod (IPE) versus IVMP alone or IVMP plus immunoglobulin (IPI) as initial treatments for antibody-mediated AE.
A retrospective, single-center, cross-sectional study was conducted to compare treatment responses between the IPE group and the IVMP or IPI groups at baseline and after 2, 4, 8, and 12 weeks. Responses were assessed using the Clinical Assessment Scale (CASE) and modified Rankin Scale (mRS) scores, with propensity score matching (PSM) applied to adjust for confounding variables.
A total of 122 patients with surface antibody-mediated AE were included. After 1:1 PSM, 16 pairs (IPE vs. IVMP) and 15 pairs (IPE vs. IPI) were matched. At 2 weeks post-treatment, the IPE group demonstrated significantly greater improvements in CASE scores from baseline (ΔCASE: 6.5 [IQR = 3.3-8.8] vs. 3.0 [0.0-6.0]) and mRS scores from baseline (ΔmRS: 1.5 [1.0-2.0] vs. 1.0 [0.0-1.0]) compared to the IVMP group, along with a higher proportion of favorable outcomes (mRS ≤ 2: 75.0% vs. 20.0%). No significant differences in CASE or mRS score changes were observed between the IPE and IPI groups over the 12-week follow-up period. Only one mild case of facial rash was reported during efgartigimod treatment, which resolved promptly.
Efgartigimod, when added to IVMP treatment, demonstrates rapid efficacy and favorable safety in antibody-positive AE. These findings suggest the need for further investigation in larger clinical trials.
自身免疫性脑炎（AE）是一种严重的神经系统疾病，需要有效且安全的治疗方案。本研究旨在比较静脉注射甲泼尼龙（IVMP）联合艾加莫德（IPE）与单纯IVMP或IVMP联合免疫球蛋白（IPI）作为抗体介导的AE初始治疗的疗效和安全性。
进行了一项回顾性、单中心横断面研究，以比较IPE组与IVMP组或IPI组在基线以及2、4、8和12周后的治疗反应。使用临床评估量表（CASE）和改良Rankin量表（mRS）评分评估反应，并应用倾向评分匹配（PSM）来调整混杂变量。
共纳入122例表面抗体介导的AE患者。经过1:1的PSM后，匹配了16对（IPE组与IVMP组）和15对（IPE组与IPI组）。治疗后2周，与IVMP组相比，IPE组的CASE评分较基线有显著更大改善（ΔCASE：6.5[四分位间距=3.3-8.8]对3.0[0.0-6.0]），mRS评分较基线也有显著更大改善（ΔmRS：1.5[1.0-2.0]对1.0[0.0-1.0]），同时良好结局的比例更高（mRS≤2：75.0%对20.0%）。在12周的随访期内，IPE组和IPI组之间在CASE或mRS评分变化方面未观察到显著差异。在艾加莫德治疗期间仅报告了1例轻度面部皮疹病例，皮疹迅速消退。
艾加莫德添加到IVMP治疗中，在抗体阳性AE中显示出快速疗效和良好安全性。这些发现表明需要在更大规模的临床试验中进行进一步研究。
