Efgartigimod as a Fast-Acting Add-On Therapy in Antibody-Mediated Autoimmune Encephalitis

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.