[Pubmed] The Clinical Efficacy and Safety of Efgartigimod in the Treatment of Autoimmune Encephalitis

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Immunol Invest. 2026 May 20:1-15. doi: 10.1080/08820139.2026.2665257. Online ahead of print.

ABSTRACT

OBJECTIVE: Efgartigimod, an innovative human IgG Fc receptor antagonist, clears pathogenic IgG autoantibodies. However, its efficacy and safety in autoimmune encephalitis (AE) remain undefined.

METHODS: We retrospectively analyzed the modified Rankin Scale (mRS) scores, Clinical Assessment Scale for Autoimmune Encephalitis (CASE) scores, immunological parameters, and routine clinical laboratory data from AE patients treated with efgartigimod.

RESULTS: The study included 15 AE patients who received efgartigimod. After the initial 4-week treatment period, a clinically meaningful improvement was observed in mRS scores [median: 4 (IQR 3, 5) vs. 2 (IQR 1, 2)]and CASE scores [median: 13 (IQR 7, 16) vs. 2 (IQR 1, 3)] (p < .001). Subgroup analyses further suggested a trend of clinical improvement in AE patients with neuronal surface autoantibodies (NSAab) (p < .01). Alongside clinical improvement, serum IgG levels decreased after the first infusion and continued to decline with subsequent treatments (p < .05). No serious adverse events were reported. Other immune markers and clinical laboratory parameters showed no observable changes (p > .05).

CONCLUSIONS: In this small retrospective cohort, efgartigimod was associated with clinical improvement and selectively reduced IgG levels with an acceptable safety profile. However, no causal relationship was established between IgG reduction and clinical response. These findings need further validation.

PMID:42159021 | DOI:10.1080/08820139.2026.2665257


Source: https://pubmed.ncbi.nlm.nih.gov/4215902 ... 0&v=2.20.0