What question did this study set out to answer?

The study aims to assess the efficacy and safety of efgartigimod in patients with Guillain–Barré syndrome.

February 12, 2026Open Access

Efgartigimod for Guillain–Barré syndrome: a retrospective analysis of efficacy and safety

ZBZhuajin Bi XYXiaodong YeSinopec (China)PLPeidong LiuFirst Affiliated Hospital of Zhengzhou University

Puntos clave

The study aims to assess the efficacy and safety of efgartigimod in patients with Guillain–Barré syndrome.
Conducted a retrospective, observational, monocentric analysis.
Included 36 patients with Guillain–Barré syndrome treated with efgartigimod.
Measured efficacy using GBS-Disability Scale and MRC scores over an 8-week period.
Performed exploratory lymphocyte subset analysis on 13 patients before and after treatment.
Both patient cohorts showed significant clinical improvement over 8 weeks (p < 0.05).
The first-line cohort had a greater reduction in GBS-DS score (ΔGBS-DS: 2.0 vs 0.0 at week 1, p = 0.003) and higher MRC score increase (ΔMRC: 9.0 vs 6.0, p = 0.016).
First-line therapy cohort achieved favorable outcomes more quickly than the escalation therapy cohort, with median times of 6 vs 25 days (p < 0.001).
Exploratory analysis indicated a decrease in specific T cell populations post-treatment.
Only two mild adverse events were reported, suggesting a favorable safety profile.

Resumen

Background: Guillain–Barré syndrome (GBS) is an acute autoimmune neuropathy with limited effective treatments. Efgartigimod, a neonatal Fc receptor (FcRn) antagonist, may offer a novel treatment by reducing pathogenic immunoglobulin G. Objectives: To evaluate the efficacy, safety, and immunological effects of efgartigimod in patients with GBS. Design: A retrospective, observational, monocentric study. Methods: The study included 36 patients with GBS who received efgartigimod treatment. Efficacy was evaluated using the GBS-Disability Scale (GBS-DS) and Medical Research Council (MRC) scores over 8 weeks. Additionally, exploratory lymphocyte subset analysis was conducted on 13 patients before and after treatment. Results: Patients were stratified into those receiving efgartigimod as first-line therapy ( n = 17) and as escalation therapy after intravenous immunoglobulin or plasma exchange ( n = 19). Both cohorts showed significant and sustained clinical improvement over 8 weeks ( p < 0.05). More importantly, the first-line cohort demonstrated a more rapid initial response, with a significantly greater reduction in GBS-DS score (ΔGBS-DS: 2.0 vs 0.0 at week 1, p = 0.003) and increase in MRC score (ΔMRC: 9.0 vs 6.0, p = 0.016) compared to the escalation cohort. This led to a higher percentage of achieving a favorable outcome (GBS-DS ⩽2) and a shorter median time to this outcome (6 vs 25 days, p < 0.001) in the first-line cohort. Regression analysis within the entire cohort confirmed that greater baseline severity and need for mechanical ventilation were associated with a longer time to favorable outcome. Exploratory analysis suggested a decrease in the proportions of CD3+CD4+ and CD4+HLA-DR+ T cells following efgartigimod treatment. Only two mild adverse events were reported. Conclusion: Efgartigimod shows efficacy as both a rapid-onset first-line therapy and a rescue option for refractory GBS, with a favorable safety profile and potential immunomodulatory effects, supporting further prospective evaluation.

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