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Objective <p>This study aimed to compare the effectiveness of intravenous efgartigimod and intravenous immunoglobulin (IVIg) in patients with Guillain–Barré syndrome (GBS).</p> Methods <p>This dual-center, retrospective study analyzed prospectively collected data from adult patients with severe GBS who received either efgartigimod or IVIg. The primary outcome was the proportion of patients who achieving a GBS Disability Scale (GBS-DS) score ≤ 2 at 4 weeks post-treatment. Secondary outcomes included the proportion of patients achieving GBS-DS ≤ 2 at week 24; ≥ 1-grade improvement in GBS-DS at weeks 4 and 24; GBS-DS grade at week 4; and changes in GBS-DS, Medical Research Council (MRC) sum score, and other validated disability measures at weeks 1, 2, 4, 8, 16, and 24. Baseline serum levels of neurofilament light chain (NfL) and anti-GM1 antibodies, and their dynamic changes at 1 week post-treatment were assessed as exploratory outcomes.</p> Results <p>Twenty-one patients were enrolled (efgartigimod: <i>n</i> = 9; IVIg: <i>n</i> = 12). The primary outcome was not achieved (OR = 0.67, 95% CI [0.10, 4.48]; <i>P</i> = 1.000). Although most secondary outcomes did not reach statistical significance, the MRC sum score demonstrated significantly greater improvement in the efgartigimod cohort than in the IVIg cohort (<i>P</i> = 0.007). In addition, efgartigimod demonstrated significantly more favorable trajectories of NfL levels and anti-GM1 antibody titers compared with IVIg (<i>P</i> &lt; 0.001).</p> Interpretation <p>Efgartigimod demonstrated superiority in one secondary outcome and two exploratory measures, suggesting its potential as alternative to IVIg in GBS management.</p>

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Comparison of intravenous efgartigimod and intravenous immunoglobulin in patients with Guillain–Barré syndrome

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年10月21日

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  • Huiqiu Zhang,
  • Feipeng Zhai,
  • Menghan Su,
  • Yi Zhang,
  • Jing Ma,
  • Junsen Zhao,
  • Juan Wang,
  • Xueli Chang,
  • Yi Liu,
  • Junhong Guo,
  • Wei Zhang

摘要

Objective

This study aimed to compare the effectiveness of intravenous efgartigimod and intravenous immunoglobulin (IVIg) in patients with Guillain–Barré syndrome (GBS).

Methods

This dual-center, retrospective study analyzed prospectively collected data from adult patients with severe GBS who received either efgartigimod or IVIg. The primary outcome was the proportion of patients who achieving a GBS Disability Scale (GBS-DS) score ≤ 2 at 4 weeks post-treatment. Secondary outcomes included the proportion of patients achieving GBS-DS ≤ 2 at week 24; ≥ 1-grade improvement in GBS-DS at weeks 4 and 24; GBS-DS grade at week 4; and changes in GBS-DS, Medical Research Council (MRC) sum score, and other validated disability measures at weeks 1, 2, 4, 8, 16, and 24. Baseline serum levels of neurofilament light chain (NfL) and anti-GM1 antibodies, and their dynamic changes at 1 week post-treatment were assessed as exploratory outcomes.

Results

Twenty-one patients were enrolled (efgartigimod: n = 9; IVIg: n = 12). The primary outcome was not achieved (OR = 0.67, 95% CI [0.10, 4.48]; P = 1.000). Although most secondary outcomes did not reach statistical significance, the MRC sum score demonstrated significantly greater improvement in the efgartigimod cohort than in the IVIg cohort (P = 0.007). In addition, efgartigimod demonstrated significantly more favorable trajectories of NfL levels and anti-GM1 antibody titers compared with IVIg (P < 0.001).

Interpretation

Efgartigimod demonstrated superiority in one secondary outcome and two exploratory measures, suggesting its potential as alternative to IVIg in GBS management.