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

This study aimed to compare the effectiveness of intravenous efgartigimod and intravenous immunoglobulin (IVIg) in patients with Guillain–Barré syndrome (GBS). 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. 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). Efgartigimod demonstrated superiority in one secondary outcome and two exploratory measures, suggesting its potential as alternative to IVIg in GBS management.