Work

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

Year: 2025

Type: article

Abstract: 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.

Source: Orphanet Journal of Rare Diseases

DOI: 10.1186/s13023-025-04060-0