Orphanet Journal of Rare Diseases | Comparative Study of Intravenous Efgartigimod and IVIg in GBS Treatment

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This study is the first systematic comparison of efgartigimod and IVIg for GBS treatment. Results show efgartigimod demonstrates superior trends in secondary endpoints and exploratory metrics, providing a potential alternative therapy for GBS.

 

Literature Overview
The article 'Comparison of intravenous efgartigimod and intravenous immunoglobulin in patients with Guillain–Barré syndrome' published in Orphanet Journal of Rare Diseases systematically reviews and summarizes the efficacy and safety comparison between efgartigimod and IVIg in treating GBS. The study conducted a dual-center retrospective analysis evaluating 21 GBS patients receiving efgartigimod or IVIg, with primary endpoint being the proportion of patients with GBS-DS score ≤2 at week 4, and secondary endpoints including multiple functional score changes and biomarker dynamics.

Background Knowledge
Guillain-Barré Syndrome (GBS) is an acute immune-mediated polyneuropathy characterized by rapidly progressive limb weakness, potentially leading to respiratory failure. Current first-line treatments include intravenous immunoglobulin (IVIg) and plasma exchange (PLEX), but blood product shortages and individual response variability necessitate novel therapeutic approaches. Efgartigimod, an Fc fragment derived from human IgG1, accelerates IgG degradation through FcRn targeting, and has shown favorable efficacy and safety profiles in various IgG-mediated autoimmune diseases. This study is the first to evaluate its comparative effectiveness against IVIg in GBS patients, providing evidence for future clinical trial designs.

 

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Research Methods and Experiments
This dual-center retrospective cohort analysis collected data from GBS patients treated with efgartigimod (n=9) or IVIg (n=12) between November 2023 and March 2025, with minimum 4-week follow-up. Primary endpoint was proportion of patients achieving GBS-DS ≤2 at week 4, while secondary endpoints included functional score changes and biomarker dynamics. All patients met NINDS and Brighton Collaboration diagnostic criteria, with serum samples collected pre-treatment and 1 week post-treatment to measure neurofilament light chain (NfL) and anti-GM1 antibody levels.

Key Findings and Perspectives

• Primary endpoint (GBS-DS ≤2) showed no significant difference between efgartigimod (33.3%) and IVIg (25.0%) groups (OR=0.67, P=1.000)
• Efgartigimod demonstrated significantly better improvement in MRC total scores (P=0.007)
• Superior decline trajectories observed in NfL and anti-GM1 antibody levels with efgartigimod (P<0.001)
• IVIg group showed significant NfL elevation at 1-week post-treatment (P=0.004) while efgartigimod maintained stability
• Both groups showed good tolerability: 3 grade 3 pulmonary infections in efgartigimod group, 1 mortality due to refusal of mechanical ventilation in IVIg group
• Significant anti-GM1 antibody elevation observed in IVIg group at 1-week post-treatment (P=0.004) while efgartigimod remained stable

Significance and Future Directions
While primary endpoint was not statistically significant, efgartigimod showed superior trends in MRC scores and biomarker dynamics (NfL and anti-GM1). These findings provide effect size estimation and dosing optimization basis for future randomized controlled trials. Further evaluation of extended treatment duration or increased cumulative dosing is needed to enhance therapeutic efficacy.

 

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Conclusion
This study with limited sample size compares efgartigimod and IVIg for GBS treatment. Results demonstrate efgartigimod shows significant advantages in MRC total score improvement, NfL level reduction, and anti-GM1 antibody suppression despite no statistical difference in primary functional scores, suggesting its potential as IVIg alternative. Future studies with larger cohorts and randomized controlled trials are required to validate efficacy and safety, optimize treatment regimens, and improve clinical response rates. The biomarkers (NfL and anti-GM1) used in this study provide novel references for precision management in GBS treatment monitoring.

 

Huiqiu Zhang, Feipeng Zhai, Menghan Su, Junhong Guo, and Wei Zhang. Comparison of intravenous efgartigimod and intravenous immunoglobulin in patients with Guillain–Barré syndrome. Orphanet Journal of Rare Diseases.