Efficacy and Safety of Efgartigimod for Patients With Guillain–Barré Syndrome: A Retrospective Cohort Study
Objective:
This study was to evaluate the efficacy and safety of efgartigimod compared to intravenous immunoglobulin G (IVIg) and IVIG plus efgartigimod for GBS patients.
Background:
Guillain–Barré syndrome (GBS) is a serious neurological condition with limited treatment options. Efgartigimod, a human IgG antibody Fc fragment that acts as a natural ligand for the FcRn, can increase IgG degradation, which thus may be a promising therapeutic drug for GBS.
Design/Methods:
A retrospective study was conducted on GBS patients who visited the first affiliated hospital of Zhengzhou university and Nanyang central hospital from February 2024 to March 2025. The medical records of these patients who received efgartigimod or IVIg alone or efgartigimod plus IVIg were reviewed. Clinical improvement was evaluated using GBS disability score (GBS-DS) and injury severity score (INCAT) at week4. Any side effects that occurred during the treatment period were recorded.
Results:
A total of 52 patients were enrolled and treated with the efgartigimod (n=16), IVIg (n=20), or efgartigimod plus IVIg (n=16). At week 4, the proportion of patients reaching GBS-DS ≤1 was numerically superior in the efgartigimod group (62.5%) compared to the IVIg (45.0%) and combination (37.5%) groups, although the difference was not statistically significant (p=0.35). In addition, the time for efgartigimod group to reach GBS≤1 was 2.6±1.17weeks, compared to the 3.11±1.05 and 2.83±1.17weeks in other two group, efgartigimod seemed to help patients achieve good functional prognosis faster, but also did not reach statistical differences(p=0.62). A lower incidence of treatment-related adverse events (TEAEs)was observed in the efgartigimod group (18.8%, 3/16) compared to the IVIg (50.0%, 10/20) and combination (43.8%, 7/16) groups.
Conclusions:
Compared with IVIg and efgartigimod plus IVIg, efgartigimod seems to be able to improve the symptoms of GBS patients more quickly and safely. This study indicates the potential role of efgartigimod as a novel treatment option for GBS.
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