Intravenous Efgartigimod or Intravenous Immunoglobulin in Guillain-Barre Syndrome: An Observational Multicenter Study.

BACKGROUND AND AIMS: Guillain-Barré syndrome (GBS) is an acute autoimmune polyradiculoneuropathy characterized by progressive flaccid paralysis. The standard treatments include intravenous immunoglobulin (IVIg) and plasma exchange (PE), with limited accessibility to these treatments. Efgartigimod is an FcRn antagonist that reduces IgG levels, similar to PE, serving as a potential alternative. This study aims to compare the efficacy of Efgartigimod with standard IVIg in GBS.

METHODS

This was a multicenter observational study in China. Eligible participants were aged ≥ 16 years, meeting the diagnostic criteria for GBS, and presented with a GBS Disability Scale score ≤ 5 at baseline assessment. Participants received efgartigimod (10 mg/kg per week up to 4 doses) or IVIg (2 g/kg once). Outcomes were assessed at baseline, week 1, and week 2 post-treatment, and at the last follow-up. The primary efficacy endpoint was the proportion of participants improving at least 1 point on the GBS-DS.

RESULTS

Between January and October 2024, 22 GBS patients were enrolled, including 11 in each group. At week 1, 14 patients improved in GBS-DS, with 7 (63.6%) in the efgartigimod group and 7 (63.6%) in the IVIg group. Median time to improvement was 4 days in the efgartigimod group and 8 days in the IVIg group. At week 2, GBS-DS response rates were 90.9% (10/11) for efgartigimod and 81.8% (9/11) for IVIg.

INTERPRETATION: Based on our preliminary findings, intravenous efgartigimod may be potentially tolerated in GBS over the short term. However, this study cannot establish comparative efficacy given the methodological limitations, and large-scale well-controlled head-to-head trials remain imperative.