Efficacy and safety of intravenous efgartigimod for chronic inflammatory demyelinating polyneuropathy: A case series real-world study

Abstract

Objective

To assess the treatment effecitiveness and safety of Chinese patients with chronic inflammatory demyelinating polyneuropathy (CIDP) treated with intravenous efgartigimod in this real-world study.

Methods

This study analyzed data retrospectively from 13 patients with CIDP treated with efgartigimod at the Department of Neurology, First Affiliated Hospital of Sun Yat-Sen University from December 2023 to September 2024. Clinical assessment scales included the Inflammatory Neuropathy Cause and Treatment (INCAT) score and the Medical Research Council (MRC) sum score were utilized to evalutate effecitiveness before and after efgartigimod treatments. Adverse events werer monitored to assess the safety of efgartigimod treatments.

Results

The mean onset age of 13 patients was 38.3 years, and the mean duration from symptom onset to diagnosis was 13.1 months. About 46 % patients in our study were typical CIDP subtype. About 77 % patients experienced relapses before efgartigimod treatments. Clinical improvements were observed in 12 patients (92.3 %) with at least 1-point decrease on INCAT scores at 12 weeks follow-up, indicating efgartigimod treatment effectiveness. Eleven patients (84.6 %) achieved clinical improvements at 24 weeks follow-up. At last follow-up, five patients (38.5 %) had discontinued immunotherapy. Adverse events monitoring revealed that only one patient had pruritus which alleviated spontaneously.

Conclusion

This case series real-world study support the efficacy and safety of efgartigimod treatment in CIDP patients, benefit on partially reducing the usage of glucocorticoids and immunosuppressants. It was well tolerated across age groups. Larger prospective studies are needed to refine dosing and establish guidelines for CIDP management.