A prospective real-world study of efgartigimod in the treatment of chronic inflammatory demyelinating polyradiculoneuropathy

Objective To evaluate the short-term clinical efficacy and safety of efgartigimod in the treatment of Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) in a real-world setting. Methods This prospective real-world study enrolled 12 CIDP patients receiving efgartigimod treatment at the Department of Neurology, The First Affiliated Hospital of Chongqing Medical University. Efficacy was comprehensively assessed using the Medical Research Council (MRC) scale for muscle strength, the inflammatory Rasch-built Overall Disability Scale (I-RODS) for disability, and the Inflammatory Neuropathy Cause and Treatment (INCAT) scale. Assessments were performed before the first dose and one week after each subsequent dose. Adverse events during treatment, concomitant use of other immunosuppressants, routine laboratory tests, electrocardiograms, and immunoglobulin levels before and after treatment were recorded. Results Among the 12 patients, 50% received subcutaneous injection and 50% received intravenous infusion of efgartigimod. During the follow-up period, significant efficacy was observed overall, MRC scores increased from 45.92 ± 15.69 to 55.00 ± 10.14, I-RODS scores increased from 49.25 ± 24.21 to 75.42 ± 28.46, and INCAT scores decreased from 4.42 ± 3.20 to 1.42 ± 2.61. By the end of the follow-up at the fifth week after the first dose, 11 patients (91.7%) achieved clinical improvement. No significant difference in efficacy was found between the subcutaneous and intravenous administration routes. Laboratory tests showed a downward trend in IgG levels after treatment, with no significant decrease in albumin. The incidence of adverse events during treatment was low, with only one patient experiencing a localized rash. Conclusion This study demonstrates the short-term efficacy and safety of efgartigimod in treating CIDP, suggesting its potential as a safe and effective alternative therapy. Further exploration is needed to determine its suitability for long-term maintenance treatment of CIDP.