ORIGINAL RESEARCH article
Front. Immunol.
Sec. Autoimmune and Autoinflammatory Disorders: Autoinflammatory Disorders
Volume 16 - 2025 | doi: 10.3389/fimmu.2025.1533167
Short-term treatment of CIDP with efgartigimod: a case series in China
Provisionally accepted
Chong Sun1,2,3
Jianian Hu1,2,3
Yanyin Zhao1,2,3
Yongsheng Zheng1,2,3Quanhua Meng4
Sushan Luo1,2,3Kai Qiao1,2,3Jian Sun1,2,3Jiahong Lu1,2,3
Jie Lin1,2,3*
Chongbo Zhao1,2,3- 1Department of Neurology, Huashan Hospital, Fudan University, Shanghai, Shanghai Municipality, China
- 2National Center for Neurological Disorders,, Fudan University, Shanghai, Shanghai Municipality, China
- 3Huashan Rare Disease Center, Shanghai Medical College, Fudan University, Shanghai, Shanghai Municipality, China
- 4People’s Hospital of Deyang City, Deyang, Sichuan Province, China
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Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is a type of autoimmune neuropathy with treatment challenges due to the limitations of standard of care therapies. Efgartigimod, a neonatal Fc receptor antagonist, has shown potential in treating antibody-mediated disorders including CIDP (ADHERE study), but real-world studies on the application of efgartigimod in CIDP are still lacking. This study aimed to evaluate the short-term efficacy and safety of efgartigimod in five patients with CIDP in China.effectiveness was assessed using the Inflammatory Neuropathy Cause and Treatment (INCAT) disability scale, Inflammatory Rasch-built Overall Disability Scale (IRODS), Medical Research Council (MRC) sum score(0-60), grip strength, Neuropathy Impairment Score (NIS), and 3-m Time Up and Go Test (TUG). Safety was evaluated by monitoring adverse events and measuring white blood cell count, serum albumin concentration, and plasma IgG concentration. Peripheral CD4 + T and CD19 + B lymphocytes were measured before and after efgartigimod treatment.All five (100%) patients responded to efgartigimod treatment, with four (80%) meeting predefined effectiveness criteria within 8 weeks. The average reduction rate in total IgG was 43%. Adverse events were minimal, with one patient experiencing transient diarrhea, and no aggravation of pre-existing conditions was noted.Efgartigimod demonstrates promising efficacy and safety for short-term treatment of CIDP, offering a potential alternative therapy. This study provides valuable evidence from the real-world application of efgartigimod in CIDP, and the results indicate further research is warranted.
Keywords: Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP), efgartigimod, Short-term, Treatment, Real-world study, China
Received: 23 Nov 2024; Accepted: 10 Apr 2025.
Copyright: © 2025 Sun, Hu, Zhao, Zheng, Meng, Luo, Qiao, Sun, Lu, Lin and Zhao. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Jie Lin, Department of Neurology, Huashan Hospital, Fudan University, Shanghai, Shanghai Municipality, China
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