Sun Chong, Hu Jianian, Zhao Yanyin, Zheng Yongsheng, Meng Quanhua, Luo Sushan, Qiao Kai, Sun Jian, Lu Jiahong, Lin Jie, Zhao Chongbo
Department of Neurology, Huashan Hospital, Fudan University, Shanghai, China.
National Center for Neurological Disorders, Fudan University, Shanghai, China.
Front Immunol. 2025 May 1;16:1533167. doi: 10.3389/fimmu.2025.1533167. eCollection 2025.
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.
Clinical 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.
慢性炎症性脱髓鞘性多发性神经根神经病（CIDP）是一种自身免疫性神经病，由于标准治疗方法的局限性，其治疗面临挑战。艾加莫德是一种新生儿Fc受体拮抗剂，已显示出在治疗包括CIDP在内的抗体介导疾病方面的潜力（ADHERE研究），但关于艾加莫德在CIDP中应用的真实世界研究仍然缺乏。本研究旨在评估艾加莫德在中国5例CIDP患者中的短期疗效和安全性。
使用炎症性神经病病因与治疗（INCAT）残疾量表、炎症性拉施构建的总体残疾量表（IRODS）、医学研究委员会（MRC）总分（0 - 60）、握力、神经病损伤评分（NIS）和3米起立行走测试（TUG）评估临床疗效。通过监测不良事件以及测量白细胞计数、血清白蛋白浓度和血浆IgG浓度来评估安全性。在艾加莫德治疗前后测量外周血CD4 T淋巴细胞和CD19 B淋巴细胞。
所有5例（100%）患者对艾加莫德治疗有反应，4例（80%）在8周内达到预定疗效标准。总IgG的平均降低率为43%。不良事件极少，1例患者出现短暂腹泻，未观察到原有病情加重。
艾加莫德在CIDP的短期治疗中显示出有前景的疗效和安全性，提供了一种潜在的替代疗法。本研究提供了艾加莫德在CIDP真实世界应用中的有价值证据，结果表明有必要进行进一步研究。
