Tai Hongfei, Niu Songtao, Pan Hua, Jian Fan, Hu Zhenxian, Chen Na, Wang Ying, Zhang Zaiqiang
Department of Neurology, Beijing Tiantan Hospital, Capital Medical University, Beijing, China.
China National Clinical Research Center for Neurological Diseases, Beijing, China.
Eur J Neurol. 2025 Jul;32(7):e70269. doi: 10.1111/ene.70269.
Autoimmune nodopathy exhibits suboptimal responses to conventional immunotherapies. This study investigates the efficacy and safety of efgartigimod, a neonatal Fc receptor blocker, in this condition.
A prospective single-center study enrolled four antibody-confirmed autoimmune nodopathy patients receiving weekly efgartigimod (10 mg/kg) over 4 weeks. Disease progression was assessed using validated neurological scales (INCAT, ISS, I-RODS, and MRC) at baseline (Week 0), weekly during treatment (Weeks 1-4), and 4-week post-treatment follow-up (Week 8).
Four patients (3 females, aged 17-72) responded to efgartigimod within 2 weeks, showing varied improvement based on antibody subtype. Patient 1 (anti-NF186 IgG3+) achieved full remission by Week 2 (INCAT 3 → 0). Patient 2 (anti-NF155 IgG4+) improved progressively (MRC 112 → 119; I-RODS 36 → 40). Patient 3 (anti-NF155 IgG1/IgG4+) quickly stabilized gait in the first week and gradually recovered (INCAT 5 → 2). Patient 4 (anti-CNTN1 IgG1/IgG2/IgG3/IgG4+) reduced tremors rapidly and improved sensorimotor function (ISS 8 → 6; I-RODS 12 → 14) despite a treatment interruption due to a fracture. Antigen-specific efficacy varied: NF186 neuropathy resolved completely, while IgG4-dominant paranodal cases (NF155/CNTN1) partially recovered, prompting sequential B-cell-targeted strategies. No severe adverse events occurred.
Efgartigimod provided rapid functional recovery in autoimmune nodopathy. Differential responses by IgG subclass and antigenic targets highlight the necessity for biomarker-guided strategies.
自身免疫性神经病对传统免疫疗法反应欠佳。本研究调查了新生儿Fc受体阻滞剂艾加莫德在这种情况下的疗效和安全性。
一项前瞻性单中心研究纳入了4例经抗体确认的自身免疫性神经病患者，他们在4周内每周接受艾加莫德（10mg/kg）治疗。在基线（第0周）、治疗期间每周（第1 - 4周）以及治疗后4周随访（第8周），使用经过验证的神经学量表（INCAT、ISS、I - RODS和MRC）评估疾病进展。
4例患者（3名女性，年龄17 - 72岁）在2周内对艾加莫德有反应，根据抗体亚型显示出不同程度的改善。患者1（抗NF186 IgG3 +）在第2周实现完全缓解（INCAT从3降至0）。患者2（抗NF155 IgG4 +）逐渐改善（MRC从112升至119；I - RODS从36升至40）。患者3（抗NF155 IgG1/IgG4 +）在第一周步态迅速稳定并逐渐恢复（INCAT从5降至2）。患者4（抗CNTN1 IgG1/IgG2/IgG3/IgG4 +）尽管因骨折中断治疗，但震颤迅速减轻且感觉运动功能改善（ISS从8降至6；I - RODS从12升至14）。抗原特异性疗效各不相同：NF186神经病完全缓解，而以IgG4为主的节旁病例（NF155/CNTN1）部分恢复，这促使采用序贯的B细胞靶向策略。未发生严重不良事件。
艾加莫德使自身免疫性神经病患者实现了快速功能恢复。IgG亚类和抗原靶点的不同反应凸显了生物标志物指导策略的必要性。
