Yang Wenjing, Chen Pei, Guo Jiaxuan, Feng Huiyu, Huang Xin
Department of Neurology, The First Affiliated Hospital, Guangdong Provincial Key Laboratory of Diagnosis and Treatment of Major Neurological Diseases, National Key Clinical Department and Key Discipline of Neurology, Sun Yat-Sen University, No. 58 Zhongshan Road 2, Guangzhou, 510080, China.
Guangxi Hospital Division of The First Affiliated Hospital, Sun Yat-Sen University, Nanning, China.
Orphanet J Rare Dis. 2024 Dec 21;19(1):483. doi: 10.1186/s13023-024-03501-6.
Neuromyelitis Optica Spectrum Disorders (NMOSD) comprise a group of autoimmune-mediated, inflammatory, demyelinating central nervous system diseases caused by aquaporin-4 (AQP4) IgG autoantibodies. Efgartigimod is a human IgG Fc fragment that reduces antibody titers by targeting the neonatal Fc receptor (FcRn). This study documents the efficacy of efgartigimod combined with intravenous methylprednisolone (IVMP) in the acute phase of NMOSD.
In this retrospective study, the medical records of NMOSD patients with acute attack who received efgartigimod plus IVMP or IVMP were reviewed. Treatment efficacy was assessed by the Expanded Disability Scale Score (EDSS) before and one month after treatment. Any side effects that occurred during the treatment period were recorded.
This study was performed on 11 patients (efgartigimod plus IVMP group [n = 4] and IVMP group [n = 7]). Efgartigimod plus IVMP was effective and had a satisfactory safety profile. EDSS was reduced by 0.5 ± 0.32 compared with the IVMP group (0.27 ± 0.02). Immunoglobulin was decreased in three patients, and the immunoglobulin G (IgG) levels gradually increased approximately 8 weeks after the last administration. Hyperlipidemia and elevated white blood cell count were common side effects. No infections or deaths occurred.
Efgartigimod plus IVMP treatment is safe and well-tolerated in patients with acute-phase NMOSD.
视神经脊髓炎谱系障碍（NMOSD）是一组由水通道蛋白4（AQP4）IgG自身抗体引起的自身免疫介导的、炎症性、脱髓鞘性中枢神经系统疾病。艾加莫德是一种人IgG Fc片段，通过靶向新生儿Fc受体（FcRn）降低抗体滴度。本研究记录了艾加莫德联合静脉注射甲泼尼龙（IVMP）在NMOSD急性期的疗效。
在这项回顾性研究中，回顾了接受艾加莫德加IVMP或IVMP治疗的急性发作NMOSD患者的病历。通过治疗前和治疗后1个月的扩展残疾量表评分（EDSS）评估治疗效果。记录治疗期间出现的任何副作用。
本研究对11例患者进行（艾加莫德加IVMP组[n = 4]和IVMP组[n = 7]）。艾加莫德加IVMP有效且安全性良好。与IVMP组（0.27±0.02）相比，EDSS降低了0.5±0.32。3例患者免疫球蛋白降低，末次给药后约8周免疫球蛋白G（IgG）水平逐渐升高。高脂血症和白细胞计数升高是常见的副作用。未发生感染或死亡。
艾加莫德加IVMP治疗对急性期NMOSD患者安全且耐受性良好。
