Efgartigimod as rescue treatment in acute phase of neuromyelitis optica spectrum disorder: A Case Report
Zhizhong Li,
Qiao Xu,
Jialu Huang,
Qiyuan Zhu,
Xiaolin Yang,
Mengjie Zhang,
Shaoru Zhang,
Siyuan Huang,
Gang Yu,
Peng Zheng,
Xinyue Qin,
Jinzhou Feng
Affiliations
Zhizhong Li
Department of Neurology, The First Affiliated Hospital of Chongqing Medical University, Chongqing, China
Qiao Xu
Department of Neurology, The First Affiliated Hospital of Chongqing Medical University, Chongqing, China
Jialu Huang
958th Hospital of the People's Liberation Army, Chongqing, 400038, China
Qiyuan Zhu
Department of Radiology, The First Affiliated Hospital of Chongqing Medical University, Chongqing, China
Xiaolin Yang
Department of Neurology, The First Affiliated Hospital of Chongqing Medical University, Chongqing, China
Mengjie Zhang
Department of Neurology, The First Affiliated Hospital of Chongqing Medical University, Chongqing, China
Shaoru Zhang
Department of Neurology, The First Affiliated Hospital of Chongqing Medical University, Chongqing, China
Siyuan Huang
Department of Neurology, The First Affiliated Hospital of Chongqing Medical University, Chongqing, China
Gang Yu
Department of Neurology, The First Affiliated Hospital of Chongqing Medical University, Chongqing, China
Peng Zheng
Department of Neurology, The First Affiliated Hospital of Chongqing Medical University, Chongqing, China
Xinyue Qin
Department of Neurology, The First Affiliated Hospital of Chongqing Medical University, Chongqing, China; Corresponding author. Department of Neurology, The First Affiliated Hospital of Chongqing Medical University, Chongqing, 400016, China.
Jinzhou Feng
Department of Neurology, The First Affiliated Hospital of Chongqing Medical University, Chongqing, China; Corresponding author. Department of Neurology, The First Affiliated Hospital of Chongqing Medical University, Chongqing, 400016, China.
Introduction: Neuromyelitis optica spectrum disorder (NMOSD) is a central nervous system demyelinating disease. Current therapy methods, however, have limited effect on acute attacks except for intravenous methylprednisolone (IVMP). Efgartigimod is a first-in-class novel human immunoglobulin G1 (IgG1) Fc fragment approved for the treatment of generalized myasthenia gravis. Its capacity to rapidly decrease serum IgG levels, including pathogenic autoantibodies, positions it as a potentially effective option for managing the acute phase of NMOSD. Case presentation: We report the case of a 59-year-old female patient with acute NMOSD, presenting with vision loss and numbness in all four limbs. Despite an initial inadequate response to intravenous methylprednisolone (IVMP), the addition of Efgartigimod to her treatment regimen led to rapid improvement, notably including a significant reduction in serum aquaporin-4 antibody titers, total IgG levels, and inflammation cytokine levels. Furthermore, no adverse events were reported during a four-month follow-up period. Conclusion: As an adjunct to glucocorticoid therapy, Efgartigimod has proven effective and safe for this patient. However, to ascertain its potential as a novel therapeutic option for acute NMOSD, larger-scale prospective clinical trials are required.