Choi Kalam, Fu Peicai, Yan Jinyi, Li Zhijun
Department of Neurology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.
Brain Behav. 2025 Aug;15(8):e70717. doi: 10.1002/brb3.70717.
Autoimmune nodopathy (AN) is a rare immune-mediated peripheral neuropathy, the diagnosis and treatment of which remain challenging. Anti-neurofascin-155 (NF155) AN present with weakness, tremor, ataxia, and cranial nerve involvement. Ofatumumab, a second-generation anti-CD20 monoclonal antibody, functions by depleting B lymphocytes. This study aimed to investigate the efficacy of ofatumumab treatment in NF155 AN.
We reviewed data on ofatumumab treatment of patients with anti-NF155 AN at the Department of Neurology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology from January 2020 to September 2024.
A total of four patients with anti-NF155 AN were included, consisting of three males and one female. The onset age ranged from 9 to 27 years. All cases exhibited distal sensory-motor neuropathy accompanied by tremors and ataxia. All the patients received corticosteroids or other immunotherapy or both before treatment with ofatumumab. The duration of treatment with ofatumumab ranged from 6 to 25 months, with a follow-up period of 6-28 months. The clinical improvements of patients were assessed using the Inflammatory Neuropathy Cause and Treatment Disability Score, the Medical Research Council Muscle Strength Score, and the Modified Rankin Scale. All four patients demonstrated significant clinical improvement following ofatumumab treatment, which was concurrently accompanied by varying levels of improvement in imaging and nerve conduction studies. Throughout the treatment period, the patients did not report any adverse reactions to ofatumumab.
Our research findings suggested that ofatumumab holds significant potential for treating anti-NF155 AN.
自身免疫性结节病（AN）是一种罕见的免疫介导性周围神经病，其诊断和治疗仍然具有挑战性。抗神经束蛋白-155（NF155）型AN表现为肌无力、震颤、共济失调和颅神经受累。奥法木单抗是一种第二代抗CD20单克隆抗体，通过消耗B淋巴细胞发挥作用。本研究旨在探讨奥法木单抗治疗NF155型AN的疗效。
我们回顾了2020年1月至2024年9月在华中科技大学同济医学院附属同济医院神经内科接受奥法木单抗治疗的抗NF155型AN患者的数据。
共纳入4例抗NF155型AN患者，其中男性3例，女性1例。发病年龄在9至27岁之间。所有病例均表现为远端感觉运动性神经病，并伴有震颤和共济失调。所有患者在接受奥法木单抗治疗前均接受了皮质类固醇或其他免疫治疗或两者兼用。奥法木单抗的治疗时间为6至25个月，随访时间为6至28个月。使用炎性神经病病因与治疗残疾评分、医学研究理事会肌力评分和改良Rankin量表评估患者的临床改善情况。所有4例患者在接受奥法木单抗治疗后均表现出显著的临床改善，同时影像学和神经传导研究也有不同程度的改善。在整个治疗期间，患者未报告对奥法木单抗有任何不良反应。
我们的研究结果表明，奥法木单抗在治疗抗NF155型AN方面具有巨大潜力。
