Hu Jianian, Zheng Yongsheng, Sun Chong, Sun Jian, Xi Jianying, Luo Sushan, Qiao Kai, Zhao Chongbo, Lin Jie
Department of Neurology, Huashan Hospital Fudan University, Shanghai, China.
Rare Disease Center, Huashan Hospital Fudan University, Shanghai, China.
J Peripher Nerv Syst. 2025 Mar;30(1):e12679. doi: 10.1111/jns.12679.
To investigate the treatment of ofatumumab in autoimmune nodopathy (AN).
An open-label, prospective, observational study was conducted in patients with AN. The regimen was 20 mg ofatumumab subcutaneously on day 0, 7, 14, 28, and subsequently every 4 weeks in a total of 24 weeks. The primary endpoint of the study was the proportion of patients with confirmed clinical improvement.
All of the eight patients (100%) improved at Week 24. The median time to improvement was 8 (IQR: 7-10) weeks. The four patients previously treated with rituximab and two with irregular injections of ofatumumab (OFA) improved. At Week 24, the adjusted INCAT score, MRC sum score, cI-RODS, and grip strength in nondominant hand significantly improved from baseline. In nerve conduction studies, all of the six patients with available data (100%) improved. The median sNfL significantly reduced from baseline at Week 8. Anti-paranodal antibody in seven patients with anti-NF155 antibodies reduced from baseline at Week 20. In seven of the eight patients, CD19+ B cells were significantly reduced at Week 4. No serious adverse events were reported.
The regimen was 20 mg ofatumumab subcutaneously on day 0, 7, 14, 28, and every 4 weeks from Week 4, in a total of 24 weeks. The ofatumumab therapy may provide a more convenient and safer treatment for patients with AN, while serving as an effective alternative for those who did not respond to rituximab.
研究奥法妥木单抗治疗自身免疫性结节病（AN）的效果。
对AN患者进行了一项开放标签、前瞻性观察性研究。治疗方案为在第0、7、14、28天皮下注射20mg奥法妥木单抗，随后每4周注射一次，共24周。该研究的主要终点是临床改善得到确认的患者比例。
8例患者（100%）在第24周时均有改善。改善的中位时间为8周（四分位间距：7 - 10周）。先前接受利妥昔单抗治疗的4例患者以及2例接受不规则奥法妥木单抗（OFA）注射的患者均有改善。在第24周时，校正后的INCAT评分、医学研究委员会（MRC）总分、cI - RODS以及非优势手的握力较基线水平均显著改善。在神经传导研究中，有可用数据的6例患者（100%）均有改善。第8周时，血清神经丝轻链（sNfL）中位数较基线水平显著降低。7例抗NF155抗体阳性患者的抗结旁抗体在第20周时较基线水平降低。8例患者中的7例在第4周时CD19 + B细胞显著减少。未报告严重不良事件。
治疗方案为在第0、7、14、28天皮下注射20mg奥法妥木单抗，从第4周开始每4周注射一次，共24周。奥法妥木单抗治疗可能为AN患者提供更方便、更安全的治疗方法，同时为那些对利妥昔单抗无反应的患者提供一种有效的替代治疗方案。
