Efficacy and Safety of Ofatumumab Treatment for Anti‐ NMDA Receptor Autoimmune Encephalitis ( OFF ‐ AE ): A Prospective, Multicenter Cohort Study

作者：Kundian Guo, Fuhua Peng, Jia Liu, Youming Long, Shougang Guo, Honghao Wang, Gang Yu, Yanlin Zhang, Xiong Han, Ewen Tu, Yake Zheng, Jialu Huang, Yanxia Zhou, Dongmei An, Guanyan Lin, Baojie Wang, Yuan Yuan Wang, Ping Yang, Yu Jiang, Beijia Cui, Zhenyu Yang, Maolin Du, Meiling Jiang, Limin Qin, Xueying Kong, Xue Gong, Xu Liu, Linjun Cai, Jinmei Li, Dong Zhou, Zhen Hong · 发表于：Annals of Neurology · 年份：2025 · DOI：10.1002/ana.27218 · 被引用次数：11 · 研究领域：Autoimmune Neurological Disorders and Treatments、Multiple Sclerosis Research Studies、Monoclonal and Polyclonal Antibodies Research

OBJECTIVE: Ofatumumab presents a potentially promising alternative to current second-line immunotherapy for refractory anti-N-methyl-D-aspartate receptor autoimmune encephalitis (NMDAR-AE). We aimed to evaluate the efficacy and safety of ofatumumab as a novel second-line immunotherapy for NMDAR-AE. METHODS: This prospective, multicenter, nested cohort study compared patients with NMDAR-AE from the CHina Autoimmune encephalitiS outcomE study registry (CHASE) recruited between October 2011 and February 2024, treated with and without ofatumumab. The primary outcome was the proportion reaching a favorable functional outcome (modified Rankin Scale [mRS] score ≤2) at the last follow-up. Secondary outcomes included mRS scores and Clinical Assessment Scale in Autoimmune Encephalitis (CASE) scores over the first 24-month follow-up and the proportion with further mRS score improvement after ofatumumab initiation. A propensity score matching was performed to balance major confounders. RESULTS: A total of 715 patients with AE were screened. Fifty-eight propensity score-matched patients with NMDAR-AE each in the ofatumumab group and non-ofatumumab group were analyzed. Fifty-four patients (93.1%) in the ofatumumab group achieved further mRS score improvement with a median time of 14 days from ofatumumab initiation, and 53 (91.4%) reached a favorable functional outcome at the last follow-up. For those who failed first-line immunotherapy, the ofatumumab group demonstrated a faster mRS score ...