Myasthenia gravis (MG) and Lambert-Eaton myasthenic syndrome (LEMS) are autoimmune disorders affecting neuromuscular transmission. Their combined occurrence is rare, and treatment remains challenging. Two women diagnosed with concomitant MG/LEMS experienced severe, increasing disease activity despite multiple immunotherapies. Anti-CD19 chimeric antigen receptor (CAR) T cells have shown promise for treating autoimmune diseases. This report details the safe application of anti-CD19 CAR T cells for treating concomitant MG/LEMS. After CAR T cell therapy, both patients experienced rapid clinical recovery and regained full mobility. Deep B cell depletion and normalization of acetylcholine receptor and voltage-gated calcium channel N-type autoantibody levels paralleled major neurological responses. Within 2 months, both patients returned to everyday life, from wheelchair dependency to bicycling and mountain hiking, and remain stable at 6 and 4 months post-CAR T cell infusion, respectively. This report highlights the potential for anti-CD19 CAR T cells to achieve profound clinical effects in the treatment of neuroimmunological diseases.

中文翻译：

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使用自体 CD19 靶向 CAR T 细胞治疗重症肌无力和 Lambert-Eaton 肌无力综合征


重症肌无力 (MG) 和兰伯特-伊顿肌无力综合征 (LEMS) 是影响神经肌肉传递的自身免疫性疾病。它们同时发生的情况很少见，治疗仍然具有挑战性。尽管接受了多种免疫治疗，两名被诊断患有 MG/LEMS 的女性仍经历了严重且不断增加的疾病活动。抗 CD19 嵌合抗原受体 (CAR) T 细胞已显示出治疗自身免疫性疾病的前景。该报告详细介绍了抗 CD19 CAR T 细胞在治疗合并 MG/LEMS 中的安全应用。经过 CAR T 细胞治疗后，两名患者均经历了快速的临床康复并恢复了完全活动能力。深层 B 细胞耗竭以及乙酰胆碱受体和电压门控钙通道 N 型自身抗体水平的正常化与主要神经系统反应平行。 2 个月内，两名患者都恢复了日常生活，从依赖轮椅转向骑自行车和登山，并分别在 CAR T 细胞输注后 6 个月和 4 个月保持稳定。该报告强调了抗CD19 CAR T细胞在神经免疫疾病治疗中取得深远临床效果的潜力。