重症肌无力
郎伯综合征
相伴的
CD19
医学
乙酰胆碱受体
自身抗体
神经肌肉传递
免疫学
自身免疫性疾病
内科学
抗原
抗体
受体
作者
Jeremias Motte,Melissa Sgodzai,Christiane Schneider‐Gold,Nina K. Steckel,Thomas Mika,Tobias Hegelmaier,Dominic Borie,Aiden Haghikia,Dimitrios Mougiakakos,Roland Schroers,Ralf Gold
出处
期刊:Neuron
[Cell Press]
日期:2024-05-01
卷期号:112 (11): 1757-1763.e2
被引量:27
标识
DOI:10.1016/j.neuron.2024.04.014
摘要
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.
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