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抗MAG神经病变:历史方面,临床病理相关性,以及对未来治疗试验的考虑

Norman Latov¹, Thomas H Brannagan², Howard W Sander³

¹Weil Medical College of Cornell University, Peripheral Neuropathy Center, New York, New York, United States.

Arquivos de neuro-psiquiatria

|February 7, 2024

概括

抗髓相关葡萄糖蛋白 (anti-MAG) 神经病变是一种渐进的疾病. 改善临床试验设计对于评估针对自抗体减少的抗B细胞疗法至关重要.

科学领域:

神经学神经学
免疫学免疫学免疫学

背景情况:

抗髓关联葡萄糖蛋白 (anti-MAG) 神经病症的特征是远程脱髓化多神经病症,IgM单克隆性血脉病症和高抗MAG抗体标位.
这种情况在进展之前可能会出现多年的惰,外围神经可能变得无法激发,掩盖了脱髓化特征.

研究的目的:

审查抗MAG神经病变的临床表现,疾病过程,病理生理学和治疗.
为设计这种疾病的有效治疗临床试验提供考虑.

主要方法:

关于抗MAG神经病变的医学和科学出版物的综合文献综述.
分析设计临床试验在外围神经病变的策略.

主要成果:

抗MAG抗体已被证实是神经病变的致病原体,尽管外围神经可能变得无法激发,掩盖脱髓化.
虽然抗B细胞疗法在病例系列中表现有前途,但对照试验的结果不确定,可能是由于试验设计不足最佳.

结论:

未来的治疗试验应招募具有典型抗MAG神经病变,虚弱,高抗体标位和脱髓化证据的患者.
治疗策略应旨在使用抗B细胞药物,可能在组合中,至少减少60%的自身抗体度.
建议使用2年的试验持续时间和下肢神经病变损伤得分 (NIS-LL) 结合下肢功能 (LLF) 得分作为合适的初级结局措施.

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