在接受托弗森治疗的SOD1-ALS患者中发生神经退行性和神经炎症性变化
Cecilia Simonini1, Elisabetta Zucchi2,3, Ilaria Martinelli1
1Department of Neurosciences, Ospedale Civile Baggiovara, Azienda Ospedaliero Universitaria di Modena, Modena, Italy.
Scientific reports
|April 1, 2025
概括
对SOD1-ALS的托弗森治疗显著降低了神经丝水平,但增加了神经炎症标志物. 这些变化与疾病进展无关,这表明对抗感性寡核酸治疗的潜在免疫反应.
科学领域:
- 神经科学是一个神经科学.
- 生物化学 生物化学
- 遗传学 遗传学 是一个
背景情况:
- 肌缩侧面硬化症 (ALS) 是一种进展性神经退行性疾病.
- 特定的反感性寡核酸 (ASO) 疗法,tofersen,针对ALS中的SOD1病理.
- 以前的研究表明,托弗森可以降低神经丝水平,但神经炎症的影响尚未评估.
研究的目的:
- 评估托弗森对SOD1-ALS患者神经炎症标志物的影响.
- 分析神经退行/神经炎症和临床疾病进展的生物标志物之间的相关性.
- 确定潜在的生物标志物来预测治疗反应或托弗森的不良影响.
主要方法:
- 在18名SOD1-ALS患者的多中心研究中,他们接受了tofersen治疗.
- 半自动免疫测试 (EllaTM技术) 用于测量NfL,NfH,CHI3L1和SerpinA1在血清和脑液中.
- 通用线性混合模型用于分析纵向生物标志物趋势和临床变量.
主要成果:
- 托弗森治疗导致脑神经丝轻链 (NfL) 和重链 (NfH) 水平逐渐下降.
- 在托弗森治疗期间,脑液中CHI3L1和SerpinA1 (神经炎症标志物) 的水平随着时间的推移而增加.
- 生物标志物趋势与疾病进展率之间没有发现相关性.
结论:
- 在SOD1-ALS患者中,托弗森有效降低神经纤维水平.
- 在托弗森治疗期间神经炎症标志物增加可能表明对ASO的免疫反应.
- 需要进一步的研究,以了解长期疗效,并确定预测性生物标志物,用于托弗森治疗ALS.
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