评估儿童多发性硬化症患者的疗效结果,同时通过USNPMSC注册表通过使用Avonex或Plegridy
Melissa A Wright1, Bradley J Barney1, Skyler Peterson1
1Department of Pediatrics (MAW, BJB, SP, MW, TCC), Utah Data Coordinating Center (BJB), and Department of Neurology (JR), University of Utah, Salt Lake City, UT, USA.
Journal of child neurology
|September 16, 2025
概括
肌内干扰素β-1a (Avonex) 和皮下干扰素β-1a (Plegridy) 在预防儿科多发性硬化症患者复发方面表现出类似的有效性. 这些疾病修饰疗法可能会使年轻患者受益,尽管缺乏正式批准.
科学领域:
- 神经学 神经学
- 免疫学 免疫学 免疫学
- 儿科 儿科 儿科
背景情况:
- 多发性硬化症 (MS) 是一种慢性自身免疫性疾病,影响中枢神经系统.
- 疾病修饰疗法 (DMT) 对于治疗多发性硬化症至关重要,但对儿科患者的数据有限.
- 肌肉内干扰素β-1a (Avonex) 和皮下干扰素β-1a (Plegridy) 是已知的多发性硬化症治疗方法.
研究的目的:
- 为了评估Avonex和Plegridy在患有多发性硬化症的儿科患者中的疗效.
- 为了在这个队列中比较Avonex和Plegridy之间的复发率和无复发概率.
- 评估这些DMT在患有MS的青少年中的潜在益处.
主要方法:
- 来自美国儿科多发性硬化中心网络的154名儿科多发性硬化患者的回顾性分析.
- 纳入标准:在18岁之前接受阿文克斯,Plegridy或两者的治疗.
- 数据收集包括3个月的"洗"期后的复发状态.
主要成果:
- 在第一年没有复发的概率:阿维尼克斯68.3%,Plegridy69.9%.
- 年化复发率:阿文尼克斯为0.50,Plegridy为0.59.
- 儿科患者的疗效与成年人群中观察到的疗效相当.
结论:
- 艾沃内克斯和Plegridy在儿童多发性硬化症患者中表现出可比的疗效.
- 这些DMT可以为患有多发性硬化症的青少年提供好处,即使没有正式的儿科批准.
- 儿童多发性硬化症的知情治疗决策需要了解DMT在这个年龄组的疗效.
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