早期使用维加巴特林治疗不能减少焦点发作或改善结核性硬化综合体的认知:PREVeNT试验
Elizabeth Martina Bebin1, Jurriaan M Peters2, Brenda E Porter3
1Department of Neurology, University of Alabama at Birmingham, Birmingham, AL, USA.
Annals of neurology
|August 28, 2023
概括
在结核性硬化综合体 (TSC) 婴儿的早期vigabatrin治疗没有改善神经认知结果. 然而,它与较晚的发病和较低的婴儿发病率有关,这表明对特定发作类型的潜在益处.
科学领域:
- 儿科神经学 儿科神经学
- 发病学 (Epileptology) 是一个专业的学科.
- 发育神经科学的发展神经科学.
背景情况:
- 结核性硬化综合体 (TSC) 是一种与和发育挑战相关的遗传疾病.
- 早期干预对于优化TSC婴儿神经发育结果至关重要.
- 维加巴特林是治疗婴儿的既定治疗方法,但其在预防其他类型的和改善TSC整体神经认知方面的作用仍在调查中.
研究的目的:
- 评估早期vigabatrin治疗的疗效,在形脑电图 (EEG) 活动开始时开始,与患有TSC的婴儿在发作时的治疗相比.
- 为了确定早期vigabatrin对24个月龄神经认知结果的影响.
- 评估早期vigabatrin对发病率,耐药性和安全性的影响.
主要方法:
- 一个IIb期,多中心,随机,双盲,安慰剂受控试验.
- 患有TSC的婴儿在第一次形脑电图 (EEG) 或在发作开始时接受了vigabatrin.
- 主要结局:贝利婴儿和幼儿发展尺度,第三版 (贝利-III). 24个月的认知评分.
主要成果:
- 在24个月后,早期vigabatrin和安慰剂组之间没有观察到贝利-III认知评分的显著差异.
- 与安慰剂相比,早期vigabatrin没有显著改变整体发病率或耐药性的发病率.
- 维加巴林组的发病率较低,婴儿的发病时间较晚,各组的不良事件概况相似.
结论:
- 在发作发作之前基于EEG型活动的vigabatrin的启动不会改善TSC婴儿24个月的神经认知结果.
- 预防性vigabatrin不会延迟发作或减少该人群中焦点发作和耐药性的发生率.
- 早期vigabatrin治疗在减少TSC婴儿婴儿发作的发生率和延迟发作方面具有好处.
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