从PREVENT试验的神经发育结果.
Sarah E O'Kelley1, Jamie K Capal2, Tarrant O McPherson3
1Departments of Psychology and Pediatrics, University of Alabama at Birmingham, Birmingham, Alabama.
Pediatric neurology
|October 8, 2025
概括
维加巴特林在患有结核性硬化综合体 (TSC) 的婴儿中没有显著改变发育或自闭症结果. 没有患有的参与者被诊断出患有自闭症谱系障碍 (ASD).
科学领域:
- 神经科学是一个神经科学.
- 发育儿科 发育儿科
- 遗传学 是一个遗传学.
背景情况:
- 结核性硬化综合体 (TSC) 是一种与,智力障碍和自闭症谱系障碍 (ASD) 的高发病率相关的遗传疾病.
- 早期干预对于管理TSC相关的神经发育挑战至关重要.
研究的目的:
- 评估vigabatrin在预防TSC的婴儿智力和发育障碍和ASD的疗效.
- 在PREVeNT试验中,评估36个月后的表型,发育和ASD特异性结果.
主要方法:
- PREVeNT试验是一项IIb期,多中心,双盲,安慰剂控制的研究,涉及84名患有TSC的婴儿.
- 从6个月到36个月进行神经发育评估,在36个月确定ASD诊断.
主要成果:
- 认知分数处于低平均范围,并随着时间的推移略有下降. 适应性得分在发作组中较低.
- 36个月后,语言分数与认知分数保持一致. 31%的参与者被诊断出患有自闭症,无论如何治疗.
- 在vigabatrin和安慰剂组之间没有观察到发育或ASD结局的显著差异.
结论:
- 维加巴特林在预防TSC的婴儿发育或自闭症特异性结局方面没有显著的有效性.
- 这项研究表明,早期发作检测和密切监测可能会影响结果.
- 没有患有的参与者患有ASD,这突显了TSC中和ASD之间的关键联系.
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