评估ALS疾病进展:预后子组和异常值
Inês Alves1, Marta Gromicho1, Miguel Oliveira Santos1,2
1Faculdade de Medicina, Centro de Estudos Egas Moniz, Instituto de Medicina Molecular João Lobo Antunes, Universidade de Lisboa, Lisbon, Portugal.
Amyotrophic lateral sclerosis & frontotemporal degeneration
|September 28, 2024
概括
经过修订的ALS功能评分尺度 (ALSFRS-R) 降低 (ΔFS) 预测了肌缩侧面硬化症 (ALS) 的存活率. 一些患者偏离预期的生存期,表明非线性疾病进展和未知的生物因素.
科学领域:
- 神经学 神经学
- 生物标志物 生物标志物
- 预测 预测 预测 预测 预测
背景情况:
- 从症状发作到诊断 (ΔFS) 的ALS功能评级尺度修订 (ALSFRS-R) 衰退是对肌缩侧面硬化症 (ALS) 存活率的验证预后生物标志物.
- 了解生存率偏差与 ΔFS 定义的预后对于患者护理至关重要.
研究的目的:
- 根据初始的 ΔFS. 来分类一个大型的 ALS 患者队列.
- 调查生存结果偏离基于 ΔFS 的预后预期.
主要方法:
- 使用ΔFS百分位数将1056名ALS患者分为缓慢,中等和快速进展的患者.
- 生存被分为短期 (<2年),平均 (2-5年) 和长期 (>5年) 的结果.
- 在子组内分析了临床和人口特征.
主要成果:
- 在 ΔFS 分层中,发现了分界值 <0.29,0.29-1.03 和 >1.03 分/月的进展类别.
- 长期生存者占26%,而21%是短暂生存者.
- 观察到的差异:6%的快速进步者活了5年以上,13%的中间进步者活了2年以下,无法通过分析的因素来解释.
结论:
- 重新证实 ΔFS 是一种预后生物标志物,用于 ALS 存活率.
- 确定了患者异常值,这些异常值不符合基于 ΔFS 的生存预测.
- 突出了ALS进展的非线性性质,可能受到遗传或未知的生物因素的影响,需要进一步研究.
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