Patients with ALS show highly correlated progression rates in left and right limb muscles.
Patients with ALS show highly correlated progression rates in left and right limb muscles.
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ALS患者在左右肢肌肉中显示出高度相关的进展率。
DOI：
10.1212/wnl.0000000000004105
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发表时间：
2017-07-11
期刊：
影响因子：
9.9
通讯作者：
Svendsen CN
中科院分区：
文献类型：
--
作者：
Rushton DJ;Andres PL;Allred P;Baloh RH;Svendsen CN
肌萎缩性侧索硬化症（ALS）以不同的速率进展，使临床试验设计变得困难，并且取决于大量患者。当前，很少有数据显示左和右肢是以相同的或不同的速率进展。这项研究解决了ALS患者的特定肌肉群体的下降率，并评估了同一患者的左右肌肉之间是否存在关系，而不管整体进展如何。
使用2种不同的方法，随着时间的推移，使用大量患者来评估左右肢肌肉强度的下降：簇定量神经肌肉检查和肢体等距强度方案的准确测试。然后应用高级线性回归统计方法来评估每个肢体中的进展速率。
该报告表明，线性化进程模型可以准确地预测一般下降的斜率。至关重要的是，数据表明，尽管总体下降是可变的，但ALS的左肌肉下降之间存在高度的相关性。这意味着无论肌肉最快或最新肌肉开始下降，其发作后的下降速度更加一致。
首先，这项研究表明，当使用单方面治疗方法检测较小的患者组中疾病进展的放缓时，这表明了高度的功率，从而允许进行配对的统计检验。这些发现将在使用肌肉下降来跟踪ALS疾病进展的移植试验中很有用。其次，这些发现讨论了方法，例如肌肉群的战术选择，这些方法可以提高所有ALS临床试验的功率效率。
Amyotrophic lateral sclerosis (ALS) progresses at different rates between patients, making clinical trial design difficult and dependent on large cohorts of patients. Currently, there are few data showing whether the left and right limbs progress at the same or different rates. This study addresses rates of decline in specific muscle groups of patients with ALS and assesses whether there is a relationship between left and right muscles in the same patient, regardless of overall progression. A large cohort of patients was used to assess decline in muscle strength in right and left limbs over time using 2 different methods: The Tufts Quantitative Neuromuscular Exam and Accurate Test of Limb Isometric Strength protocol. Then advanced linear regression statistical methods were applied to assess progression rates in each limb. This report shows that linearized progression models can predict general slopes of decline with good accuracy. Critically, the data demonstrate that while overall decline is variable, there is a high degree of correlation between left and right muscle decline in ALS. This implies that irrespective of which muscle starts declining soonest or latest, their rates of decline following onset are more consistent. First, this study demonstrates a high degree of power when using unilateral treatment approaches to detect a slowing in disease progression in smaller groups of patients, thus allowing for paired statistical tests. These findings will be useful in transplantation trials that use muscle decline to track disease progression in ALS. Second, these findings discuss methods, such as tactical selection of muscle groups, which can improve the power efficiency of all ALS clinical trials.