The natural history of amyotrophic lateral sclerosis and the use of natural history controls in therapeutic trials
The natural history of amyotrophic lateral sclerosis and the use of natural history controls in therapeutic trials
复制标题
肌萎缩侧索硬化症的自然病程以及自然病程对照在治疗试验中的应用
DOI：
10.1212/wnl.43.4.751
复制
发表时间：
1993
期刊：
影响因子：
9.9
通讯作者：
T. Munsat
中科院分区：
文献类型：
--
作者：
J. Pradas;L. Finison;P. Andres;B. Thornell;D. Hollander;T. Munsat
我们分析了277例肌萎缩侧索硬化症患者的自然病程。我们的目标是更好地了解临床疾病，从而改善治疗试验的设计。Tufts定量神经肌肉检查（TQNE）被用作主要评估工具。我们的分析表明，虽然需要更多的观察，但6个月的TQNE足以确定疾病进展的速度。我们观察到一系列恶化率，没有可定义的亚组。证实了恶化的显著线性。我们发现个体患者手臂和腿部力量的恶化率之间存在高度相关性，但不同患者之间的相关性很大。临床发病时的性别和年龄不影响恶化率。与没有阳性家族史的患者相比，有其他受影响家庭成员的患者手臂力量下降较慢，但腿部力量没有下降。我们建议，自然史对照可以有效地用于ALS治疗试验的设计。
We analyzed the natural history of amyotrophic lateral sclerosis in 277 patients. Our goal was to develop a better understanding of the clinical disease and thus improve the design of therapeutic trials. The Tufts Quantitative Neuromuscular Exam (TQNE) was used as the primary assessment instrument. Our analysis suggested that although more observations are desirable, six monthly TQNEs were adequate to establish the rate of disease progression. We observed a spectrum of deterioration rates without definable subgroups. The striking linearity of deterioration was confirmed. We found a high correlation between deterioration rates in arm and leg strength for individual patients, but a wide range between different patients. Sex and the age at clinical onset did not affect the deterioration rate. As compared with patients without a positive family history, those with other affected family members had a slower loss in arm but not leg strength. We propose that natural history controls can be used effectively in the design of ALS therapeutic trials.