Pradas J, Finison L, Andres P L, Thornell B, Hollander D, Munsat T L
Department of Neurology, New England Medical Center, Tufts University, Boston, MA.
Neurology. 1993 Apr;43(4):751-5. doi: 10.1212/wnl.43.4.751.
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.
我们分析了277例肌萎缩侧索硬化症患者的疾病自然史。我们的目标是更好地了解这种临床疾病，从而改进治疗试验的设计。塔夫茨定量神经肌肉检查（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数据，但每六个月进行一次TQNE足以确定疾病进展速度。我们观察到一系列恶化速度，未发现可明确划分的亚组。恶化的显著线性关系得到了证实。我们发现，个体患者手臂和腿部力量的恶化速度之间存在高度相关性，但不同患者之间的差异很大。性别和临床发病年龄不影响恶化速度。与无阳性家族史的患者相比，有其他家庭成员患病的患者手臂力量丧失较慢，但腿部力量丧失情况并非如此。我们建议，疾病自然史对照可有效地用于肌萎缩侧索硬化症治疗试验的设计。
