Qureshi Muddasir, Schoenfeld David A, Paliwal Yuvika, Shui Amy, Cudkowicz Merit E
Neurology Clinical Trials Unit, Massachusetts General Hospital, Harvard Medical School, 13th Street, Boston, MA 02129, USA.
Amyotroph Lateral Scler. 2009 Oct-Dec;10(5-6):324-31. doi: 10.3109/17482960903009054.
In the past two decades new practice parameters for clinical care in ALS were developed and several clinical trials were performed. We sought to review information in these prospective datasets and assess whether natural history of ALS has changed over time. Survival and the rate of functional decline were compared across the placebo arms of efficacy trials conducted from 1999 to 2005 by the Northeast ALS Consortium (NEALS). Similar data from the placebo arms of 12 other published efficacy ALS trials conducted between 1990 and 2008 were compared descriptively. In the three NEALS clinical trials, survival improved over time in the placebo cohort (p=0.05) while the rate of change in maximum voluntary isometric contraction (MVIC) (p=0.15), ALS Functional Rating Scale (ALSFRS) (p=0.6) and vital capacity (%VC) (p=0.5) was unchanged. No differences were observed in the mean rates of decline of these outcome measures in the published clinical trials. However, survival improved in the more recently conducted trials. Survival improved over time in placebo controlled participants enrolled in clinical trials in ALS since 1990. However, the decline in measures of function appears unchanged since then. These changes in natural history reflect improvements in symptomatic care of ALS.
在过去二十年中，制定了肌萎缩侧索硬化症（ALS）临床护理的新实践参数，并开展了多项临床试验。我们试图回顾这些前瞻性数据集中的信息，并评估ALS的自然病程是否随时间发生了变化。比较了东北ALS联盟（NEALS）在1999年至2005年进行的疗效试验中安慰剂组的生存率和功能衰退率。对1990年至2008年期间其他12项已发表的ALS疗效试验安慰剂组的类似数据进行了描述性比较。在NEALS的三项临床试验中，安慰剂组的生存率随时间有所提高（p = 0.05），而最大随意等长收缩（MVIC）的变化率（p = 0.15）、ALS功能评定量表（ALSFRS）（p = 0.6）和肺活量（%VC）（p = 0.5）没有变化。在已发表的临床试验中，这些结局指标的平均衰退率没有观察到差异。然而，在最近进行的试验中生存率有所提高。自1990年以来，参加ALS临床试验的安慰剂对照参与者的生存率随时间有所提高。然而，从那时起，功能指标的下降似乎没有变化。自然病程的这些变化反映了ALS症状性护理的改善。
