本帖最后由 菡萏尘雾 于 2012-12-3 12:22 编辑
Why did the high-dose group have an effect andnot the low-dose group?
为什么高剂量组有效而低剂量组没有？
Understanding how/how much to dose drugs in people is a key part of theclinical trial process. Typically this requires that multiple doses be tried inorder to find the optimal dosing stratagem, especially for novel drugs such asNP001. It is not surprising that one dose had a different effect from the otherin this trial. In fact, it is encouraging as it may be leading this company tothe optimal dose for this drug that could be used in the Phase III clinicaltria.
了解药物的剂量是临床实验过程的关键部分。有代表性的是需要实验多重剂量以找到最佳的剂量策略，特别是对于像NP001这种新药。在实验中发现某一剂量与其他剂量的疗效不同并不奇怪。事实上，如果该公司能找到最佳剂量并用于3期临床实验是令人鼓舞的。
Why did some people respond and others didn’t?
为什么有些病人有反应而其他的没有？
This is a clear example of the heterogeneity of this disease. Every caseof ALS is different in site of onset and progression rate. It is not surprisingthat some patients responded while others didn’t to NP001 at either dose. Ithas long been thoughts and now generally accepted that it will take a number oftherapeutics to cover the vast array of ALS cases that exist. Similar tomultiple sclerosis, where there are nine approved compounds for that diseasebut it is common that a patient will respond to only one or two of them.
这是该病异质性的一个明显例子。每个ALS病例的发病情况和发展速度都是不同的。所以一些病人对NP001反应良好而其他病人对任一剂量都反应不好就不奇怪了。经过长时间探讨现在被广泛接受的是：需要一系列的疗法才能涵盖大量存在的ALS病例。与多发性硬化类似的是，虽然有9种药物获批，但一般病人只对其中的一两种有反应。
This is likely to be the similar case for ALS andthe results from this trial highlights that likelihood. Understanding which patients are likely to respond to each availabletreatment that emerges for ALS will be a key endeavor and we encourage allthose operating clinical trials to think about potential biomarkers that can beused to help identify the most likely treatment responders for each.
对于ALS这可能是类似的，结果从这突显出来。认识到哪种ALS病人对哪种治疗手段更可能反应良好是努力的关键点。我们鼓励所有正在进行的临床实验考虑潜在的生物标记物，以帮助我们确定病人对疗法的反应。
If NP001 missed “statistical significance” whyis it moving forward into a Phase III?
如果NP001缺少“统计学显著性”，为何还要推进3期实验？
There can be no doubt that this trial and its result is significant tothe ALS community. However, this trial was powered and designed to primarilyaddress whether or not NP001 was safe and tolerable in ALS patients, notefficacy from a purely statistical perspective. It is not uncommon for trialsto move forward when there is a trend toward significance only.
毫无疑问的是，该实验及其结果对ALS群体是有意义的。但是，该实验（2期）的立足点和设计初衷主要是为了验证NP001是否安全和可耐受，而非纯粹统计学上的疗效。即使只有某种趋势上的意义，推进继续实验也并不罕见。
楼主: 兰花粥
狗仔卡
发表于 2012-11-29 12:34:33
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