Comparison of relative change with effect size metrics in Alzheimer's disease clinical trials.
Comparison of relative change with effect size metrics in Alzheimer's disease clinical trials.
复制标题
阿尔茨海默病临床试验中相对变化与效应大小指标的比较。
DOI：
10.1136/jnnp-2023-331941
复制
发表时间：
2023
期刊：
影响因子：
--
通讯作者：
Schneider,LonS
中科院分区：
文献类型：
--
作者：
Goldberg,TerryE;Lee,Seonjoo;Devanand,DavangereP;Schneider,LonS
在阿尔茨海默病（AD）临床试验中，经常使用下降减缓百分比作为结果的度量，但它可能具有误导性。我们的目标是确定是否下降或科恩的D是更有效和翔实的措施efficacy.MethodsOutcome的措施的兴趣下降的百分比放缓;科恩的D效应大小和数量需要治疗（NNT）。来自图形的数据用于模拟Cohen d、安慰剂原始评分单位下降和活性治疗下降减缓百分比之间的相互关系。NNT是基于d的不同幅度计算的。最后，我们列出了最近的AD抗淀粉样蛋白的临床试验，报告百分比放缓，我们计算各自的d的和NNTs.ResultsWe证明，d和百分比放缓是潜在的独立。虽然下降的减缓百分比取决于安慰剂下降，并且在其计算中不包括方差，但d取决于组平均差异和合并SD。我们接下来证明了d是NNT的一个关键决定因素，使得当d较大时，NNT一致较小。在最近的AD相关试验中，包括那些专注于抗淀粉样蛋白生物制剂的试验，d低于0.23，因此被认为是小的，而百分比减缓在22-29%范围内，NNT范围从14到18。结论标准化效应量是比百分比减缓下降更有意义的结果，因为它确定了组重叠，这可以直接影响NNT计算，并产生关于最小临床重要差异的可能性的信息。在AD中，更多地使用效应量，NNT，而不是相对百分比减缓，将提高解释临床试验结果和评估统计学显著结果的临床意义的能力。
BackgroundPer cent slowing of decline is frequently used as a metric of outcome in Alzheimer’s disease (AD) clinical trials, but it may be misleading. Our objective was to determine whether per cent slowing of decline or Cohen’s d is the more valid and informative measure of efficacy.MethodsOutcome measures of interest were per cent slowing of decline; Cohen’s d effect size and number-needed-to-treat (NNT). Data from a graphic were used to model the inter-relationships among Cohen’s d, placebo decline in raw score units and per cent slowing of decline with active treatment. NNTs were computed based on different magnitudes of d. Last, we tabulated recent AD anti-amyloid clinical trials that reported per cent slowing and for which we computed their respective d’s and NNTs.ResultsWe demonstrated that d and per cent slowing were potentially independent. While per cent slowing of decline was dependent on placebo decline and did not include variance in its computation, d was dependent on both group mean difference and pooled SD. We next showed that d was a critical determinant of NNT, such that NNT was uniformly smaller when d was larger. In recent AD associated trials including those focused on anti-amyloid biologics, d’s were below 0.23 and thus considered small, while per cent slowing was in the 22–29% range and NNTs ranged from 14 to 18.ConclusionsStandardised effect size is a more meaningful outcome than per cent slowing of decline because it determines group overlap, which can directly influence NNT computations, and yield information on the likelihood of minimum clinically important differences. In AD, greater use of effect sizes, NNTs, rather than relative per cent slowing, will improve the ability to interpret clinical trial results and evaluate the clinical meaningfulness of statistically significant results.
登录
查看更多内容
影响因子：
11.8
作者：
MacArthur, Rodger D.;DuPont, Herbert L.
通讯作者：
DuPont, Herbert L.
评估引起艾滋病毒/艾滋病患者胃肠道感染的寄生虫。
影响因子：
1.7
作者：
Santosh Kotgire;N. Tankhiwale;S. Tankhiwale;Abhijit De;D. Das
通讯作者：
D. Das
印度南部艾滋病毒感染者腹泻中等孢子虫病的比例很高。
影响因子：
3.3
作者：
R. Vignesh;P. Balakrishnan;E. Shankar;K. Murugavel;S. Hanas;A. Cecelia;S. Thyagarajan;S. Solomon;N. Kumarasamy
通讯作者：
N. Kumarasamy
肠结核是人类免疫缺陷病毒感染患者慢性腹泻的原因：两例报告。
影响因子：
4.5
作者：
S. Antinori;L. Galimberti;F. Parente
通讯作者：
F. Parente