进行性核上性麻痹未来临床试验的功效计算与安慰剂效应
Power calculations and placebo effect for future clinical trials in progressive supranuclear palsy.
作者信息
Stamelou Maria, Schöpe Jakob, Wagenpfeil Stefan, Del Ser Teodoro, Bang Jee, Lobach Iryna Y, Luong Phi, Respondek Gesine, Oertel Wolfgang H, Boxer AdamL, Höglinger Günter U
机构信息
Department of Neurology, Philipps University, Marburg, Germany.
Second Department of Neurology, Attikon University Hospital, University of Athens, Athens, Greece.
出版信息
Mov Disord. 2016 May;31(5):742-7. doi: 10.1002/mds.26580. Epub 2016 Mar 7.
BACKGROUND
Two recent randomized, placebo-controlled trials of putative disease-modifying agents (davunetide, tideglusib) in progressive supranuclear palsy (PSP) failed to show efficacy, but generated data relevant for future trials.
METHODS
We provide sample size calculations based on data collected in 187 PSP patients assigned to placebo in these trials. A placebo effect was calculated.
RESULTS
The total PSP-Rating Scale required the least number of patients per group (N = 51) to detect a 50% change in the 1-year progression and 39 when including patients with ≤ 5 years disease duration. The Schwab and England Activities of Daily Living required 70 patients per group and was highly correlated with the PSP-Rating Scale. A placebo effect was not detected in these scales.
CONCLUSIONS
We propose the 1-year PSP-Rating Scale score change as the single primary readout in clinical neuroprotective or disease-modifying trials. The Schwab and England Activities of Daily Living could be used as a secondary outcome. © 2016 International Parkinson and Movement Disorder Society.
背景
最近两项针对进行性核上性麻痹（PSP）的假定疾病修饰药物（达武奈肽、替格列净）的随机、安慰剂对照试验未能显示出疗效，但产生了与未来试验相关的数据。
方法
我们根据在这些试验中分配到安慰剂组的187例PSP患者收集的数据进行样本量计算。计算了安慰剂效应。
结果
总PSP评定量表每组检测1年病情进展50%变化所需患者数量最少（N = 51），若纳入病程≤5年的患者则每组需要39例。施瓦布和英格兰日常生活活动量表每组需要70例患者，且与PSP评定量表高度相关。在这些量表中未检测到安慰剂效应。
结论
我们建议将1年PSP评定量表评分变化作为临床神经保护或疾病修饰试验的单一主要指标。施瓦布和英格兰日常生活活动量表可作为次要结局指标。© 2016国际帕金森病和运动障碍协会。
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