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干眼临床试验中安慰剂作用的预测因素:三项临床试验的汇总分析 Predictive factors for the placebo effect in clinical trials for dry eye: a pooled analysis of three clinical trials

Imanaka, T., Sato, I., Tanaka, S., Kawakami, K.

期刊名称:The British journal of ophthalmology



摘要:背景:安慰剂效应是干眼临床试验中方法学难题中的一种。如果我们能够阐明安慰剂反应的趋势并找到预测指标,那么我们就可以减少干眼症临床试验中的安慰剂反应。在这项研究中,我们调查了干眼临床试验中安慰剂效应的预测因素。方法:通过简单和多变量回归分析,共对分配到三个安慰剂对照随机临床试验安慰剂组的205例干眼患者进行了研究。在第4周时,研究了角膜荧光素(FL)染色评分和干眼症状。所关注的变量包括性别,年龄,Sjgren综合征并发症,Schirmer's测试I值,泪膜破裂时间和结膜充血评分。我们还根据患者的年龄进行了分层分析。结果:在所有研究终点中,基线评分与相应的安慰剂反应显着相关。此外,对于FL评分和干燥评分,年龄是安慰剂反应的显着预测因子(分别为p = 0.04p <0.0001)。年龄分层分析显示, 40岁以上的患者的FL和干燥评分可能具有更强的安慰剂反应。结论:常用的终点指标中,基线评分和年龄是安慰剂反应的预测因素,例如FL评分或干燥症状。这些患者的特征可以通过研究设计来控制,并且我们的发现使得能够设计更有效的安慰剂对照研究,并具有良好的统计效力。

BACKGROUND:Placebo effect is one of the methodological difficulties in dry eye clinical trials. If we could elucidate the tendencies of the placebo response and find predictors, we could reduce the placebo response in clinical trials for dry eye. In this study, we investigated the predictive factors for the placebo effect in dry eye clinical trials.METHODS:A total of 205 patients with dry eye assigned to the placebo arms of three placebo-controlled randomised clinical trials were analysed by simple and multivariable regression analysis. The corneal fluorescein (FL) staining score and dry eye symptoms were studied at week 4. The variables of interest included gender, age, complications of Sjgren's syndrome, Schirmer's test I value, tear break-up time and conjunctival hyperaemia score. We also conducted a stratified analysis according to the patients' age.RESULTS:Among all the studied endpoints, the baseline scores were significantly related to the corresponding placebo response. In addition, for the FL score and the dryness score, age was a significant predictor of the placebo response (p=0.04 and p<0.0001, respectively). Stratified analysis by age showed that patients more than 40 years of age are more likely to have a stronger placebo response in the FL and dryness scores.CONCLUSION:The baseline scores and age were predictive factors of the placebo response in frequently used endpoints, such as FL score or dryness symptoms. These patient characteristics can be controlled by study design, and our findings enable the design of more efficient placebo-controlled studies with good statistical power.


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