Simultaneous evaluation of the harms and benefits of treatments in randomized clinical trials: demonstration of a new approach.
Simultaneous evaluation of the harms and benefits of treatments in randomized clinical trials: demonstration of a new approach.
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在随机临床试验中同时评估治疗的危害和益处：新方法的演示。
DOI：
10.1017/s0033291711001619
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发表时间：
2012-04
影响因子：
6.9
通讯作者：
Kraemer, H. C.
中科院分区：
文献类型：
--
作者：
Frank, E.;Kupfer, D. J.;Rucci, P.;Lotz-Wallace, M.;Levenson, J.;Fournier, J.;Kraemer, H. C.
个性化医疗的一个目标是，在提供所有患者信息的情况下，确定对单个患者首选哪种治疗。然而，特别是在精神健康方面，缺乏一种对患者之间关键的个体差异敏感的单一客观、可靠的结果衡量标准。我们研究了在单一指标中量化一种治疗提供的总临床价值(以危害和益处衡量)的可行性。一个专家小组被要求比较100对患者，每个治疗组一对，这些患者参加了一项涉及人际心理治疗(IPT)和艾司匹兰的随机临床试验(RCT)，选择既考虑益处又考虑危害的首选结果的患者。根据这些结果，得出了一个综合偏好评分(IPS)，因此任何两个患者的IPSS之间的差异可以预测临床医生的偏好。然后为RCT中的所有患者计算这一IPS。评审团对第二组100双鞋进行了评级。他们的偏好与IPS差异高度相关(r=0.84)。最后，将IPT与艾司匹兰进行比较，以IPS作为疗效评价指标。疗效大小比较治疗的95%可信区间(CI)表明两种治疗的临床等效性。结合治疗的益处和危害的衡量标准可以通过更清楚地说明哪些治疗方案更可取，以及最终对谁更有利，从而增加随机对照试验的价值。这种方法可以在不增加所需样本量的情况下，更准确地估计效果大小。
One aim of personalized medicine is to determine which treatment is to be preferred for an individual patient, given all patient information available. Particularly in mental health, however, there is a lack of a single objective, reliable measure of outcome that is sensitive to crucial individual differences among patients. We examined the feasibility of quantifying the total clinical value provided by a treatment (measured by both harms and benefits) in a single metric. An expert panel was asked to compare 100 pairs of patients, one from each treatment group, who had participated in a randomized clinical trial (RCT) involving interpersonal psychotherapy (IPT) and escitalopram, selecting the patient with the preferred outcome considering both benefits and harms. From these results, an integrated preference score (IPS) was derived, such that the differences between any two patients’ IPSs would predict the clinicians’ preferences. This IPS was then computed for all patients in the RCT. A second set of 100 pairs was rated by the panel. Their preferences were highly correlated with the IPS differences (r=0.84). Finally, the IPS was used as the outcome measure comparing IPT and escitalopram. The 95% confidence interval (CI) for the effect size comparing treatments indicated clinical equivalence of the treatments. A metric that combines benefits and harms of treatments could increase the value of RCTs by making clearer which treatments are preferable and, ultimately, for whom. Such methods result in more precise estimation of effect sizes, without increasing the required sample size.