Profiling placebo responders by self-consistent partitioning of functional data
Profiling placebo responders by self-consistent partitioning of functional data
复制标题
DOI：
10.1198/016214503000000783
复制
发表时间：
2003-12-01
影响因子：
3.7
通讯作者：
Ogden, RT
中科院分区：
文献类型：
--
作者：
Tarpey, T;Petkova, E;Ogden, RT
在接受活性药物治疗的受试者中识别安慰剂应答者具有重要的临床和研究意义。在临床实践中，当患者接受药物治疗后病情有所改善时，这种改善可能归因于药物本身的化学成分，“安慰剂效应”或这些因素的某种组合。通过了解患者改善的机制，可以极大地帮助确定患者的适当后续治疗和维护。在研究背景下，患者反应的分类与如何设计和进行疗效和有效性临床试验有关。本文提出了一个框架，研究安慰剂反应在不同领域的医学。为了确定药物治疗患者中的安慰剂应答者，估计每例受试者随时间推移的临床状态特征（结局特征）。自洽分区技术用于基于曲线中的曲率量以及曲线中的总体趋势对受试者进行分组。所得分区确定药物组中受试者的代表性特征，随后可用于对患者进行分类。所提出的方法是适用于从临床试验的数据，用于治疗抑郁症，涉及安慰剂和活性药物苯乙肼。来自研究的安慰剂组的数据用于帮助验证该程序，因为药物治疗和安慰剂治疗的受试者应该具有共同的特征。
Identification of placebo responders among subjects treated with active drug has significant clinical and research implications. In clinical practice, when a patient treated with medication improves, this improvement may be attributed to the chemical component of the drug itself, a "placebo effect," or some combination of these. Determining the proper subsequent treatment and maintenance of the patient may be greatly aided by understanding the mechanism of patient improvement. In a research context, classification of patient response has bearing on how efficacy and effectiveness clinical trials are designed and conducted. This article presents a framework for studying placebo response in diverse areas of medicine. To identify placebo responders among drug-treated patients, a profile of the clinical status over time (outcome profile) is estimated for each subject. Self-consistent partitioning techniques are used to group subjects based on the amount of curvature in the profile as well as the overall trend in the profile. The resulting partitions determine representative profiles for subjects in the drug group that subsequently can be used to classify patients. The proposed method is applied to data from a clinical trial for treatment of depression involving placebo and the active drug phenelzine. Data from the placebo arm of the study is used to help validate the procedure, because the drug-treated and placebo-treated subjects should share common profiles.