Aggregating single patient (n-of-1) trials in populations where recruitment and retention was difficult: The case of palliative care
Aggregating single patient (n-of-1) trials in populations where recruitment and retention was difficult: The case of palliative care
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DOI：
10.1016/j.jclinepi.2010.05.009
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发表时间：
2011-05-01
影响因子：
7.2
通讯作者：
Currow, David
中科院分区：
文献类型：
--
作者：
Nikles, Jane;Mitchell, Geoffrey K.;Currow, David
随机对照试验(RCT)是评估新干预措施的黄金标准。不同的随机对照试验设计适用于不同的患者群体、临床环境和正在评估的干预措施。一种可能有助于在招募是挑战的某些临床领域产生证据的设计是聚合n-of-1试验。N-of-1试验是干预和对照治疗的随机、双盲和多重交叉比较。方法学上稳健的n-of-1试验提供了一种在个体参与者中测试治疗效果的客观手段。多个周期相同进行的n-of-1试验的聚合产生了总体效应估计，这可能与其他随机对照试验设计得出的结果相称。试验参与者为干预和对照治疗贡献数据，创建匹配的数据集，同时使用通常比传统RCT试验更小的样本量。需要谨慎选择症状和药物才能使n-of-1试验可行。仍然需要对变化敏感的有效和可靠的结果测量。本文回顾了在人群中收集证据的聚集n-of-1试验的实用性和局限性，这些人群很难进行正式的随机对照试验，因为基础条件或临床条件的低患病率使得招募和保留困难。这篇文章审查了一个拟议的姑息治疗试验作为测试案例。(C)2011 Elsevier Inc.保留所有权利。
Randomized controlled trials (RCTs) are the gold standard for evaluating new interventions. Different RCT designs apply depending on the patient population, clinical setting, and intervention being evaluated. A design that may help to generate evidence in some clinical areas where recruitment is a challenge is aggregated n-of-1 trials.N-of-1 trials are randomized, double-blind, and multiple crossover comparisons of an intervention and a control treatment. Methodologically robust n-of-1 trials provide an objective means of testing the effectiveness of treatments within individual participants. Aggregation of multiple cycle identically conducted n-of-1 trials yield a population estimate of effect, which potentially commensurate with that derived from other RCT designs. Trial participants contribute data for both intervention and control treatments creating matched data sets while using generally smaller sample sizes than conventional RCT trials.Careful choice of symptoms and medications are required for n-of-1 trials to be feasible. A validated and reliable outcome measure sensitive to change is still required.This article reviews the utility and limitations of aggregated n-of-1 trials to gather evidence in populations where conducting formal RCTs is difficult because of the low prevalence of the underlying condition or the clinical condition making recruitment and retention difficult. The article examines a proposed palliative care trial as a test case. (C) 2011 Elsevier Inc. All rights reserved.