Yang Wei, Propert Kathleen J, Landis J Richard
Department of Biostatistics and Epidemiology, Center for Clinical Epidemiology and Biostatistics, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, U.S.A.
Stat Med. 2014 Sep 10;33(20):3547-55. doi: 10.1002/sim.5702. Epub 2012 Dec 10.
We are motivated by a randomized clinical trial evaluating the efficacy of amitriptyline for the treatment of interstitial cystitis and painful bladder syndrome in treatment-naïve patients. In the trial, both the non-adherence rate and the rate of loss to follow-up are fairly high. To estimate the effect of the treatment received on the outcome, we use the generalized structural mean model (GSMM), originally proposed to deal with non-adherence, to adjust for both non-adherence and loss to follow-up. In the model, loss to follow-up is handled by weighting the estimation equations for GSMM with one over the probability of not being lost to follow-up, estimated using a logistic regression model. We re-analyzed the data from the trial and found a possible benefit of amitriptyline when administered at a high-dose level.
我们的研究动机源于一项随机临床试验，该试验评估了阿米替林在初治患者中治疗间质性膀胱炎和膀胱疼痛综合征的疗效。在该试验中，不依从率和失访率都相当高。为了估计所接受治疗对结果的影响，我们使用最初为处理不依从情况而提出的广义结构均值模型（GSMM），对不依从和失访情况进行调整。在该模型中，通过用1除以未失访概率对GSMM的估计方程进行加权来处理失访情况，未失访概率使用逻辑回归模型进行估计。我们重新分析了该试验的数据，发现高剂量使用阿米替林可能有益。
