Shortreed Susan M, Moodie Erica E M
McGill University, School of Computer Science; Group Health Research Institute, Biostatistics Unit.
J R Stat Soc Ser C Appl Stat. 2012 Aug 1;61(4):577-599. doi: 10.1111/j.1467-9876.2012.01041.x. Epub 2012 May 31.
Treatment of schizophrenia is notoriously difficult and typically requires personalized adaption of treatment due to lack of efficacy of treatment, poor adherence, or intolerable side effects. The Clinical Antipsychotic Trials in Intervention Effectiveness (CATIE) Schizophrenia Study is a sequential multiple assignment randomized trial comparing the typical antipsychotic medication, perphenazine, to several newer atypical antipsychotics. This paper describes the marginal structural modeling method for estimating optimal dynamic treatment regimes and applies the approach to the CATIE Schizophrenia Study. Missing data and valid estimation of confidence intervals are also addressed.
精神分裂症的治疗 notoriously difficult，由于治疗效果不佳、依从性差或副作用难以忍受，通常需要进行个性化的治疗调整。干预有效性临床抗精神病药物试验（CATIE）精神分裂症研究是一项序贯多重分配随机试验，将典型抗精神病药物奋乃静与几种新型非典型抗精神病药物进行比较。本文描述了用于估计最佳动态治疗方案的边际结构建模方法，并将该方法应用于CATIE精神分裂症研究。还讨论了缺失数据和置信区间的有效估计问题。
