Almirall Daniel, Compton Scott N, Rynn Moira A, Walkup John T, Murphy Susan A
1 Survey Research Center, Institute for Social Research, University of Michigan , Ann Arbor, Michigan.
J Child Adolesc Psychopharmacol. 2012 Oct;22(5):364-74. doi: 10.1089/cap.2011.0073.
Developing evidenced-based practices for the management of childhood psychiatric disorders requires research studies that address how to treat children during both the acute phase of the disorder and beyond. Given the selection of a medication for acute treatment, discontinuation trials are used to evaluate the effects of treatment duration (e.g., time on medication) and/or maintenance strategies following successful acute-phase treatment. Recently, sequential multiple assignment randomized trials (SMART) have been proposed for use in informing sequences of critical clinical decisions such as those mentioned. The objective of this article is to illustrate how a SMART study is related to the standard discontinuation trial design, while addressing additional clinically important questions with similar trial resources.
The recently completed Child/Adolescent Anxiety Multimodal Study (CAMS), a randomized trial that examined the relative efficacy of three acute-phase treatments for pediatric anxiety disorders, along with a next logical step, a standard discontinuation trial design, is used to clarify the ideas. This example is used to compare the discontinuation trial design relative to the SMART design.
We find that the standard discontinuation trial can be modified slightly using a SMART design to yield high-quality data that can be used to address a wider variety of questions in addition to the impact of treatment duration. We discuss how this innovative trial design is ultimately more efficient and less costly than the standard discontinuation trial, and may result in more representative comparisons between treatments.
Mental health researchers who are interested in addressing questions concerning the effects of continued treatment (for different durations) following successful acute-phase treatment should consider SMART designs in place of discontinuation trial designs in their research. SMART designs can be used to address these and other questions concerning individualized sequences of treatment, such as the choice of a rescue treatment in case of postacute phase relapse.
制定基于证据的儿童精神障碍管理方法需要开展研究，以探讨在疾病急性期及之后如何治疗儿童。在选择急性治疗药物后，停药试验用于评估治疗持续时间（如服药时间）的效果和/或急性期治疗成功后的维持策略。最近，有人提议使用序贯多重分配随机试验（SMART）来为关键临床决策序列提供信息，比如上述决策。本文的目的是说明SMART研究与标准停药试验设计的关系，同时利用相似的试验资源解决其他临床上重要的问题。
最近完成的儿童/青少年焦虑多模式研究（CAMS）是一项随机试验，该试验检验了三种儿童焦虑症急性期治疗方法的相对疗效，以及接下来合理的步骤——标准停药试验设计，以此来阐明观点。这个例子用于比较停药试验设计与SMART设计。
我们发现，使用SMART设计可以对标准停药试验进行轻微修改，从而产生高质量的数据，这些数据除了可用于解决治疗持续时间的影响外，还能用于解决更广泛的问题。我们讨论了这种创新的试验设计最终如何比标准停药试验更高效、成本更低，并且可能在治疗之间产生更具代表性的比较。
对解决有关急性期治疗成功后持续治疗（不同持续时间）效果问题感兴趣的心理健康研究人员，在其研究中应考虑采用SMART设计而非停药试验设计。SMART设计可用于解决这些以及其他有关个体化治疗序列的问题，例如急性期后复发时抢救治疗方法的选择。
