Discovering Tailoring Variables in Childhood Mental Health Treatment Research

描述(由申请人提供)：儿童精神疾病通常要求临床医生做出个性化的治疗决定。在儿童和青少年心理健康(CAMH)研究和实践的一个令人兴奋的趋势中，人们对制定个性化决策规则(IDR)越来越感兴趣和意识。IDR使用社会人口信息、病史、合并症、家庭/人际关系测量或生物标志物作为输入，然后建议后续治疗的类型、方式和强度或提供作为输出(S)。IDR为个性化医疗提供了一个框架：个性化程度与在IDR中用作输入的有用信息(定制变量)的数量成比例地增加。开发用于治疗精神疾病儿童的IDR是干预研究中重要的“下一步”。大规模的临床试验已经为许多常见的CAMH疾病建立了有效的干预措施。然而，尽管有强有力的证据表明治疗平均有效，但人们几乎不知道哪些儿童更有可能从一种或另一种治疗中受益。在IDR能够发挥其潜力之前，关于如何利用目前可用的数据来协助其发展，仍有许多方法学工作要做。制定有效的IDR的关键一步是确定调整变量以 用作输入。量身定制变量是一种特殊类型的前处理措施，可以精确地确定哪种治疗对谁最好。现有的方法没有明确地(A)识别哪些度量是最有用的(定制变量选择)，或者(B)以最佳方式组合它们(定制可变特征构造)。本研究的具体目的是：目标1(方法)。开发和评估一个框架，用于(A)定制变量选择和(B)定制用于个体化CAMH治疗的变量特征结构。新方法将用于解决科学问题，例如：(A)用于量身定制治疗的最重要的衡量标准(例如，人口、病情和病程、合并症、父母精神病、包括家庭和同伴关系在内的心理社会环境、生物学)是什么？(B)我们如何将它们结合起来，以指导治疗决定(例如，个别心理治疗、药物治疗或它们的组合)？目的2(应用)：通过将该方法应用于儿童/青少年焦虑症多模式研究的随机试验数据来说明和评价该方法，以构建用于选择SSRI、CBT或SSRI+CBT治疗焦虑症儿童的IDR。我们将应用新的方法来确定变量和特征，以确定哪些儿童或多或少可能受益于SSRI、CBT或联合SSRI+CBT。研究结果将被用于构建针对儿童焦虑症的建议IDR。我们将比较IDR、所有人的SSRI、所有人的CBT和所有人的SSRI+CBT的平均症状和严重程度结果的差异。这项研究将为CAMH的研究人员提供一种建立IDR的新工具。这项研究具有重大的公共卫生影响，因为它旨在开发工具，对患有精神健康障碍的儿童和青少年进行个性化治疗。这是国家精神卫生研究所的一个高度优先的研究领域。

DESCRIPTION (provided by applicant): Childhood psychiatric conditions often require that clinicians make individualized treatment decisions. In an exciting trend in child and adolescent mental health (CAMH) research and practice, there is growing interest and awareness in developing individualized decision rules (IDRs). IDRs use sociodemographic information, illness history, comorbidities, family/interpersonal measures, or biomarkers as inputs and then recommend the type, modality, and intensity or delivery of subsequent treatment(s) as outputs. IDRs provide a framework for personalized medicine: the level of personalization increases in proportion to the amount of useful information (tailoring variables) used as inputs in an IDR. The development of IDRs for the treatment of children with mental illness is an important "next step" in interventions research. Large scale clinical trials have established efficacious interventions fr many of the common CAMH disorders. However, despite a strong evidence base for treatments that are effective on average, little is known about which children are more or less likely to benefit from one treatment or another. Before IDRs can fulfill their potential, much methodological work remains to be done regarding how to use currently available data to assist in their development. A key step in developing effective IDRs is identifying tailoring variables to use as inputs. Tailoring variables are special types of pre-treatment measures that pinpoint which treatment is best for whom. Existing methods do not explicitly (a) identify which measures are most useful (tailoring variable selection), or (b) combine them in an optimal fashion (tailorin variable feature construction). The specific aims of this study are: Aim 1 (Methods). To develop and evaluate a framework for (a) tailoring variable selection and (b) tailoring variable feature construction for individualizing CAMH treatment. The new method will be used to address scientific questions such as: (a) What are the most important measures (e.g., demographic, severity and duration of illness, comorbidity, parental psychopathology, psychosocial environment including family and peer relationships, biological) to use for tailoring treatment? (b) How do we combine them in order to guide treatment decisions (e.g., individual psychotherapy treatment, medication, or their combination)? Aim 2 (Application): To illustrate and evaluate the method by applying it to the Child/Adolescent Anxiety Multimodal Study randomized trial data to construct an IDR for choosing between SSRI, CBT, or SSRI+CBT for children with anxiety disorders. We will apply the new methodology to identify variables and features pinpointing children who are more or less likely to benefit from SSRI, CBT, or combined SSRI+CBT. Results will be used to construct a proposed IDR for pediatric anxiety disorders. We will compare how average symptom and severity outcomes differ on the IDR vs SSRI for all vs CBT for all vs SSRI+CBT for all. This study will provide researchers in CAMH with a novel tool for building IDRs. This study has significant public health impact because it aims to develop tools to personalize treatment for children and adolescents with mental health disorders. This is a high-priority area of research for the National Institute of Mental Health.