Toward Identifying Clinically Useful Definitions of Treatment Success

 描述（由申请人提供）：酒精使用障碍（AUD）的治疗历来是通过基于消费的结果进行评估的，例如戒酒天数（PDA）的百分比。饮酒作为主要结果有许多优点，因为它很容易量化（例如，通过标准饮料），并且因为酒精消费与AUD固有地相关。然而，研究人员、客户和治疗提供者都同意，从AUD中恢复的过程不仅仅涉及消费（例如，Donovan等人，2005; Kaskutas等人，2014; Neale等人，2015）。鉴于主要通过消耗量定义治疗成功通常无法充分描述恢复过程（例如，Donovan等人，2012年;物质滥用和精神卫生服务管理局，2012年;蒂芙尼等人，2012年）。然而，由于两个主要原因，努力将非消费结果措施包括在内的努力受到了阻碍。首先，假设消耗量指标更敏感，因此，必须将消耗量用作更具临床意义的非消耗量指标的“替代”指标（FDA，2015，第2页）。其次，需要研究指导研究人员在AUD人群中使用最佳的“金标准”测量方法，并且这些测量方法随时间推移的测量不变性研究基本上不存在。拟议研究的目的将通过对在美国进行的两项最大的AUD治疗研究的二次数据分析来解决这两个问题：联合收割机研究和项目MATCH（Anton等人，2006年;项目匹配研究小组，1997年）。本研究将检查敏感性和特异性（目标1），以及可靠性，有效性和测量不变性（目标2），非消费措施，在联合收割机研究和项目匹配。要检查的非消费结果变量与研究人员，客户及其亲人和治疗提供者确定为重要的变量一致：1）酒精相关问题，2）心理健康，3）渴望/诱惑，4）心理社会功能/生活质量，5）身体健康，6）财务状况，7）自我效能。将使用接受者操作特征（ROC）曲线分析（汉利和麦克尼尔，1982）来评估灵敏度和特异性，预测治疗后即刻和治疗后12个月的结果数据：1）戒酒，2）无大量饮酒日（Falk等人，2010），3）世界卫生组织危险饮酒水平（欧洲药品管理局，2010），和4）消费和酒精相关问题定义的复合临床结果（Cisler & Zweben，1999）。将使用潜变量建模方法评估可靠性、有效性和测量不变性。本研究的两个目的将解决长期存在且未经检验的假设，这些假设阻止研究人员转向更有临床意义的治疗成功定义，包括非消耗性结局。

 DESCRIPTION (provided by applicant): Treatments for alcohol use disorder (AUD) have been historically evaluated by consumption-based outcomes, such as the percentage of days abstinent from alcohol (PDA). There are many advantages of alcohol consumption as a primary outcome, given that it is easily quantified (e.g., via standard drinks) and because alcohol consumption is inherently associated with AUD. However, researchers, clients, and treatment providers have all agreed that the process of recovering from AUD involves more than consumption alone (e.g., Donovan et al., 2005; Kaskutas et al., 2014; Neale et al., 2015). Recent efforts have called for a shift away from defining consumption as the marker of AUD treatment success (Witkiewitz, 2013a), given that defining treatment success primarily by amount of consumption often fails to adequately portray the recovery process (e.g., Donovan et al., 2012; Substance Abuse and Mental Health Services Administration, 2012; Tiffany et al., 2012). Yet, efforts to move toward including non-consumption outcome measures have been stymied for two primary reasons. First, it is assumed that consumption measures are more sensitive and, therefore, consumption must be used as a "surrogate" measure for more clinically meaningful non- consumption measures (FDA, 2015, p. 2). Second, research is needed to direct researchers toward the best "gold standard" measures to use among AUD populations and studies of measurement invariance over time of these measures are largely non-existent. The aims of the proposed study will address these two concerns via secondary data analysis of two of the largest AUD treatment research studies conducted in the United States: the COMBINE Study and Project MATCH (Anton et al., 2006; Project MATCH Research Group, 1997). The present study will examine the sensitivity and specificity (Aim 1), as well as the reliability, validity, and measurement invariance (Aim 2), of non-consumption measures available in the COMBINE study and Project MATCH. The non-consumption outcome variables to be examined are consistent with the variables identified as important to researchers, clients and their loved ones, and treatment providers: 1) alcohol-related problems, 2) mental health, 3) craving/temptation, 4) psychosocial functioning/quality of life, 5) physical health, 6) financial status, and 7) self-efficacy. Sensitivity and specificity will be evaluated using Receiver Operatin Characteristic (ROC) curve analyses (Hanley & McNeil, 1982) predicting outcome data from immediately post- and 12-month post-treatment for: 1) abstinence, 2) no heavy drinking days (Falk et al., 2010), 3) the World Health Organization risky drinking levels (European Medicines Agency, 2010), and 4) a composite clinical outcome defined by consumption and alcohol-related problems (Cisler & Zweben, 1999). Reliability, validity, and measurement invariance will be assessed using latent variable modeling approaches. The two aims of this study will address longstanding and untested assumptions that have prevented researchers from shifting toward more clinically meaningful definitions of treatment success that include non-consumption outcomes.