Understanding the Outcomes Measures used in Huntington Disease Pharmacological Trials: A Systematic Review.
Understanding the Outcomes Measures used in Huntington Disease Pharmacological Trials: A Systematic Review.
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DOI：
10.3233/jhd-140115
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发表时间：
2014
期刊：
影响因子：
--
通讯作者：
Dayalu P
中科院分区：
文献类型：
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作者：
Carlozzi NE;Miciura A;Migliore N;Dayalu P
导致亨廷顿病的基因突变的鉴定为缓解症状和减缓功能衰退的新疗法带来了希望。因此，一直在推动设计有效的药理学试验（即，药物试验），特别是在选择对整个疾病过程中的变化既简单又敏感的结果测量方面，从微妙的前驱变化到更严重的终末期变化。最近，为了帮助有效开发新的HD研究，国家神经疾病和卒中研究所（NINDS）发布了HD测量选择的建议。虽然这些建议是有帮助的，但许多建议的措施在HD中几乎没有公布的数据。因此，我们对文献进行了系统性综述，以确定HD临床试验中使用的最常见结局指标。主要的医学数据库，包括PubMed、Embase、CINAHL和对照试验的科克伦中心注册中心，用于识别2001年至2013年4月的英文同行评审期刊文章;识别了151项药理学试验。大多数HD临床试验采用临床医生报告的结局指标（93%）;患者报告的结局指标（11%）和观察者报告的结局指标（3%）的使用频率要低得多。我们对这些试验中最常用的措施进行了审查，将这些措施与NINDS工作组提出的临床建议进行了比较，并为未来临床试验选择符合食品和药物管理局标准的措施提供了建议。
The identification of the gene mutation causing Huntington disease has raised hopes for new treatments to ease symptoms and slow functional decline. As such, there has been a push towards designing efficient pharmacological trials (i.e., drug trials), especially with regard to selecting outcomes measures that are both brief and sensitive to changes across the course of the disease, from subtle prodromal changes, to more severe end-stage changes. Recently, to aid in efficient development of new HD research studies, the National Institute of Neurological Disorders and Stroke (NINDS) published recommendations for measurement selection in HD. While these recommendations are helpful, many of the recommended measures have little published data in HD. As such, we conducted a systematic review of the literature to identify the most common outcomes measures used in HD clinical trials. Major medical databases, including PubMed, Embase, CINAHL, and the Cochrane Central Register of Controlled Trials, were used to identify peer-reviewed journal articles in English from 2001 through April 2013; 151 pharmacological trials were identified. The majority of HD clinical trials employed clinician-reported outcomes measures (93%); patient reported outcome measures (11%) and observer reported outcome measures (3%) were used with much less frequency. We provide a review of the most commonly used measures across these trials, compare these measures to the clinical recommendations made by the NINDS working groups, and provide recommendations for selecting measures for future clinical trials that meet the Food and Drug Administration standards.