Common data elements for clinical research in Friedreich's ataxia.
Common data elements for clinical research in Friedreich's ataxia.
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DOI：
10.1002/mds.25201
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发表时间：
2013-02
影响因子：
8.6
通讯作者：
Kaufmann, Petra
中科院分区：
文献类型：
--
作者：
Lynch, David R.;Pandolfo, Massimo;Schulz, Jorg B.;Perlman, Susan;Delatycki, Martin B.;Payne, R. Mark;Shaddy, Robert;Fischbeck, Kenneth H.;Farmer, Jennifer;Kantor, Paul;Raman, Subha V.;Hunegs, Lisa;Odenkirchen, Joanne;Miller, Kristy;Kaufmann, Petra
为了减少研究启动时间、增加数据共享并协助研究人员进行临床研究，国家神经疾病和中风研究所发起了一项举措，为神经科学临床研究创建通用数据元素。通用数据元素团队开发了一般通用数据元素，这些元素通常在临床研究中收集，无论治疗领域如何，例如人口统计。在本项目中，我们将这种方法应用于弗里德赖希共济失调（一种涉及多个器官系统的神经系统疾病）的数据收集。为了开发 Friedreich 的共济失调通用数据元素，Friedreich 的共济失调专家成立了一个工作组和小组来定义以下元素：共济失调和绩效衡量；生物标志物；心脏和其他临床结果；人口统计、实验室测试和病史。基本研发流程包括： 确定弗里德赖希共济失调临床研究的国际专家；通过电话会议制定标准化通用数据元素建议草案；审查各个小组的建议；向研究界传播建议以征询公众意见。完整的建议于 2011 年 9 月在线发布：http://www.commondataelements.ninds.nih.gov/FA.aspx。小组的建议分为核心、补充或探索性建议。为许多核心测试创建了模板案例报告表。理想情况下，当前的数据元素集应该会缩短临床研究的启动时间，并提高跨研究比较和分析数据的能力。将持续评估它们纳入新的和正在进行的研究，以确定它们在弗里德赖希共济失调中的效用。
To reduce study start-up time, increase data sharing, and assist investigators conducting clinical studies, the National Institute of Neurological Disorders and Stroke embarked on an initiative to create common data elements for neuroscience clinical research. The Common Data Element Team developed general common data elements which are commonly collected in clinical studies regardless of therapeutic area, such as demographics. In the present project, we applied such approaches to data collection in Friedreich ataxia, a neurological disorder that involves multiple organ systems. To develop Friedreich’s ataxia common data elements, Friedreich’s ataxia experts formed a working group and subgroups to define elements in: Ataxia and Performance Measures; Biomarkers; Cardiac and Other Clinical Outcomes; and Demographics, Laboratory Tests and Medical History. The basic development process included: Identification of international experts in Friedreich’s ataxia clinical research; Meeting via teleconference to develop a draft of standardized common data elements recommendations; Vetting of recommendations across the subgroups; Dissemination of recommendations to the research community for public comment. The full recommendations were published online in September 2011 at http://www.commondataelements.ninds.nih.gov/FA.aspx. The Subgroups’ recommendations are classified as core, supplemental or exploratory. Template case report forms were created for many of the core tests. The present set of data elements should ideally lead to decreased initiation time for clinical research studies and greater ability to compare and analyze data across studies. Their incorporation into new and ongoing studies will be assessed in an ongoing fashion to define their utility in Friedreich’s ataxia.
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Frataxin缺乏症的功能基因组分析揭示了组织特异性的改变，并将PPARGAMMA途径鉴定为弗里德里希共济失调的治疗靶标。
影响因子：
3.5
作者：
Coppola G;Marmolino D;Lu D;Wang Q;Cnop M;Rai M;Acquaviva F;Cocozza S;Pandolfo M;Geschwind DH
通讯作者：
Geschwind DH
影响因子：
4.2
作者：
Adelson, P. David;Pineda, Jose;Wainwright, Mark S.
通讯作者：
Wainwright, Mark S.
影响因子：
8.6
作者：
Buerk, Katrin;Maelzig, Ulrike;Schulz, Joerg B.
通讯作者：
Schulz, Joerg B.
影响因子：
3.7
作者：
Marmolino D;Manto M;Acquaviva F;Vergara P;Ravella A;Monticelli A;Pandolfo M
通讯作者：
Pandolfo M
影响因子：
9.9
作者：
FINOCCHIARO, G;BAIO, G;DIDONATO, S
通讯作者：
DIDONATO, S