Transforming Traumatic Brain Injury Research and Clinical Care

描述(由申请人提供)：创伤性脑损伤(TBI)仍然是医学和公共卫生领域最大的未得到满足的需求之一。在过去的20年里，基础科学研究的进步为脑外伤的靶向治疗创造了新的机会。然而，这些进展未能转化为成功的临床试验或治疗脑损伤的新方法。最近由NINDS、NIDRR、DVBIC和DCOE主办的两个研讨会确定了翻译失败的重要原因，包括数据收集缺乏标准化、对TBI分类和结果的方法过时。人们一致认为，脑损伤研究中变量的选择应该在不同的研究中标准化，并强烈建议开发新的和改进的脑损伤分类方法。还建议应用新兴技术。在广泛机构的支持下，一个由思想领袖组成的多学科小组被聚集在一起，起草了一份关于跨TBI研究的数据收集标准化的提案，重点是人口统计学、神经成像、结果测量、生物标记物和心理健康，称为TBI公共数据元素(TBI-CDE)。这现在为所有未来的TBI研究提供了一个潜在的框架，但人们承认，为了实施和广泛采用TBI-CDE，需要一种基于网络的数据输入格式，并在临床实践中进一步完善和测试测试版。这项提案的总体目标是测试和完善适用于各种类型的脑损伤研究的数据收集标准(目标1)，并利用新兴技术探索脑损伤分类(目标2)和脑损伤后结果(目标3)的新方法。此外，我们的目标是制定一套试行的绩效指标，用于评估TBI的医疗质量和有效性(目标4)。测试和验证TBI-CDE将在3个TBI中心(UCSF、UPMC、UT Houston)和1个TBI康复中心(西奈山)的多中心观察性研究中进行。这些中心拥有多中心的TBI研究经验和现有的基础设施，可以快速、成功地完成研究。我们将创建或扩展现有的患者人口统计学、神经成像、血清生物标记物和基因组学数据库，从而为研究人员和临床医生提供基础设施，以在协调一致的多学科努力中发展脑损伤领域。该项目旨在开发基础设施、协作和工具。它将建立跨学科的桥梁，打破阻碍进展的人工构造和障碍，并促进跨研究的个体患者数据的比较和荟萃分析。它代表了迄今为止最具综合性的TBI努力之一。一个独特的特点是，它将跨越从轻度到重度，从早期到晚期，从婴儿期到老年人的整个脑损伤范围。将首次探讨制定TBI具体绩效指标的问题。综合来看，该项目的成果有可能改变TBI的研究和临床护理。 公共卫生相关性：创伤性脑损伤(TBI)仍然是医学和公共卫生领域最大的未得到满足的需求之一。这项建议的总体目标是测试和完善脑损伤研究中的数据收集标准，以便共同改进分类、结果评估和医疗质量和有效性研究，该项目的交付成果具有改变脑损伤研究和临床护理的潜力。

DESCRIPTION (provided by applicant): Traumatic Brain Injury (TBI) remains one of the greatest unmet needs in medicine and public health. Advances in basic science research in the past 20 years have created new opportunities for targeted therapies for TBI. However, these advances have failed to translate into a successful clinical trial or a new treatment for TBI. Two recent workshops sponsored by NINDS, NIDRR, DVBIC, and DCOE identified important reasons for the failure in translation that include lack of standardisation in data collection, outdated approaches to TBI classification and outcome. There was consensus that the selection of variables in TBI studies should be standardized across studies and a strong recommendation was made to develop novel and improved approaches to TBI classification. The application of emerging technologies was also recommended. With broad-agency support, a multidisciplinary group of thought leaders was brought together to draft a proposal for standardisation of data collection across TBI studies with an emphasis on demographics, neuroimaging, outcome measures, biomarkers, and psychological health, referred to as the TBI Common Data Elements (TBI-CDE). This now provides a potential framework for all future TBI research, but it was acknowledged that a web-based data entry format and further refinement and testing of the beta-version in clinical practice would be required for implementation and widespread adoption of the TBI-CDE. The global aim of this proposal is to test and refine standards for data collection in TBI studies, suitable for use across the broad spectrum of TBI (Aim 1), and to explore novel approaches for TBI classification (Aim 2) and outcome after TBI (Aim 3), making use of emerging technology. In addition, we aim to develop a pilot set of performance indicators for assessment of the health care quality and effectiveness in TBI (Aim 4). Testing and validating the TBI-CDE will be performed in a multicenter observational study with 3 TBI Centers (UCSF, UPMC, UT Houston) and a TBI Rehabilitation Center (Mount Sinai). These Centers have multicenter TBI research experience and existing infrastructure to rapidly and successfully complete the study. We will create, or expand existing, data repositories for patient demographics, neuroimaging, serum biomarkers and genomics thereby providing researchers and clinicians with the infrastructure to develop the TBI field in a concerted multidisciplinary effort. The project is aimed at developing infrastructure, collaborations, and tools. It will build bridges across disciplines, break down artificial constructs and barriers that have slowed progress and facilitate comparison and meta-analyses of individual patient data across studies. It represents one of the most integrative TBI efforts to date. A unique feature is that it will span the entire spectrum of TBI from mild to severe, from early to late presentation and from infancy to the age of seniors. For the first time, the development of TBI specific performance indicators will be explored. Taken in combination, the deliverables of this project have the potential to transform TBI research and clinical care. PUBLIC HEALTH RELEVANCE: Traumatic Brain Injury (TBI) remains one of the greatest unmet needs in medicine and public health. The overall goal of this proposal is to test and refine standards for data collection in TBI studies in order to improve classification, outcome assessment, and healthcare quality and effectiveness research Together, the deliverables of this project have the potential to transform TBI research and clinical care.