Component C - The Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) in Utah

组件 C - 犹他州肌营养不良症监测、跟踪和研究网络 (MD STARnet)

• 批准号: 10220733
• 负责人: Marcia Lynn Feldkamp
• 金额: $ 8万
• 依托单位: UNIVERSITY OF UTAH
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-09-01 至 2024-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10220733
• 关键词: Component; Muscular; Dystrophy; Surveillance; Tracking

PROJECT SUMMARY/ABSTRACT The muscular dystrophies (MD) are disorders of muscle leading to progressive muscle weakness, and reduced quality of life and mortality due to respiratory and cardiac complications. In addition to significant motor, respiratory, and cardiac complications, affected individuals and families have social, psychological, and cognitive issues which are not well understood. For the MD STARnet project, nine muscular dystrophies are included: Duchenne and Becker muscular dystrophy (DBMD), distal, Emery-Dreifuss, limb-girdle, oculopharyngeal, congenital, myotonic dystrophy, and facioscapulohumeral dystrophy. Recent development and implementation of standardized care guidelines for DBMD, and other neuromuscular conditions are expected to result in improved clinical outcomes; however, published evidence remains limited. Emerging therapeutic options for these disorders are a source of great hope and opportunity for realizing improved outcomes; however, a better understanding of the range of disease severity and current care burden in these disorders is critical to the success of such initiatives. The University of Utah MD STARnet team has been active during the current funding cycle (DD001108: 9/1/2014-8/31/2019) and is uniquely situated to continue to conduct research as part of Component C. As part of Component A, our engagement with local and national neuromuscular foundations has been important to determine questions that our research can answer in order to address gaps in knowledge. For Component C, we will focus on the objectives and approach outlined in the funding opportunity announcement with an overall goal to continue our existing projects with proposed new research projects using MD STARnet data. Furthermore, we plan to conduct surveys to evaluate the psychological and neurobehavioral function, and health-related quality of life in patients with MDs. We will disseminate our findings through local, national, and international presentations at professional society meetings and submit manuscripts to peer-reviewed journals. Our existing program’s strengths and resources will increase knowledge of the medical issues and barriers to care facing these individuals, and enhance our ability to provide more consistent quality care to our patients. Furthermore, we propose that this project will allow us to assess the impact of implementation of standardized care guidelines on clinical outcomes and cost of care for such patients. We will analyze high-priority questions addressed in the MD STARnet Research Agenda (Aim 1), identify additional gaps in understanding with a focus on psychological and neurobehavioral comorbidities and health-related quality of life (Aim 2), and provide secondary analyst support for other MD STARnet sites on a minimum of three research projects (Aim 3).

项目摘要/摘要 肌营养不良症(MD)是一种肌肉疾病，导致进行性肌肉无力，并减少 生活质量和呼吸系统和心脏并发症的死亡率。除了重要的马达， 呼吸道和心脏并发症，受影响的个人和家庭有社会、心理和 没有被很好理解的认知问题。对于MD STARnet项目，九种肌肉营养不良症是 包括：Duchenne和Becker肌营养不良症(DBMD)，远端肌营养不良症，Emery-Dreifuss，肢体带， 眼咽部、先天性、强直性肌营养不良和面肩肩关节营养不良。最近的发展 以及对DBMD和其他神经肌肉疾病的标准化护理指南的实施 预计将导致改善临床结果；然而，已发表的证据仍然有限。新兴 这些疾病的治疗选择是实现改善的巨大希望和机会的来源 结果；然而，更好地了解这些患者的疾病严重程度和目前的护理负担 障碍是此类倡议成功的关键。犹他大学MD STARnet团队已经被 在本供资周期内活跃(DD001108：2014年9月1日-2019年8月31日)，并处于继续 作为组件C的一部分进行研究。作为组件A的一部分，我们与当地和国家的合作 神经肌肉基础对于确定我们的研究可以按顺序回答的问题非常重要 以弥补知识上的差距。对于C部分，我们将重点放在 宣布融资机会，总体目标是用拟议的新项目继续我们现有的项目 使用MD STARnet数据的研究项目。此外，我们计划进行调查，以评估 精神分裂症患者的心理和神经行为功能及健康相关生活质量。我们会 通过在专业协会的本地、国家和国际演示来传播我们的研究结果 会议并向同行评议的期刊提交稿件。我们现有计划的优势和资源 将增加对这些人面临的医疗问题和护理障碍的了解，并加强我们的 有能力为我们的患者提供更一致的高质量护理。此外，我们建议这个项目将 使我们能够评估标准化护理指南的实施对临床结果和成本的影响 对这样的病人的护理。我们将分析MD STARnet研究中解决的高优先级问题 议程(目标1)，确定在理解方面的其他差距，重点是心理和神经行为 合并症和健康相关的生活质量(目标2)，并为其他MD提供二级分析师支持 至少有三个研究项目的STARNet站点(目标3)。