Component A: Iowa MD STARnet Core Site

组件 A：爱荷华州 MD STARnet 核心站点

• 批准号: 10662427
• 负责人: Katherine Dianne Mathews
• 金额: $ 41.5万
• 依托单位: UNIVERSITY OF IOWA
• 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/10662427
• 关键词: Component; Iowa; MD; STARnet; Core

PROJECT SUMMARY The Muscular Dystrophy Community Assistance, Research, and Education Amendments of 2001 authorized the Centers for Disease Control and Prevention to create a National Muscular Dystrophy Epidemiology Program. This program – The Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) – was established in 2002 to conduct population-based surveillance and long-term follow-up of individuals diagnosed with Duchenne and Becker muscular dystrophies (DBMD). In 2011, the MD STARnet expanded to include cross-sectional surveillance for seven additional MDs (congenital, distal, Emery- Dreifuss, facioscapulohumeral, limb-girdle, myotonic, and oculopharyngeal), and in 2014, this expanded surveillance was continued. Our Iowa MD STARnet Core Site was one of four charter awardees for the MD STARnet in 2002 and was successfully refunded in 2006, 2011, and 2014. As an MD STARnet Core Site, we successfully integrated clinical, programmatic, and referral resources provided by key partners in Iowa, such as the Iowa Neuromuscular Program and the Iowa Muscular Dystrophy Association, with the active, population-based resources of the Iowa Registry for Congenital and Inherited Disorders to conduct longitudinal surveillance of DBMD and cross-sectional surveillance for the seven additional MDs. Our partnership with the Reproductive Molecular Epidemiology Research and Education Program at The University of Iowa has provided experienced investigators to conduct data analyses and publish MD STARnet data. For the proposed project, we will use our highly skilled and successful team to extend and expand our leadership role in population-based longitudinal surveillance and research for the nine MDs. We propose to continue working collaboratively with CDC investigators and other grant awardees to implement standardized methods and data collection activities. To accomplish this, we aim to: 1) build new and maintain existing relationships with MD partners to enhance and promote surveillance efforts; 2) continue to conduct longitudinal, population-based surveillance using existing MD STARnet methods to identify and gather data on individuals with one of the nine MDs; and 3) develop research methods and tools to augment surveillance data collection. Among our planned research activities will be examining pregnancy among individuals with MD, evaluating clinical trial participation and non-participation, and assessing the impact of clinical trial participation on the natural history of MD. Findings generated from our proposed surveillance and research activities will identify the public health impact of MD and contribute to the clinical understanding of the natural histories, survival and mortality, and healthcare needs of individuals and families affected by MD. These findings also will guide public health agencies in the development and provision of policies and programs that will ensure access to comprehensive healthcare services and link individuals with MD and their families to social services that will improve quality of life.

项目概要 2001 年肌肉萎缩症社区援助、研究和教育修正案获得授权 疾病控制和预防中心创建国家肌营养不良症流行病学 程序。该计划 – 肌营养不良症监测、追踪和研究网络 (MD STARnet）——成立于 2002 年，旨在进行基于人群的监测和长期随访 被诊断患有杜氏肌营养不良症和贝克尔肌营养不良症 (DBMD) 的个体。 2011年，MD STARnet 扩大到包括对另外 7 个 MD（先天性、远端、Emery- Dreifuss、面肩肱、肢带、肌强直和眼咽），2014 年，这一范围扩大 监视工作继续进行。我们的爱荷华州 MD STARnet 核心站点是 MD 的四个特许获得者之一 2002 年 STARnet 并于 2006 年、2011 年和 2014 年成功退款。作为 MD STARnet 核心站点，我们 成功整合了爱荷华州主要合作伙伴提供的临床、计划和转诊资源，例如 作为爱荷华州神经肌肉计划和爱荷华州肌营养不良协会，与积极的， 爱荷华州先天性和遗传性疾病登记处以人口为基础的资源，以开展 DBMD 的纵向监测和另外 7 个 MD 的横断面监测。我们的 与生殖分子流行病学研究和教育计划合作 爱荷华大学提供了经验丰富的研究人员进行数据分析并发布MD STARnet 数据。对于拟议的项目，我们将利用我们高技能且成功的团队来扩展和扩展我们的 在九个医学博士的基于人群的纵向监测和研究中发挥领导作用。我们建议 继续与 CDC 研究人员和其他赠款获得者合作，实施标准化 方法和数据收集活动。为了实现这一目标，我们的目标是：1）建造新的并维护现有的 与 MD 合作伙伴的关系，以加强和促进监测工作； 2）继续进行 使用现有 MD STARnet 方法进行纵向、基于人群的监测来识别和收集数据 对于拥有九个 MD 之一的个人； 3）开发研究方法和工具以加强监测 数据收集。我们计划的研究活动之一是检查患有以下疾病的个体的怀孕情况 MD，评估临床试验参与和不参与，并评估临床试验的影响 参与MD的自然史。我们提议的监测和研究得出的结果 活动将确定MD对公共卫生的影响，并有助于临床理解自然 受MD影响的个人和家庭的病史、生存和死亡率以及医疗保健需求。这些 研究结果还将指导公共卫生机构制定和提供政策和计划， 将确保获得全面的医疗保健服务，并将 MD 患者及其家人与 社会服务将改善生活质量。

项目成果

Prophylactic use of cardiac medications for delay of left ventricular dysfunction in Duchenne muscular dystrophy.

预防性使用心脏药物来延迟杜氏肌营养不良症的左心室功能障碍。

• DOI: 10.1002/bdr2.2260
• 发表时间: 2024
• 期刊: Birth defects research
• 影响因子: 2.1
• 作者: Conway,KristinM;Thomas,Shiny;Ciafaloni,Emma;Khan,RabiaS;Mann,JoshuaR;Romitti,PaulA;Mathews,KatherineD;MuscularDystrophySurveillance,TrackingandResearchNetwork(MDSTARnet)
• 通讯作者: MuscularDystrophySurveillance,TrackingandResearchNetwork(MDSTARnet)