MYOCARDIAL CHANGES BY MRI IN DMD

DMD 患者的 MRI 心肌变化

• 批准号: 7604661
• 负责人: DANIEL B DRACHMAN
• 金额: $ 0.03万
• 依托单位: JOHNS HOPKINS UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-12-01 至 2007-09-16
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7604661
• 关键词: Arrhythmia; Birth; Blood; Cardiac; Cohort Studies; Computer Retrieval of Information on Scientific Projects Database; Dobutamine; Dose; Duchenne muscular dystrophy; Echocardiography; Enrollment; Funding; Grant; Heart Diseases; Institution; Left ventricular structure; Life; Magnetic Resonance Imaging; Morbidity - disease rate; Muscular Dystrophies; Myocardial; Myopathy; Patients; Pilot Projects; Research; Research Personnel; Resources; Risk; Series; Skeletal system; Source; Standards of Weights and Measures; Sudden Death; Testing; United States National Institutes of Health; Urine; aged; clinically significant; day; follow-up; male; mortality; pre-clinical

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Cardiac disease eventually becomes manivest in almost all patients with Duchenne muscular dystrophy, the most common of the muscular dystrophies (1/3500 live male births) and is likely to become the major cause of morbidity and mortality in the next decade (given expected treatment advances to treat skeletal muscle disease). For this reason, we plan to enroll 30 patients with DMD, aged 7-40, to undergo a series of cardiac tests over the course of 2 days as part of a 2-year pilot study. Study assessments will include a cardiac MRI, advanced echocardiogram studies (to qualtify regional strain all over the left ventricle), cardiac functional reserve testing with low-dose dobutamine (for those 18 and older), blood and urine studies, and several ECG-related tests to determine the risk for arrhythmia/sudden death. This research promises to help identify patients with preclinical cardiac disease who might benefit from early targeted therapy, identify patients with overt cardiac disease at higher risk for sudden death, clarify important cardiac management issues in hospitalized patients with DMD, and help clarify the clinical significance of abnormal findings with these tests, which may eventually become standard for all patients with DMD. We will also establish a well-studied cohort for long-term follow up so that we may later identify predictors of long-term cardiac morbidity and mortality.

该副本是利用众多研究子项目之一 由NIH/NCRR资助的中心赠款提供的资源。子弹和 调查员（PI）可能已经从其他NIH来源获得了主要资金， 因此可以在其他清晰的条目中代表。列出的机构是 对于中心，这不一定是调查员的机构。 在几乎所有Duchenne肌肉营养不良的患者中，心脏疾病最终都会变得生动，这是最常见的肌肉营养不良症（1/3500 Live雄性出生），并且很可能在接下来的十年中成为发病率和死亡率的主要原因（预期治疗治疗骨骼肌疾病的预期治疗方法）。 因此，作为一项为期2年的试点研究的一部分，我们计划在2天内招募30名DMD患者，年龄在7-40岁的DMD患者2天内进行一系列心脏测试。研究评估将包括心脏MRI，晚期超声心动图研究（以使左心室的区域性菌株降低区域应变），使用低剂量多巴胺（对于18岁及以上的人），血液和尿液研究，几项ECG相关的测试，以确定心律失常/突然死亡的风险。 这项研究有望帮助识别患有临床前疾病的患者，这些患者可能会从早期的靶向治疗中受益，识别患有较高猝死风险的明显心脏病患者，阐明住院DMD患者的重要心脏管理问题，并有助于阐明这些检查异常发现的临床意义，这些检查最终可能成为所有DMD患者的标准患者。 我们还将建立一个经过良好研究的队列，以进行长期随访，以便稍后可以确定长期心脏发病率和死亡率的预测指标。