A device for rapid, painless, bedside muscle evaluation of children with neuromus

一种对神经肌肉儿童进行快速、无痛、床旁肌肉评估的设备

• 批准号: 8838869
• 负责人: Jose Luis Bohorquez
• 金额: $ 75.47万
• 依托单位: MYOLEX, INC.
• 依托单位国家: 美国
• 财政年份: 2011
• 资助国家: 美国
• 起止时间: 2011-04-01 至 2017-01-18
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8838869
• 关键词: 12 year old; 18 year old; Age; Biopsy; Businesses; Caring; Child; Child Care; Child Development; Childhood; Clinic; Clinical; Clinical Trials; Computer software; Country; Data; Databases; Development; Device or Instrument Development; Devices; Diagnosis; Diagnostic; Disease; Disease Progression; Duchenne muscular dystrophy; Electrodes; Electromyography; Evaluation; Fatty acid glycerol esters; Female; Fibrosis; Foundations; Frequencies; Funding; Future; Gender; Goals; Health; Heart; Height; Individual; Industry; Interest Group; Intramuscular; Lung; Magnetic Resonance Imaging; Measurement; Measures; Medical Device; Muscle; Myography; Neuromuscular Diseases; Normal Range; Outcome; Painless; Pathology; Patient Care; Patients; Pattern; Phase; Population; Pulmonary function tests; Reference Values; Research Personnel; Resource Sharing; Resources; Sample Size; Severity of illness; Small Business Innovation Research Grant; Surface; System; Techniques; Technology; Testing; Therapeutic Clinical Trial; Therapeutic Trials; Time; Treatment Efficacy; Weight; Work; age group; aged; base; boys; cohort; commercialization; cost effective; data sharing; efficacy trial; electric impedance; functional decline; functional outcomes; girls; improved; innovation; interest; male; neuromuscular; neuromuscular system; open source; rapid technique; response; software development; standard measure; success; tool; user friendly software; user-friendly; voltage

DESCRIPTION (provided by applicant): The neuromuscular assessment of children is uniquely challenging. Unlike examination of the heart or lung, the complete bedside assessment of the neuromuscular system requires the active participation of the child and results must be evaluated in the context of normal child development and degree of cooperation. Moreover, neuromuscular disease severity is highly variable and progression, if present, is typically very slow. This impacts not only diagnosis, but also assessment of therapy. Indeed, clinical therapeutic trials and regular patient care may require children to undergo invasive or inconvenient tests, including muscle biopsy, magnetic resonance imaging and electromyography. A sensitive, noninvasive test to detect and measure neuromuscular pathology in children would greatly improve the ability to evaluate new therapies as well as to assist in the regular care of children suffering from these conditions. Electrical impedance myography (EIM) is one technique that may be especially useful in this regard. In EIM, an imperceptible high-frequency electrical current is applied via surface electrodes to a muscle or muscle group of interest and alterations in the consequent surface voltage pattern are measured. Alterations in the surface voltage patterns provide information on disease status and can track change over time. Convergence Medical Devices, Inc. is a small business whose main interest is in the commercialization of devices for the rapid application of EIM. In this applicatin, Convergence plans to build upon its success in its Phase 1 SBIR by further refining EIM technology for application to a pediatric population. We will achieve this by teaming up with several well- respected pediatric neuromuscular researchers across the country to further refine and test our system through four specific aims. In Specific Aim 1, we will obtain normative data on multiple muscles in 256 boys and girls aged 0-18 years and develop a user-friendly scoring system based on the percent predicted normal (akin that performed for pulmonary function testing). In Specific Aim 2, with the input of several experts, we will improve our software to incorporate these scores aiming to create a straightforward user interface. In Specific Aims 3 and 4, we will test our improved system in one of the most common and devastating neuromuscular diseases of childhood: Duchenne muscular dystrophy (DMD). We will first validate several optimized EIM scores that effectively track disease status in 5-12 year-old children over 1-year in whom functional measures can generally be accurately obtained. We will then evaluate the success of these EIM scores in their ability to detect disease progression over 3- and 6-month periods of time in DMD boys as compared to 3 groups of healthy boys: 0-4 years, 5-12 years, and 13-18 years of age. In addition to further refining pediatric EIM, we will also create innovative shared resources, including an online calculator to assist with EIM sample size calculations and an open-source online database of normal and DMD data which can serve as a foundation for future shared pediatric neuromuscular disease resources to both academic and industry investigators alike.

描述（由申请人提供）：儿童的神经肌肉评估具有独特的挑战性。与心脏或肺部检查不同，神经肌肉系统的完整床边评估需要儿童的积极参与，并且必须在儿童正常发育和合作程度的背景下评估结果。此外，神经肌肉疾病的严重程度变化很大，并且如果存在的话，进展通常非常缓慢。这不仅影响诊断，还影响治疗评估。事实上，临床治疗试验和定期患者护理可能需要儿童接受侵入性或不方便的测试，包括肌肉活检、磁共振成像和肌电图。一种敏感的、无创的检测和测量儿童神经肌肉病理学的测试将大大提高评估新疗法的能力，并协助定期护理患有这些疾病的儿童。电阻抗肌电图（EIM）是一种在这方面特别有用的技术。在 EIM 中，通过表面电极将难以察觉的高频电流施加到感兴趣的肌肉或肌肉群，并测量随之而来的表面电压模式的变化。表面电压模式的变化提供了有关疾病状态的信息，并且可以跟踪随时间的变化。 Convergence Medical Devices, Inc. 是一家小型企业，其主要兴趣在于快速应用 EIM 的设备商业化。在此应用中，Convergence 计划在其第一阶段 SBIR 的成功基础上，进一步完善 EIM 技术以应用于儿科人群。我们将通过与全国几位备受尊敬的儿科神经肌肉研究人员合作，通过四个具体目标进一步完善和测试我们的系统来实现这一目标。在具体目标 1 中，我们将获得 256 名 0-18 岁男孩和女孩的多个肌肉的规范数据，并根据预测正常百分比（类似于肺功能测试的百分比）开发一个用户友好的评分系统。在特定目标 2 中，根据几位专家的意见，我们将改进我们的软件以纳入这些分数，旨在创建一个简单的用户界面。在具体目标 3 和 4 中，我们将在儿童最常见和最具破坏性的神经肌肉疾病之一：杜氏肌营养不良症 (DMD) 中测试我们改进的系统。我们将首先验证几个优化的 EIM 评分，这些评分可以有效跟踪 1 岁以上 5-12 岁儿童的疾病状态，通常可以准确地获得功能测量。然后，我们将评估这些 EIM 评分在检测 DMD 男孩 3 个月和 6 个月内疾病进展的能力方面是否成功，并与 3 组健康男孩（0-4 岁、5-12 岁和13-18岁。除了进一步完善儿科 EIM 之外，我们还将创建创新的共享资源，包括帮助 EIM 样本量计算的在线计算器以及正常和 DMD 数据的开源在线数据库，该数据库可以作为未来共享儿科神经肌肉的基础为学术和行业研究人员提供疾病资源。

项目成果

Electrical impedance myography for reducing sample size in Duchenne muscular dystrophy trials.

电阻抗肌动描记法用于减少杜氏肌营养不良症试验中的样本量。

• 发表时间: 2020
• 作者: Leitner, Melanie L;Kapur, Kush;Darras, Basil T;Yang, Michele;Wong, Brenda;Dalle Pazze, Laura;Florence, Julaine;Buck, Martin;Freedman, Laura;Bohorquez, Jose;Rutkove, Seward;Zaidman, Craig
• 通讯作者: Zaidman, Craig