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

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，结果必须在正常儿童发育和合作程度的背景下进行评估。此外，神经肌肉疾病的严重程度是高度可变的，并且如果存在，进展通常非常缓慢。这不仅影响诊断，而且影响治疗评估。事实上，临床治疗试验和常规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。一个敏感的，非侵入性的测试，以检测和测量神经肌肉病理的儿童将大大提高能力，以评估新的治疗方法，以及协助定期护理的儿童患有这些条件。电阻抗肌描记术（EIM）是一种在这方面可能特别有用的技术。在EIM中，通过表面电极将不可感知的高频电流施加到感兴趣的肌肉或肌肉群，并测量随之发生的表面电压模式的变化。表面电压模式的变化提供了疾病状态的信息，并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EIM快速应用设备的商业化。在这一应用中，Convergence计划通过进一步完善EIM技术应用于儿科人群，以其在1期SBIR中的成功为基础。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岁男孩和女孩多块肌肉的正常数据，并根据预测正常百分比（类似于肺功能测试）开发一个用户友好的评分系统。在具体目标2中，在几位专家的投入下，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，我们将在儿童期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。我们将首先验证几种优化的EIM评分，这些评分可有效跟踪5-12岁儿童1岁以上的疾病状态，这些儿童通常可以准确获得功能测量。然后，我们将评估这些EIM评分在DMD男孩中检测3个月和6个月期间疾病进展的能力，并与3组健康男孩进行比较：0-4岁，5-12岁和13-18岁。除了进一步完善儿科EIM外，我们还将创建创新的共享资源，包括在线计算器，以协助EIM样本量计算和正常和DMD数据的开源在线数据库，这可以作为未来共享儿科神经肌肉疾病资源的基础，供学术和行业研究人员使用。

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.