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

描述（由申请人提供）：儿童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，结果必须在儿童正常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，如果有进展，通常是非常缓慢的。这不仅影响诊断，也影响治疗的评估。事实上，临床治疗试验和常规病人护理可能需要儿童接受侵入性或不方便的检查，包括肌肉活检、磁共振成像和肌电图。一种灵敏、无创的检测和测量儿童神经肌肉病理的方法将极大地提高评估新疗法的能力，并有助于对患有这些疾病的儿童进行常规护理。电阻抗肌图（EIM）是在这方面特别有用的一种技术。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上，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，其主要兴趣是用于快速应用EIM的设备的商业化。在这一应用中，Convergence计划在第一阶段SBIR成功的基础上，进一步完善EIM技术，将其应用于儿科人群。我们将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，从而实现这一目标。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，并基于预测正常百分比（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，与几个专家的输入，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，这些评分可以有效地跟踪1岁以上的5-12岁儿童的疾病状态，这些儿童的功能测量通常可以准确地获得。然后，与3组健康男孩（0-4岁、5-12岁和13-18岁）相比，我们将评估这些EIM评分在检测DMD男孩3个月和6个月期间疾病进展的能力方面的成功。除了进一步完善儿科EIM，我们还将创建创新的共享资源，包括一个在线计算器，以协助EIM样本量的计算和一个开放源代码的在线数据库，正常和DMD数据，可以作为未来儿科神经肌肉疾病资源共享的基础，学术和行业研究者都一样。

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.