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

描述（由申请人提供）： 我们的目标是为神经科医生和其他临床医生提供一种快速，无痛，无创评估儿童神经肌肉疾病的方法。小儿神经肌肉疾病包括肌营养不良症、脊髓性肌萎缩症和炎性肌炎，仅在美国就有超过100，000名儿童受到影响。目前用于评估疾病状态的临床测量（诸如肌肉力量测试或Hammersmith评级量表）在许多方面受到限制，包括它们具有有限的再现性、对细微疾病变化缺乏敏感性、它们强烈依赖于优秀的受试者合作、显著的地板和天花板效应（即，这些测试在非常轻微或非常严重的疾病中没有信息），并且它们需要相当多的评估者专业知识和培训。为了更有效地护理受神经肌肉疾病影响的儿童，非常需要一种非侵入性和无痛的工具，可以快速准确地评估疾病状态，最小的主体合作。这种工具可以用于个体患者，提供有关正在进行的治疗的有效性或改变治疗的潜在需求的有价值的数据。同样重要的是，该工具可用于临床药物试验，有助于更灵敏、更快速地确定潜在的治疗方法，并且受试者数量比目前可能的更少，从而大大降低研究成本。我们相信电阻抗肌电图（EIM）就是这样一种工具。在EIM中，通过两个表面电极施加不可检测的低强度交流电流，并测量所得的表面电压。多项已发表的成人和儿童研究都支持EIM具有高度可重复性，并且对疾病状态非常敏感。此外，由于其完全无创，无痛的性质，它使其成为一个特别有前途的工具，用于儿童。Convergence Medical Devices，Inc.作为其主要重点和核心竞争力的应用EIM的评估肌肉健康。第一阶段SBIR的具体目标是：（1）开发一种用于儿童神经肌肉评估的手持式EIM设备，（2）在一组健康成人中测试该设备，以验证其安全性并产生高质量的EIM数据，以及（3）研究一组15名健康儿童和15名患有一种相对常见的儿科疾病脊髓性肌萎缩症的儿童，以确定系统的容差和可重复性，并初步评估其对健康和疾病状况的敏感性。这三个目标的成功完成将产生一个初步的原型EIM设备优化用于儿童。我们在健康和患病儿童中使用该器械获得的见解将为在启动纵向研究评估临床疗效（作为计划的II期SBIR的一部分）之前进一步完善器械/软件提供坚实的基础。 公共卫生相关性： 仅在美国就有超过10万名儿童患有一种或多种严重的神经肌肉疾病。然而，目前用于诊断疾病和评估严重程度的方法是不敏感和有限的。电阻抗肌电描记术技术作为一种提供肌肉健康详细数据而不受当前方法限制的技术，已显示出很大的前景。因此，该项目的总体目标是开发一种手持式，非侵入性电阻抗设备，可以对儿童的肌肉健康进行准确，快速的评估。

DESCRIPTION (provided by applicant): Our goal is to provide neurologists and other clinicians a method for the rapid, painless, non-invasive evaluation of children with neuromuscular disease. Pediatric neuromuscular disorders include muscular dystrophies, spinal muscular atrophy, and inflammatory myositis, and affect well over 100,000 children in the United States alone. Present-day clinical measures to assess disease status such as muscle strength testing or the Hammersmith rating scale are limited in a number of ways, including their having limited reproducibility, a lack of sensitivity to subtle disease change, their being strongly dependent on excellent subject cooperation, marked floor and ceiling effects (i.e., the tests are uninformative in very mild or very advanced disease), and their requiring considerable evaluator expertise and training. In order to care for children affected by neuromuscular disorders more effectively, a non-invasive and painless tool that can rapidly and accurately assess disease status with minimal subject cooperation is greatly needed. Such a tool could be used with individual patients, providing valuable data on the effectiveness of ongoing treatment or the potential need to alter therapy. Equally important, the tool could be used in clinical drug trials, helping to identify potential therapies more sensitively, more quickly and with fewer subjects than currently possible, thus greatly reducing study cost. Electrical impedance myography (EIM), we believe, is such a tool. In EIM, non-detectible, low- intensity, alternating electrical current is applied through two surface electrodes and resulting surface voltages are measured. Multiple published studies, both in adults and children, support that EIM is both highly reproducible and very sensitive to disease status. Moreover, given its entirely non-invasive, painless nature, it makes it an especially promising tool for use in children. Convergence Medical Devices, Inc. has as its primary focus and core competency the application of EIM for the assessment of muscle health. The specific aims of this Phase I SBIR, are to: (1) develop a hand-held EIM device for neuromuscular assessment in children, (2) test the device in a group of healthy adults to verify that it is safe and yields quality EIM data, and (3) study a group of 15 healthy children and 15 children with one relatively common pediatric disease, spinal muscular atrophy, to identify the system's tolerances and repeatability and preliminarily assess its sensitivity to health and disease status. The successful completion of these three aims will yield a preliminary prototype EIM device optimized for use in children. The insights we obtain using this device in both healthy and diseased children will offer a strong foundation for further device/software refinement necessary before launching a longitudinal study assessing clinical efficacy as part of a planned Phase II SBIR. PUBLIC HEALTH RELEVANCE: Over 100,000 children in the United States alone suffer from one or more serious neuromuscular diseases. However, current methods for diagnosing disease and assessing severity are insensitive and limited. The technique of electrical impedance myography has shown great promise as a technique for providing detailed data on muscle health without the limitations of current methods. Thus, the overall goal of this project is to develop a handheld, noninvasive electrical impedance device that can perform accurate, rapid assessments of muscle health in children.