Upper Extremity Examination for Neuromuscular Diseases (U-EXTEND): Protocol for a Multimodal Feasibility Study.
Upper Extremity Examination for Neuromuscular Diseases (U-EXTEND): Protocol for a Multimodal Feasibility Study.
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DOI：
10.2196/40856
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发表时间：
2022-10-27
影响因子：
1.7
通讯作者：
Scharf, Rebecca
中科院分区：
文献类型：
--
作者：
Gutierrez, Robert;McCrady, Allison;Masterson, Chelsea;Tolman, Sarah;Boukhechba, Mehdi;Barnes, Laura;Blemker, Silvia;Scharf, Rebecca
关键词：
神经肌肉疾病，如脊髓性肌萎缩症(SMA)和杜氏肌营养不良症(DMD)，可能会导致幼儿和成人运动丧失、呼吸衰竭和早期死亡。随着新的治疗方法的出现，需要新的评估方法来评估目前在现有运动评分测试中没有记录到的进展。通过我们的可行性研究，我们的跨学科研究团队旨在开发一种新的、多模式的测量神经肌肉疾病儿童运动功能的范例，这将彻底改变临床试验终点的测量方式，从而加速儿童神经肌肉疾病的新治疗方法的流水线。通过上肢神经肌肉疾病检查(U-EXTEND)研究，我们假设本文提出的新的上肢肌肉结构和功能的客观测量方法将能够捕捉到用当前临床测量方法无法测量的微小变化和功能差异。U-EXTEND引入了一种新的范式，其中具体的、定量的测量被用来评估SMA和DMD患者的运动功能。目标1将专注于使用超声波技术来研究肌肉的大小、质量和功能，特别是分离上肢的二头肌和旋前肌，以便随着时间的推移进行随访。为了实现这一点，临床研究人员将通过使用超声成像和手持测力仪提取一组与肌肉结构、质量和功能相关的测量数据。Aim 2将专注于利用可穿戴无线传感器技术在参与者进行日常生活活动时捕获运动数据。测量数据将被检查并与来自健康队列的数据进行比较，并计算运动功能评分。这两个目标的数据收集工作于2021年1月开始。截至2022年7月，我们已招募了44名参与者(9名患有SMA，20名患有DMD，15名健康参与者)。我们预计初步结果将于2022年夏天公布。我们假设，通过应用所描述的测量肌肉结构和上肢功能的工具和技术，我们将创建一个精确量化神经肌肉疾病患者运动功能变化的系统。我们的研究将使我们能够跟踪随着时间的推移在临床上最小的重要差异，以评估新疗法的进展。通过比较多次访问的肌肉评分和功能评分，我们将能够检测到参与者执行功能任务的能力和内在肌肉属性的微小变化。DERR1-10.2196/40856
Neuromuscular diseases, such as spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD), may result in the loss of motor movements, respiratory failure, and early mortality in young children and in adulthood. With novel treatments now available, new evaluation methods are needed to assess progress that is not currently captured in existing motor scale tests. With our feasibility study, our interdisciplinary team of investigators aims to develop a novel, multimodal paradigm of measuring motor function in children with neuromuscular diseases that will revolutionize the way that clinical trial end points are measured, thereby accelerating the pipeline of new treatments for childhood neuromuscular diseases. Through the Upper Extremity Examination for Neuromuscular Diseases (U-EXTEND) study, we hypothesize that the novel objective measures of upper extremity muscle structure and function proposed herein will be able to capture small changes and differences in function that cannot be measured with current clinical metrics. U-EXTEND introduces a novel paradigm in which concrete, quantitative measures are used to assess motor function in patients with SMA and DMD. Aim 1 will focus on the use of ultrasound techniques to study muscle size, quality, and function, specifically isolating the biceps and pronator muscles of the upper extremities for follow-ups over time. To achieve this, clinical investigators will extract a set of measurements related to muscle structure, quality, and function by using ultrasound imaging and handheld dynamometry. Aim 2 will focus on leveraging wearable wireless sensor technology to capture motion data as participants perform activities of daily living. Measurement data will be examined and compared to those from a healthy cohort, and a motor function score will be calculated. Data collection for both aims began in January 2021. As of July 2022, we have enrolled 44 participants (9 with SMA, 20 with DMD, and 15 healthy participants). We expect the initial results to be published in summer 2022. We hypothesize that by applying the described tools and techniques for measuring muscle structure and upper extremity function, we will have created a system for the precise quantification of changes in motor function among patients with neuromuscular diseases. Our study will allow us to track the minimal clinically important difference over time to assess progress in novel treatments. By comparing the muscle scores and functional scores over multiple visits, we will be able to detect small changes in both the ability of the participants to perform the functional tasks and their intrinsic muscle properties. DERR1-10.2196/40856
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