Development of Novel Upper Extremity Outcome Measures Using 3D-Vision Technology

描述（由申请人提供）：本提案的主要目的是为神经肌肉疾病（NMD）的临床试验开发一种新的上肢结局指标。与传统的静态肌肉力量或孤立关节活动范围的测量不同，上肢功能性能测量有望对肌肉功能的早期变化更敏感。这样的上肢功能结果测量可以显着缩短早期临床试验，并加快治疗发现过程。随着三维（3D）视觉传感器技术的最新进展，研究人员率先开发出一种创新而简单，直观，低成本的方法，仅使用一个便携式传感器系统来定量评估上肢的3D可达工作空间;基本上利用了一个完整的运动分析实验室的功能在一个传感器平台上，提供非常详细的3D运动数据。上肢可达工作空间分析特别适用于各种NMD，因为典型的无力模式涉及肩带肌肉。该提案建立在研究人员之前的基础工作之上，将已经开发的方法和软件扩展到新的商用传感器平台（Microsoft Kinect），该平台有望实现准确，具有成本效益，并具有更大的可扩展性和可持续性潜力。除了健康对照外，拟议的研究还将在两种典型和常见的NMD中测试开发的系统，这些NMD具有肩带肌无力，这将提供整个生命谱和不同程度的上肢功能障碍的试验台病例：杜氏肌营养不良症（DMD）和面肩肱营养不良症（FSHD）。这些代表了快速进展的儿科疾病（DMD）和缓慢进展的迟发性疾病过程（FSHD）。具体目标是：1）确定Kinect测量的上肢可达工作空间相对于同时收集的亚毫米全尺寸运动分析系统的可达工作空间的可靠性和有效性; 2）通过评估上肢功能的其他已建立的测量来确定Kinect测量的可达工作空间的并发有效性，包括：布鲁克量表、9孔钉试验（9 HPT）、手动肌肉测试（MMT）、测角术和NIH开发的NeuroQOL个人报告上肢功能结局;和3）评估1.5年（18个月）期间Kinect测量的可达工作空间变化的响应性纵向研究，每3个月收集一次数据，涉及40名DMD和40名FSHD患者。将Kinect可达工作空间对疾病进展的反应性与传统结局指标观察到的变化进行比较。我们还将检查Kinect测量的可达工作空间，以预测作为临床意义里程碑的自我喂养丧失。开发一种准确、可靠、具有成本效益且具有临床意义的上肢结局指标，不仅将显著促进NMD的治疗发现过程，还将促进各种其他肌肉骨骼疾病的治疗发现过程。

DESCRIPTION (provided by applicant): The primary objective of this proposal is to develop a novel upper extremity outcome measure for clinical trials in neuromuscular diseases (NMDs). An upper limb functional performance measure, unlike traditional measurement of static muscle strength or isolated joint range of motion, promises to be more sensitive to early changes in muscle function. Such an upper limb functional outcome measure could significantly shorten early phase clinical trials and hasten the therapeutic discovery process. With recent advances in 3-dimensional (3D) vision sensor technology, the study investigators were the first to develop an innovative yet simple, intuitive, low-cost methodology to quantitatively assess the 3D reachable workspace of the upper extremity using just one portable sensor system; essentially harnessing the capabilities of a full-scale motion analysis laboratory in a single sensor platform that provides comparably detailed 3D motion data. An upper extremity reachable workspace analysis is especially applicable to a variety of NMDs, since the typical pattern of weakness involves shoulder girdle muscles. This proposal builds upon the investigators' previous foundational work by extending the already developed methodology and software to a new commercially-available sensor platform (Microsoft Kinect) that promises to be accurate, cost-effective, and with greater potential for scalability and sustainability. In addition to healthy controls, the proposed study will test the developed system in two prototypical and common NMDs with shoulder girdle muscle weakness that will provide test-bed cases across the life spectrum and varying degrees of upper limb dysfunction: Duchenne muscular dystrophy (DMD) and Facioscapulohumeral dystrophy (FSHD). These represent both a rapidly progressive pediatric disease (DMD) and a slowly progressive late-onset disease process (FSHD). The specific aims are: 1) To determine the reliability and validity of the Kinect-measured upper limb reachable workspace against a simultaneously collected sub-millimeter full-scale motion analysis system's reachable workspace; 2) To determine the concurrent validity of Kinect-measured reachable workspace by evaluating against the other established measures of upper limb function including: Brooke scale, 9-hole peg test (9HPT), manual muscle testing (MMT), goniometry, and the NIH-developed NeuroQOL person reported outcome for upper limb function; and 3) To assess the responsiveness to change of the Kinect-measured reachable workspace during 1.5-year (18mo) longitudinal study with every 3 months data collection involving 40 DMD and 40 FSHD patients. The responsiveness to change of the Kinect's reachable workspace to disease progression will be compared against changes observed by traditional outcome measures. We will also examine Kinect-measured reachable workspace to predict loss of self-feeding as a clinically-meaningful milestone. Development of an accurate, reliable, cost-effective, and clinically-meaningful upper extremity outcome measure will significantly facilitate the therapeutic discovery process not only in NMDs but also for various other musculoskeletal conditions.