Reliability and Validity of the Reachable Workspace Total Score With Wrist Weight in Facioscapulohumeral Muscular Dystrophy.

IF 3.1 3区医学 Q2 CLINICAL NEUROLOGY

Muscle & Nerve Pub Date : 2025-11-01 Epub Date: 2025-08-13 DOI:10.1002/mus.70002

Lena Hubig, Adi Eldar-Lissai, Siu Hing Lo, John Jiang, David Cella, Sarah Acaster

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引用次数: 0

Abstract

Introduction/aims: Facioscapulohumeral muscular dystrophy (FSHD) is characterized by relentlessly progressive muscular weakness, impacting patients' daily functioning, health-related quality of life (HRQoL), and activities of daily living (ADLs), particularly those that require upper extremity (UE) function. Reachable workspace (RWS) is a performance outcome (PerfO) measure for UE function in FSHD. This study aimed to explore the reliability and validity of RWS average (for both arms) Total relative surface area (RSA) Q1-Q5 with 500 g wrist weight score ("Total Q1-Q5 RSA score") in FSHD.

Methods: Data were sourced from the Phase 2 ReDUX4 clinical trial. Test-retest reliability and convergent validity of the Total Q1-Q5 RSA score were evaluated. The following outcome measures were included in the analyses: Timed up and go (TUG) and FSHD TUG, manual dynamometry, FSHD-health index, patient's global impression of change, Ricci Clinical Severity Scale (CSS), and musculoskeletal magnetic resonance imaging (MRI).

Results: Almost perfect test-retest reliability (ICC = 0.98) and good convergent validity with moderate (|r| ≥ 0.3) correlations with most ADL items, clinical severity and UE muscle strength were shown.

Discussion: Psychometric evidence supports the reliability and validity of the Total RSA Q1-Q5 score as a measure of UE function in FSHD and contributes to the evidence for its use as an outcome in clinical trials of FSHD. Additional study is required to explore responsiveness to change.

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面肩肱骨肌萎缩症可及工作空间总分与腕重的信度和效度。

简介/目的：面肩肱骨肌营养不良症（FSHD）的特征是持续进行性肌肉无力，影响患者的日常功能、健康相关生活质量（HRQoL）和日常生活活动（adl），特别是那些需要上肢（UE）功能的患者。可达工作空间（RWS）是FSHD中UE功能的性能结果（PerfO）度量。本研究旨在探讨RWS平均（双臂）总相对表面积（RSA） Q1-Q5与500 g腕重评分（“总Q1-Q5 RSA评分”）在FSHD中的信度和效度。方法：数据来源于ReDUX4二期临床试验。评估总Q1-Q5 RSA评分的重测信度和收敛效度。以下结果测量包括在分析中：time up and go （TUG）和FSHD TUG、手动动力测量、FSHD健康指数、患者总体变化印象、Ricci临床严重程度量表（CSS）和肌肉骨骼磁共振成像（MRI）。结果：测试重测信度（ICC = 0.98）接近完美，收敛效度良好，与大多数ADL项目、临床严重程度和UE肌力呈中等相关性（|或|≥0.3）。讨论：心理测量证据支持RSA Q1-Q5总分作为FSHD中UE功能测量的可靠性和有效性，并为其作为FSHD临床试验结果的证据提供了依据。需要进一步的研究来探索对变化的反应能力。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Muscle & Nerve 医学-临床神经学

CiteScore

6.40

自引率

5.90%

发文量

287

审稿时长

3-6 weeks

期刊介绍： Muscle & Nerve is an international and interdisciplinary publication of original contributions, in both health and disease, concerning studies of the muscle, the neuromuscular junction, the peripheral motor, sensory and autonomic neurons, and the central nervous system where the behavior of the peripheral nervous system is clarified. Appearing monthly, Muscle & Nerve publishes clinical studies and clinically relevant research reports in the fields of anatomy, biochemistry, cell biology, electrophysiology and electrodiagnosis, epidemiology, genetics, immunology, pathology, pharmacology, physiology, toxicology, and virology. The Journal welcomes articles and reports on basic clinical electrophysiology and electrodiagnosis. We expedite some papers dealing with timely topics to keep up with the fast-moving pace of science, based on the referees'' recommendation.