面肩肱型肌营养不良功能综合结局指标
Facioscapulohumeral muscular dystrophy functional composite outcome measure.
作者信息
Eichinger Katy, Heatwole Chad, Iyadurai Stanley, King Wendy, Baker Lindsay, Heininger Susanne, Bartlett Amy, Dilek Nuran, Martens William B, Mcdermott Michael, Kissel John T, Tawil Rabi, Statland Jeffrey M
机构信息
Department of Neurology, University of Rochester Medical Center, 601 Elmwood Avenue, Box 673 Rochester, New York, 14642, USA.
Department of Neurology, The Ohio State University Wexner Medical Center, Columbus, Ohio, USA.
出版信息
Muscle Nerve. 2018 Jan 30. doi: 10.1002/mus.26088.
INTRODUCTION
We developed an evaluator-administered functional facioscapulohumeral muscular dystrophy composite outcome measure (FSHD-COM) comprising patient-identified areas of functional burden for future clinical trials.
METHODS
We performed a prospective observational study of 41 patients with FSHD at 2 sites. The FSHD-COM includes functional assessment of the legs, shoulders and arms, trunk, hands, and balance/mobility. We determined the test-retest reliability and convergent validity compared to established FSHD disease metrics.
RESULTS
The FSHD-COM demonstrated excellent test-retest reliability (intraclass correlation coefficient [ICC] 0.96; subscale ICC range, 0.90-0.94). Cross-sectional associations between the FSHD-COM and disease duration, clinical severity, and strength were moderate to strong (Pearson correlation coefficient range |0.51-0.92|).
DISCUSSION
The FSHD-COM is a disease-relevant, functional composite outcome measure suitable for future FSHD clinical trials that shows excellent test-retest reliability and cross-sectional associations to disease measures. Future directions include determining multisite reliability, sensitivity to change, and the minimal clinically important change in the FSHD-COM. Muscle Nerve, 2018.
引言
我们开发了一种由评估者实施的功能性面肩肱型肌营养不良综合结局指标（FSHD-COM），该指标包含患者确定的功能负担领域，用于未来的临床试验。
方法
我们在2个地点对41例FSHD患者进行了一项前瞻性观察研究。FSHD-COM包括对腿部、肩部和手臂、躯干、手部以及平衡/移动能力的功能评估。我们将其与已确立的FSHD疾病指标进行比较，确定了重测信度和收敛效度。
结果
FSHD-COM表现出出色的重测信度（组内相关系数[ICC]为0.96；各子量表ICC范围为0.90 - 0.94）。FSHD-COM与疾病持续时间、临床严重程度和肌力之间的横断面关联为中度至高度（皮尔逊相关系数范围为|0.51 - 0.92|）。
讨论
FSHD-COM是一种与疾病相关的功能性综合结局指标，适用于未来的FSHD临床试验，显示出出色的重测信度以及与疾病指标的横断面关联。未来的方向包括确定多地点信度、对变化的敏感性以及FSHD-COM中最小临床重要变化。《肌肉与神经》，2018年。
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