杜氏肌营养不良患者运动健康状态的描述性特征：运动功能轨迹和运动能力丧失的时间。

IF 3.4 4区医学 Q2 CLINICAL NEUROLOGY

Journal of neuromuscular diseases Pub Date : 2025-09-23 DOI:10.1177/22143602251364694

Francesco Muntoni, James Signorovitch, Michaela Johnson, Andres Gomez-Lievano, Nate Posner, Patricia Dorling, Katherine Beaverson, Jose Alvir, Matthias Mahn, Susan J Ward, Nathalie Goemans, Krista Vandenborne, Eugenio Mercuri, Craig M McDonald

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引用次数: 0

摘要

我们描述了动态杜氏肌营养不良症（DMD）的进展，通过多种功能测量，通过先前建立的行走能力丧失（LoA）和健康状态的预后组。离LoA较近的患者与离LoA较远的患者相比，由于地板效应，某些指标（如6分钟步行距离）的下降幅度较大，而其他指标（如从地板速度计时上升）的变化较小。晚期和早期流动健康状态的患者一致向较高的LoA风险转移。研究结果进一步表征了动态DMD的健康状态和预后因素，并强调了多种功能测量对充分表征疾病进展的重要性。

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Descriptive characterization of ambulatory health states in Duchenne muscular dystrophy: Motor function trajectories and times to loss of ambulation.

We described ambulatory Duchenne muscular dystrophy (DMD) progression, across multiple functional measures, via previously established prognostic groups for loss of ambulation (LoA) and health states. Patients closer to vs. farther from LoA had greater declines in some measures (e.g., 6-min walk distance) and less change in others (e.g., timed rise from floor velocity) due to floor effects. Patients in the late vs. early ambulatory health state were concordantly shifted towards higher LoA risk. Findings further characterize health states and prognostic factors in ambulatory DMD and highlight the importance of multiple measures of function to fully characterize disease progression.

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来源期刊

Journal of neuromuscular diseases Medicine-Neurology (clinical)

CiteScore

5.10

自引率

6.10%

发文量

102

期刊介绍： The Journal of Neuromuscular Diseases aims to facilitate progress in understanding the molecular genetics/correlates, pathogenesis, pharmacology, diagnosis and treatment of acquired and genetic neuromuscular diseases (including muscular dystrophy, myasthenia gravis, spinal muscular atrophy, neuropathies, myopathies, myotonias and myositis). The journal publishes research reports, reviews, short communications, letters-to-the-editor, and will consider research that has negative findings. The journal is dedicated to providing an open forum for original research in basic science, translational and clinical research that will improve our fundamental understanding and lead to effective treatments of neuromuscular diseases.