178PTwo-year stride-level evaluation of ambulatory function in ambulant DMD patients above 4 years old

IF 2.8 4区医学 Q2 CLINICAL NEUROLOGY

Neuromuscular Disorders Pub Date : 2025-09-01 DOI:10.1016/j.nmd.2025.105531

M. Poleur , G. Parinello , E. Vrščaj , A. Dolanska , P. Nowakowski , C. Anghelescu , L. Szabo , M. Leanca , A. Mirea , S. Kodsy , A. Saleh , D. Osredkar , J. Haberlova , A. Potulska-Chromik , N. Butoianu , P. Delmar , P. Strijbos , D. Eggenspieler , L. Servais , the ActiLiège-Next study group

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引用次数: 0

Abstract

Duchenne muscular dystrophy (DMD) is characterized by severe and progressive muscle weakness. Assessing investigational drug efficacy in a reasonable timeframe is challenging due to a lack of objective, reliable, and sensitive outcome measures. The wearable sensor ActiMyo/Syde was developed to assess motor function accurately and precisely at the stride level during daily life. One variable derived from these sensor data, SV95C (measuring the 5% most rapid strides), was recently qualified as primary endpoint for ambulant DMD by the European Medicines Agency. To gather 3-year longitudinal functional data using this sensor, ambulant DMD patients and controls were enrolled in the ActiLiège-Next study. Patients were asked to wear sensors at both ankles daily during the first 3-12 months and for 1 month every 3 months afterwards, and controls were asked to wear them for 1 month every 12 months. Eighty-seven ambulant DMD patients aged 4 to 14 years (median: 8.0y) and 37 controls (9.8y) were enrolled. As of Jan 2025, >95% of patients showed good adherence with sensor wear at baseline. SV95C reliability was excellent, with an intraclass correlation coefficient of 0.96 for patients and 0.87 for controls. The Spearman correlation with North Star ambulatory assessment, 6-minute walk test, 4-stair climbing test and time to rise from floor was 0.65, 0.54, -0.71 and -0.66, respectively (n=76-81; p<0.001). For patients between 4 and 8 years old, mean SV95C improved from baseline at 6 months (0.08m/s, SRM [standardized response mean]=0.55, n=37), and then declined at 12 and 18 months (-0.06m/s, SRM=ns, n=27; and -0.13m/s, SRM=-0.52, n=18, respectively). Patients ≥8 years old experienced a marked decline at 6, 12 and 18 months: -0.09m/s (SRM=-0.51, n=30), -0.15m/s (SRM=-0.99, n=24) and -0.24m/s (SRM=-2.10, n=16), respectively. These data confirm the excellent reliability, external validity and responsiveness of SV95C in ambulant DMD above 4 years old. All available 18- and 24-month data will be shared at the congress.

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178p4岁以上非卧床DMD患者2年行走功能跨步水平评价

杜氏肌营养不良症（DMD）的特点是严重和进行性肌肉无力。由于缺乏客观、可靠和敏感的结果测量，在合理的时间范围内评估研究药物的疗效具有挑战性。可穿戴传感器ActiMyo/Syde是为了在日常生活中准确准确地评估运动功能而开发的。从这些传感器数据中得出的一个变量SV95C（测量5%最快步幅）最近被欧洲药品管理局认定为动态DMD的主要终点。为了使用该传感器收集3年的纵向功能数据，活动DMD患者和对照组被纳入actili - next研究。患者被要求在前3-12个月内每天在双脚踝佩戴传感器，之后每3个月佩戴1个月，对照组被要求每12个月佩戴1个月。87例4 - 14岁的DMD患者（中位数：8.0岁）和37例对照组（9.8岁）被纳入研究。截至2025年1月，95%的患者在基线时表现出良好的传感器佩戴依从性。SV95C的可靠性非常好，患者的类内相关系数为0.96，对照组为0.87。与North Star动态活动评估、6分钟步行测试、爬4级楼梯测试和从地板上升时间的Spearman相关性分别为0.65、0.54、-0.71和-0.66 （n=76 ~ 81; p<0.001）。对于4 - 8岁的患者，平均SV95C在6个月时较基线有所改善（0.08m/s， SRM[标准化反应平均值]=0.55,n=37），然后在12和18个月时下降（-0.06m/s， SRM=ns, n=27; -0.13m/s， SRM=-0.52, n=18）。≥8岁的患者在6、12、18个月时出现明显下降，分别为-0.09m/s （SRM=-0.51, n=30）、-0.15m/s （SRM=-0.99, n=24）、-0.24m/s （SRM=-2.10, n=16）。这些数据证实了SV95C在4岁以上的动态DMD中具有优异的信度、外部效度和响应性。所有18个月和24个月的可用数据将在大会上共享。

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来源期刊

Neuromuscular Disorders 医学-临床神经学

CiteScore

4.60

自引率

3.60%

发文量

543

审稿时长

53 days

期刊介绍： This international, multidisciplinary journal covers all aspects of neuromuscular disorders in childhood and adult life (including the muscular dystrophies, spinal muscular atrophies, hereditary neuropathies, congenital myopathies, myasthenias, myotonic syndromes, metabolic myopathies and inflammatory myopathies). The Editors welcome original articles from all areas of the field: • Clinical aspects, such as new clinical entities, case studies of interest, treatment, management and rehabilitation (including biomechanics, orthotic design and surgery). • Basic scientific studies of relevance to the clinical syndromes, including advances in the fields of molecular biology and genetics. • Studies of animal models relevant to the human diseases. The journal is aimed at a wide range of clinicians, pathologists, associated paramedical professionals and clinical and basic scientists with an interest in the study of neuromuscular disorders.