177PLongitudinal stride-level evaluation of ambulatory function with ankle wearable technology in ambulant DMD patients below 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.2025.105530

M. Poleur , G. Parinello , E. Vrščaj , C. Bisson , C. Cluzeau , A. Daron , University Children's Hospital L , D. Osredkar , P. Strijbos , D. Eggenspieler , L. Servais

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引用次数: 0

Abstract

Assessing ambulation in patients with Duchenne muscular dystrophy (DMD) below 4 years old (yo) is challenging with available in-clinic outcomes, yet crucial for therapeutic development. Using a wearable device to accurately measure ambulation in daily life, the European Medicines Agency recently qualified SV95C (measuring the 5% most rapid strides) as primary endpoint in ambulant DMD ≥4 yo. The ActiLiège-Next study enrolled patients and controls to assess adherence to device wear, and SV95C robustness & sensitivity in children 1-4 yo. Patients were asked to wear 2 ankle sensors daily for the first 3 months and then for 1 month every 3 months, and controls for 1 month every 6 months. Twenty-six patients (median age, min-max: 36 months, 16-47) and 32 controls (31 months, 13-48) were enrolled. Three patients were on steroid before enrolment, 2 started upon enrolment and 3 started after the 6-month visit. As of Jan 2025, all but one patients (n=25) and all controls (n=32) showed good adherence to sensor wear. SV95C reliability was excellent with an intraclass correlation coefficient of 0.99 for patients (n=22) and controls (n=30). Mean baseline SV95C was significantly different between the 2 groups (p<0.005). Available 6- and 12-month data suggested a stronger SV95C increase for controls than patients: mean change from baseline was 0.21m/s (standardized response mean, SRM=0.87, n=15) and 0.35m/s (SRM=3.33, n=6) for controls, and 0.12m/s (SRM=0.68, n=23) and 0.18m/s (SRM=1.53, n=7) for patients, respectively. For the 3 patients starting steroid after the 6-month visit, mean SV95C decreased by -0.2m/s/year before starting steroids and increased by 1.13m/s/year after starting steroids. All available longitudinal data and steroid effect on SV95C change will be shared at the congress. These results strongly suggest that SV95C could be the first robust functional motor measure that can be used in all ambulant DMD including those <4 yo, and enable development of treatments or therapeutic strategy in this age group.

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1774岁以下动态DMD患者踝部可穿戴技术纵向步幅水平的动态功能评价

评估4岁以下杜氏肌营养不良症（DMD）患者的活动能力是一项具有挑战性的临床结果，但对治疗发展至关重要。使用可穿戴设备精确测量日常生活中的行走，欧洲药品管理局最近批准SV95C（测量5%最快步幅）作为行走DMD≥4岁的主要终点。actili - next研究纳入了患者和对照组，以评估1-4岁儿童对设备佩戴的依从性和SV95C的稳健性和敏感性。患者被要求在前3个月每天佩戴2个脚踝传感器，然后每3个月佩戴1个月，对照组每6个月佩戴1个月。纳入26例患者（中位年龄，最小-最大年龄：36个月，16-47个月）和32例对照组（31个月，13-48个月）。3例患者在入组前使用类固醇，2例在入组时使用，3例在随访6个月后使用。截至2025年1月，除1名患者（n=25）和所有对照组（n=32）外，所有患者均表现出良好的传感器佩戴依从性。SV95C的可靠性非常好，患者（n=22）和对照组（n=30）的类内相关系数为0.99。两组患者平均基线SV95C差异有统计学意义（p<0.005）。现有的6个月和12个月数据显示，对照组的SV95C比患者有更强的增加：与基线相比，对照组的平均变化为0.21m/s（标准化反应平均值，SRM=0.87, n=15）和0.35m/s (SRM=3.33, n=6)，而患者的平均变化为0.12m/s （SRM=0.68, n=23）和0.18m/s （SRM=1.53, n=7）。随访6个月后开始使用类固醇的3例患者，开始使用类固醇前平均SV95C下降-0.2m/s/年，开始使用类固醇后平均SV95C增加1.13m/s/年。所有可用的纵向数据和类固醇对SV95C变化的影响将在大会上分享。这些结果强烈表明，SV95C可能是第一个可用于所有动态DMD（包括4岁）的强大功能运动测量，并为该年龄组的治疗或治疗策略的开发提供了可能。

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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.