184超声与MRI对杜氏肌营养不良患者EDB肌的评价

IF 2.8 4区医学 Q2 CLINICAL NEUROLOGY

Neuromuscular Disorders Pub Date : 2025-09-01 DOI:10.1016/j.nmd.2025.105537

P. Karachunski, J. Martone, M. Stark, D. Nascene, T. Nordseth, P. Kang

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引用次数: 0

摘要

尽管最近在杜氏肌营养不良症（DMD）的疾病修饰疗法方面取得了进展，但治疗效果仍然有限。需要新的方法来恢复肌肉纤维，更好地保持肌肉结构和功能。在临床试验中识别和频繁监测适当的肌肉需要非侵入性床边技术，如超声（US）。为了实现这一目标，我们研究了使用US评估DMD患者指短伸肌（EDB）变化的可行性，并将这些结果与MRI相关联。EDB肌是首次在人体治疗研究中可接近的靶肌。我们比较了EDB和胫骨前肌（TA）肌肉变化的严重程度。7例年龄在10岁以上的DMD患者行脑电导超声检查，并使用ImageJ软件进行灰度分析定量评估。其中，5名参与者也接受了EDB肌肉的MRI检查，由放射科医生对肌肉受累程度进行半定量分级。四名参与者同时接受了超声和MRI评估。4名受试者接受脑电成像功能评估测试，并对所有受试者的活动状态进行评估。对比分析US和MR EDB肌肉图像显示，相关决定系数（r2）为0.58。100%的患者TA的灰度值高于EDB，（t(5) = 3.14,p = 0.0257）。通过US和MRI测量的肌肉体积可比较。功能评估，包括运动状态和握力与EDB平均灰度值负相关。这项初步研究提供了超声与MRI结果和功能谱相关的证据，证明了超声在临床试验中作为监测疾病进展和治疗反应的可靠、非侵入性工具的价值。该研究还强调了EDB肌的延迟受累，使其适用于晚期疾病患者甚至非活动DMD个体的早期临床试验。

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184PUltrasound vs MRI for assessing the EDB muscle in Duchenne muscular dystrophy

Despite recent advances in disease modifying therapies for Duchenne muscular dystrophy (DMD), treatment efficacy remains limited. New approaches are needed to restore muscle fibers and better preserve muscle structure and function. Identification and frequent monitoring of appropriate muscles in clinical trials require non-invasive bedside techniques such as ultrasound (US). To meet this goal, we studied the feasibility of using US to assess changes in the extensor digitorum brevis (EDB) muscle in DMD patients and correlated these findings with MRI. The EDB muscle is an accessible target muscle for first-in-human therapeutic studies. We compared the severity of muscle changes in the EDB and tibialis anterior (TA) muscles. Seven DMD patients, aged over 10 years, underwent EDB ultrasound and were quantitatively assessed using grayscale analysis with ImageJ software. Among these, five participants also underwent MRI of the EDB muscle, with muscle involvement graded semi-quantitatively by a radiologist. Four participants underwent both US and MRI evaluations. Four participants with EDB images underwent functional assessment testing, and all participants’ ambulatory status was assessed. Comparative analysis of US and MR EDB muscle images showed correlation with a coefficient of determination (R 2) of 0.58. The TA exhibited a higher grayscale value than the EDB in 100% of patients, (t(5) = 3.14, p = 0.0257). Muscle volumes measured by US and MRI were comparable. Functional assessments, including ambulatory status and hand-grip strength correlated negatively with the mean EDB gray-scale values. This pilot study provides evidence that US correlates with MRI findings and functional profiles, demonstrating the value of US as a reliable, non-invasive tool for monitoring disease progression and treatment responses in clinical trials. This study also highlighted the delayed involvement of the EDB muscle, making it suitable for early clinical trials involving patients with advanced disease and even non-ambulatory DMD individuals.

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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.