Taruta Atsuki, Hiyoshi Tetsuaki, Harada Akina, Nakashima Masato
Neuroscience Translational Medicine, Neuroscience Drug Discovery Unit, Research, Takeda Pharmaceutical Company Limited, 26-1, Muraoka-Higashi 2-chome, Fujisawa, Kanagawa 251-8555, Japan.
Muscular Disease and Neuropathy Unit, Neuroscience Drug Discovery Unit, Research, Takeda Pharmaceutical Company Limited, 26-1, Muraoka-Higashi 2-chome, Fujisawa, Kanagawa 251-8555, Japan.
Exp Neurol. 2025 Mar;385:115111. doi: 10.1016/j.expneurol.2024.115111. Epub 2024 Dec 10.
Charcot-Marie-Tooth type 1A (CMT1A) is the most common inherited peripheral dysmyelinating neuropathy. Although lower limb muscle weakness is the most important factor affecting the quality of life of patients with CMT1A, existing clinical measures for its evaluation have limitations, including low sensitivity in detecting disease progression. Electrical impedance myography (EIM) is a newer tool that enables noninvasive evaluation of muscle state by measuring muscle composition, and potentially supports the evaluation of neuromuscular disease progression and treatment effects. To determine the potential of EIM as a CMT1A biomarker, we obtained natural history data for EIM from the gastrocnemius muscle of the PMP22-C3 mice, an animal model of CMT1A. Alterations in the EIM parameters, weak hindlimb grip strength, decreased muscle fiber size, and changes in the mRNA expression of genes related to neuromuscular junction dysfunction were found. These changes were more pronounced at later stages (12 and 18 weeks of age) than at earlier stage (6 weeks of age), indicating that EIM can detect disease progression in PMP22-C3 mice. Our preclinical findings support the use of EIM as a potential translational biomarker for assessing progressive changes in the pathological muscle state in CMT1A.
1型遗传性运动感觉神经病A亚型(CMT1A)是最常见的遗传性周围脱髓鞘性神经病。虽然下肢肌肉无力是影响CMT1A患者生活质量的最重要因素,但现有的评估其临床方法存在局限性,包括在检测疾病进展方面敏感性较低。电阻抗肌电图(EIM)是一种更新的工具,可通过测量肌肉成分对肌肉状态进行无创评估,并可能有助于评估神经肌肉疾病的进展和治疗效果。为了确定EIM作为CMT1A生物标志物的潜力,我们从CMT1A动物模型PMP22-C3小鼠的腓肠肌中获取了EIM的自然史数据。发现EIM参数改变、后肢握力减弱、肌纤维大小减小以及与神经肌肉接头功能障碍相关基因的mRNA表达变化。这些变化在后期(12周和18周龄)比早期(6周龄)更为明显,表明EIM可以检测PMP22-C3小鼠的疾病进展。我们的临床前研究结果支持将EIM用作评估CMT1A病理性肌肉状态进行性变化的潜在转化生物标志物。