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使用定量MRI对CMT1A患者的一年纵向评估

Etienne Fortanier¹, Marc Adrien Hostin¹, Constance Michel¹

¹From the Reference Center for Neuromuscular Diseases and ALS (E.F., E.D., S.A.), La Timone University Hospital, Center for Magnetic Resonance in Biology and Medicine (M.A.H., C.M., M.G., D.B.), UMR CNRS 7339, UMR 7286 (E.D.), Medicine Faculty, CNRS, LIS (M.A.H.,M.-E.B.), and Inserm (S.A.), GMGF, Aix-Marseille University, France.

|April 17, 2024

概括

定量MRI (qMRI) 显示,随着时间的推移,Charcot-Marie-Tooth型1A (CMT1A) 患者的肌肉内脂肪分数 (FF) 正在增加. 这一FF指标与疾病严重程度有很强的相关性,支持其在临床试验中的使用.

科学领域:

生物医学成像技术生物医学成像技术
神经学神经学
量化MRI是指数量化的MRI.

背景情况:

查尔科-玛丽-牙1A型 (CMT1A) 是一种渐进的外围神经病变.
通过定量MRI (qMRI) 测量肌肉内脂肪分数 (FF) 是CMT1A.的敏感结果指标.
对qMRI生物标志物的纵向跟踪对于CMT1A的临床试验开发至关重要.

研究的目的:

评估多个qMRI生物标志物的相关性,以跟踪CMT1A的纵向变化.
评估CMT1A患者的qMRI指标和临床参数之间的相关性.

主要方法:

量化MRI (qMRI) 对CMT1A患者进行了两次时间点,间隔一年.
包括质子密度 (PD),磁化转移比率 (MTR) 和肌内脂肪分数 (FF) 在内的指标从大腿和腿部感兴趣的3D体积中提取出来.
使用查科玛丽牙神经学评分 (CMTNSv2),整体神经病症限制量表和医学研究委员会 (MRC) 肌肉力量来评估疾病的严重程度.

主要成果:

一年来,肌肉内脂肪分数 (FF) 在大腿 (+1.04%) 和腿部 (+1.36%) 3D体积中显著增加.
观察到FF的长度依赖的梯度,与大腿相比,腿部的值更高.
在FF和临床测量之间发现了显著的相关性:CMTNSv2 (ρ = 0.656) 和下肢MRC强度 (ρ = -0.877).

结论:

定量核磁共振 (qMRI),特别是肌内脂肪分数 (FF),是监测CMT1A.纵向疾病进展的有希望的生物标志物.
三维体积分析比单片分析更适合,因为FF分布和肌肉特异性变化各不相同.
观察到的每年最小变化需要谨慎的数据提取方法,以便可靠的临床试验应用.

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