应用磁共振指纹技术评估贝克型肌营养不良症的细胞外容积分数。
Assessment of Extracellular Volume Fraction in Becker Muscular Dystrophy by Using MR Fingerprinting.
机构信息
From the Institute of Myology, Neuromuscular Investigation Center, NMR Laboratory, Bâtiment Babinski, Groupe Hospitalier Pitié-Salpêtrière, 47-83 boulevard Vincent Auriol, 75651 Paris Cedex 13, France (B.M., P.Y.B., E.C.d.A.A., Y.F., H.R.); and Institute of Myology, Reference Center for Muscle Diseases Paris-Est, Paris, France (K.W.).
出版信息
Radiology. 2023 May;307(3):e221115. doi: 10.1148/radiol.221115. Epub 2023 Mar 7.
Background Quantitative MRI is increasingly proposed in clinical trials related to dystrophinopathies, including Becker muscular dystrophy (BMD). Purpose To establish the sensitivity of extracellular volume fraction (ECV) quantification using an MR fingerprinting sequence with water and fat separation as a quantitative imaging biomarker of skeletal muscle tissue alterations in BMD compared with fat fraction (FF) and water relaxation time quantification. Materials and Methods In this prospective study, study participants with BMD and healthy volunteers were included from April 2018 until October 2022 ( identifier NCT02020954). The MRI examination comprised FF mapping with the three-point Dixon method, water T2 mapping, and water T1 mapping before and after an intravenous injection of a gadolinium-based contrast agent by using MR fingerprinting, from which ECV was calculated. Functional status was measured with use of the Walton and Gardner-Medwin scale. This clinical evaluation tool stratifies disease severity from grade 0 (preclinical; elevated creatine phosphokinase; all activities normal) to grade 9 (unable to eat, drink, or sit without assistance). Mann-Whitney tests, Kruskal-Wallis tests, and Spearman rank correlation tests were performed. Results Twenty-eight participants with BMD (median age, 42 years [IQR, 34-52 years]; 28 male) and 19 healthy volunteers (median age, 39 years [IQR, 33-55 years]; 19 male) were evaluated. ECV was higher in participants with dystrophy than in controls (median, 0.21 [IQR, 0.16-0.28] vs 0.07 [IQR, 0.07-0.08]; < .001). In muscles of participants with BMD with normal FF, ECV was also higher than in muscles of healthy controls (median, 0.11 [IQR, 0.10-0.15] vs 0.07 [IQR, 0.07-0.08]; = .02). ECV was correlated with FF (ρ = 0.56, = .003), Walton and Gardner-Medwin scale score (ρ = 0.52, = .006), and serum cardiac troponin T level (ρ = 0.60, < .001). Conclusion Quantitative MR relaxometry with water and fat separation indicates a significant increase of skeletal muscle extracellular volume fraction in study participants with Becker muscular dystrophy. Clinical trial registration no. NCT02020954 Published under a CC BY 4.0 license.
背景 定量 MRI 越来越多地应用于与肌营养不良症相关的临床试验中，包括 Becker 型肌营养不良症（BMD）。目的 与脂肪分数（FF）和水弛豫时间定量相比，使用带水脂分离的磁共振指纹序列定量评估骨骼肌组织改变，以建立细胞外容积分数（ECV）定量成像生物标志物的灵敏度。材料与方法 本前瞻性研究纳入 2018 年 4 月至 2022 年 10 月期间的 BMD 患者和健康志愿者（标识符 NCT02020954）。MRI 检查包括采用三点 Dixon 方法进行 FF 制图、水 T2 制图、水 T1 制图，以及静脉注射钆类造影剂前后使用磁共振指纹图谱进行 ECV 计算。使用 Walton 和 Gardner-Medwin 量表评估功能状态。该临床评估工具将疾病严重程度从 0 级（临床前；肌酸磷酸激酶升高；所有活动正常）到 9 级（无法进食、饮水或无辅助情况下坐立）进行分层。进行了 Mann-Whitney U 检验、Kruskal-Wallis 检验和 Spearman 秩相关检验。结果 共评估了 28 名 BMD 患者（中位年龄，42 岁[四分位距，34-52 岁]；28 名男性）和 19 名健康志愿者（中位年龄，39 岁[四分位距，33-55 岁]；19 名男性）。与对照组相比，肌营养不良症患者的 ECV 更高（中位数，0.21[四分位距，0.16-0.28] vs 0.07[四分位距，0.07-0.08]；<0.001）。在 BMD 患者的正常 FF 肌肉中，ECV 也高于健康对照组的肌肉（中位数，0.11[四分位距，0.10-0.15] vs 0.07[四分位距，0.07-0.08]；=0.02）。ECV 与 FF（ρ=0.56，=0.003）、Walton 和 Gardner-Medwin 量表评分（ρ=0.52，=0.006）和血清心肌肌钙蛋白 T 水平（ρ=0.60，<0.001）相关。结论 带水脂分离的定量 MRI 弛豫度表明，Becker 型肌营养不良症患者的骨骼肌细胞外容积分数显著增加。临床试验注册号 NCT02020954 基于 CC BY 4.0 许可发布。
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