Quantitative muscle ultrasound detects disease progression in Duchenne muscular dystrophy.
Quantitative muscle ultrasound detects disease progression in Duchenne muscular dystrophy.
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DOI：
10.1002/ana.24904
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发表时间：
2017-05
影响因子：
11.2
通讯作者：
Rutkove SB
中科院分区：
文献类型：
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作者：
Zaidman CM;Wu JS;Kapur K;Pasternak A;Madabusi L;Yim S;Pacheck A;Szelag H;Harrington T;Darras BT;Rutkove SB
我们评估了患有杜氏肌营养不良症 (DMD) 的男孩和健康对照组的定量肌肉超声数据的变化，以确定超声是否可以作为疾病进展的生物标志物。使用了两种方法：从超声图像测量的灰度级（GSL），以及直接从接收到的回波测量的定量反向散射分析（QBA）。 GSL 和 QBA 是从 36 名患有 DMD 的男孩和 28 名健康男孩（2-14 岁）的 6 个单侧手臂/腿部肌肉中获得的，持续时间长达 2 年。我们使用具有随机截距和斜率项的线性混合效应模型来比较 GSL、QBA 和功能评估的轨迹。我们单独分析了一部分开始使用皮质类固醇的男孩。与健康男孩相比，>7.0 岁的 DMD 男孩的 GSL 增加在 6 个月时首次被发现（例如，前臂斜率差异为 1.16 任意单位/月 p=0.004，95% 置信区间 (CI) [0.38,1.94]）；在≤7岁的男孩中，GSL差异在12个月时首次出现（股直肌为0.82任意单位/月，p=0.04 95%CI [0.075,1.565]）。 QBA 的表现与 GSL 类似（例如，>7 岁的 DMD 男孩为 0.41dB/月，p=0.01，6 个月时前臂前臂 95%CI [0.096,0.72]）。美国比功能测量（包括 6 分钟步行和仰卧站立测试）更早发现差异。然而，QBA 和 GSL 均未显示皮质类固醇起始的效果。 QBA 的表现与 GSL 类似，并且在检测 DMD 中的肌肉退化方面，两者都比功能评估更敏感。还需要进行更多研究来确定定量肌肉超声是否可以检测治疗效果。
We assessed changes in quantitative muscle ultrasound data in boys with Duchenne muscular dystrophy (DMD) and healthy controls to determine if ultrasound can serve as a biomarker of disease progression. Two approaches were used: grey scale level (GSL), measured from the ultrasound image, and quantitative backscatter analysis (QBA) measured directly from the received echoes. GSL and QBA were obtained from six unilateral arm/leg muscles in 36 boys with DMD and 28 healthy boys (age 2–14 years) for up to 2 years. We used a linear mixed-effects model with random intercept and slope terms to compare trajectories of GSL, QBA, and functional assessments. We analyzed separately a subset of boys who initiated corticosteroids. Compared to healthy boys, increasing GSL in DMD boys >7.0 years was first identified at 6 months (e.g., anterior forearm slope difference of 1.16 arbitrary units/month p=0.004, 95% confidence interval (CI) [0.38,1.94]); in boys ≤7 years, differences in GSL first appeared at 12 months (0.82 arbitrary units /month, p=0.04 95%CI [0.075,1.565] in rectus femoris). QBA performed similarly to GSL (e.g., DMD boys >7 years of 0.41dB/month, p=0.01, 95%CI [0.096,0.72] in anterior forearm at 6 months). US identified differences earlier than functional measures including 6-minute walk and supine-to-stand tests. However, neither QBA nor GSL showed an effect of corticosteroid initiation. QBA performs similarly to GSL and both appear more sensitive than functional assessments for detecting muscle deterioration in DMD. Additional studies will be required to determine if quantitative muscle ultrasound can detect therapeutic efficacy.