Electrical impedance myography for assessment of Duchenne muscular dystrophy.
Electrical impedance myography for assessment of Duchenne muscular dystrophy.
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DOI：
10.1002/ana.24874
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发表时间：
2017-05
影响因子：
11.2
通讯作者：
Darras BT
中科院分区：
文献类型：
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作者：
Rutkove SB;Kapur K;Zaidman CM;Wu JS;Pasternak A;Madabusi L;Yim S;Pacheck A;Szelag H;Harrington T;Darras BT
杜氏肌营养不良（DMD）的临床试验需要敏感、客观和易于应用的方法来评估疾病进展和对治疗的反应。在这项研究中，我们评估了电阻抗肌图（EIM）是否可以达到这一目的。在这项非盲法研究中，36名患有DMD的男孩和29名年龄相似的健康男孩在长达2年的时间里对6块肌肉进行了多频EIM测量，并进行了功能评估。采用随机截距和斜率项的线性混合效应模型对多频EIM值和功能测度进行了分析。7名DMD男孩开始使用皮质类固醇；这些数据使用分段线性混合效应模型进行分析。在0 ~ 7.0岁的男孩中，上肢EIM相比轨迹的斜率在6个月时观察到显著差异-0.074/月，p=0.023, 95%可信区间（CI）[- 0.013, - 0.14]；两年时，这一差异为- 0.048/月，p<0.0001 95%CI[- 0.028, - 0.068]。在≤7.0岁的男孩中，6个月腓肠肌出现差异（EIM相斜率为- 0.83°/kHz-month, p=0.007 95%CI[- 0.26, - 1.40]）。EIM结果比功能测试更早显示出显著差异。开始使用皮质类固醇可显著改善EIM相比斜率（0.057°/月，p=0.00019 95%CI[0.028,0.086]）和EIM相斜率（0.14°/ khz -月，p=0.013 95%CI[0.028,0.25]），与皮质类固醇已知的临床益处一致。EIM在6个月时检测到小男孩和大男孩的肌肉退化；它还确定了皮质类固醇起始的治疗效果。由于EIM应用迅速，无痛，并且需要最少的操作员培训，因此该技术值得在DMD临床治疗试验中作为生物标志物进行进一步评估。
Sensitive, objective and easily applied methods for evaluating disease progression and response to therapy are needed for clinical trials in Duchenne muscular dystrophy (DMD). In this study, we evaluated whether electrical impedance myography (EIM) could serve this purpose. In this non-blinded study, 36 boys with DMD and 29 age-similar healthy boys underwent multifrequency EIM measurements for up to 2 years on 6 muscles unilaterally along with functional assessments. A linear mixed-effects model with random intercept and slope terms was used for the analysis of multifrequency EIM values and functional measures. Seven DMD boys were initiated on corticosteroids; these data were analyzed using a piecewise linear mixed-effects model. In boys >7.0 years, a significant difference in the slope of EIM phase-ratio trajectories in the upper extremity was observed by 6 months of -0.074/month, p=0.023, 95% confidence interval (CI)[−0.013,−0.14]); at two years, this difference was −0.048/month, p<0.0001 95%CI[−0.028,−0.068]. In boys ≤7.0 years, differences appeared at 6 months in gastrocnemius (EIM phase-slope −0.83°/kHz-month, p=0.007 95%CI[−0.26,−1.40]). EIM outcomes showed significant differences earlier than functional tests. Initiation of corticosteroids significantly improved the slope of EIM phase-ratio (0.057/month, p=0.00019 95%CI[0.028,0.086]) and EIM phase-slope (0.14°/kHz-month, p=0.013 95%CI[0.028,0.25]), consistent with corticosteroids’ known clinical benefit. EIM detects deterioration in muscles of both younger and older boys by 6 months; it also identifies the therapeutic effect of corticosteroid initiation. Since EIM is rapid to apply, painless, and requires minimal operator training, the technique deserves to be further evaluated as a biomarker in DMD clinical therapeutic trials.