Longitudinal study of multi-parameter quantitative magnetic resonance imaging in Duchenne muscular dystrophy: hyperresponsiveness of gluteus maximus and detection of subclinical disease progression in functionally stable patients
Longitudinal study of multi-parameter quantitative magnetic resonance imaging in Duchenne muscular dystrophy: hyperresponsiveness of gluteus maximus and detection of subclinical disease progression in functionally stable patients
复制标题
杜氏肌营养不良症多参数定量磁共振成像的纵向研究：臀大肌高反应性和功能稳定患者亚临床疾病进展的检测
DOI：
10.1007/s00415-022-11470-8
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发表时间：
2022-11-16
影响因子：
6
通讯作者：
Gong, Lianggeng
中科院分区：
文献类型：
--
作者：
Peng, Fei;Xu, Huayan;Gong, Lianggeng
目的应用多参数定量磁共振成像（qMRI）技术描述骨盆和大腿肌肉Duchenne肌营养不良症（DMD） 1年内的疾病进展，确定功能稳定患者最敏感的肌肉并预测亚临床疾病进展。方法54例DMD患者（平均年龄8.9±2.5岁，年龄范围5-15岁）完成基线和1年随访qMRI检查/生物标志物[3点Dixon/脂肪分数（FF）]；T1映射/ T1;T2映射/ T2]。同时对DMD患者进行临床评估[NorthStar动态评估（NSAA）评分]和定时功能测试。24名健康男性对照（5-15岁）完成了qMRI基线检查。采用Wilcoxon检验比较组间差异。采用标准化反应均值（SRM）作为对疾病进展指数的反应性。结果DMD各年龄组的ff、T1、T2在1年内变化均有统计学意义（P< 0.05）。即使在功能稳定的患者（NSAA评分升高、不变或降低1分）1年内，臀大肌（GMa）、臀中肌、股外侧肌和大收肌的FF和T2显著升高，T1显著降低（P< 0.05）。总体而言，FF、T1和T2的SRM在GMa中均最高，分别为1.25、- 0.92和0.93。结论sqmri生物标志物对疾病进展有反应，也可以检测功能稳定的DMD患者1年以上的亚临床疾病进展。GMa是所分析的所有肌肉中对疾病进展最敏感的。试验注册：中国临床试验注册中心（http://www.chictr.org.cn/index.aspx） ChiCTR1800018340, 2018年9月12日，前瞻性注册。
ObjectiveTo describe the disease progression of Duchenne muscular dystrophy (DMD) in the pelvic and thigh muscles over 1-year using multiple-parameter quantitative magnetic resonance imaging (qMRI), and to determine the most responsive muscle and predict subclinical disease progression in functionally stable patients.MethodsFifty-four DMD patients (mean age 8.9 ± 2.5, range 5–15 years) completed baseline and 1-year follow-up qMRI examinations/biomarkers [3-point Dixon/fat fraction (FF); T1 mapping/T1; T2 mapping/T2]. Meanwhile, clinical assessments [NorthStar ambulatory assessment (NSAA) score] and timed function tests were performed in DMD patients. Twenty-four healthy male controls (range 5–15 years) accomplished baseline qMRI examinations. Group differences were compared using the Wilcoxon test. The standardized response mean (SRM) was taken as the responsiveness to the disease progression index.ResultsFF, T1, and T2 in all DMD age subgroups changed significantly over 1-year (P< 0.05). Even in functionally stable patients (NSAA score increased, unchanged, or decreased by 1-point) over 1-year, significant increases in FF and T2 and decreases in T1 were observed in gluteus maximus (GMa), gluteus medius, vastus lateralis, and adductor magnus (P< 0.05). Overall, the SRM of FF, T1, and T2 was all the highest in GMa, which were 1.25, − 0.92, and 0.93, respectively.ConclusionsqMRI biomarkers are responsive to disease progression and can also detect subclinical disease progression in functionally stable DMD patients over 1-year. GMa is the most responsive to disease progression of all the muscles analyzed.Trial registrationChinese Clinical Trial Registry (http://www.chictr.org.cn/index.aspx) ChiCTR1800018340, 09/12/2018, prospectively registered.