Duchenne 型肌营养不良症的纵向时间功能测试：ImagingDMD 队列的自然史。
Longitudinal timed function tests in Duchenne muscular dystrophy: ImagingDMD cohort natural history.
机构信息
Department of Physical Therapy, University of Florida, Box 100154, UFHSC, Gainesville, Florida, 32610, USA.
The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
出版信息
Muscle Nerve. 2018 Nov;58(5):631-638. doi: 10.1002/mus.26161. Epub 2018 Jul 24.
INTRODUCTION
Tests of ambulatory function are common clinical trial endpoints in Duchenne muscular dystrophy (DMD). Using these tests, the ImagingDMD study has generated a large data set that can describe the contemporary natural history of DMD in 5-12.9-year-olds.
METHODS
Ninety-two corticosteroid-treated boys with DMD and 45 controls participated in this longitudinal study. Participants performed the 6-minute walk test (6MWT) and timed function tests (TFT: 10-m walk/run, climbing 4 stairs, supine to stand).
RESULTS
Boys with DMD had impaired functional performance even at 5-6.9 years old. Boys older than 7 had significant declines in function over 1 year for 10-m walk/run and 6MWT. Eighty percent of participants could perform all functional tests at 9 years old. TFTs appear to be slightly more responsive and predictive of disease progression than the 6MWT in 7-12.9 year olds.
DISCUSSION
This study provides insight into the contemporary natural history of key functional endpoints in DMD. Muscle Nerve 58: 631-638, 2018.
简介
在杜氏肌营养不良症（DMD）中，运动功能测试是常见的临床试验终点。通过这些测试，ImagingDMD 研究产生了一个大型数据集，可描述 5-12.9 岁 DMD 患儿的当代自然病史。
方法
92 名接受皮质类固醇治疗的 DMD 男孩和 45 名对照者参加了这项纵向研究。参与者进行了 6 分钟步行测试（6MWT）和计时功能测试（TFT：10 米步行/跑，爬 4 级楼梯，仰卧位站立）。
结果
即使在 5-6.9 岁时，患有 DMD 的男孩的功能表现也受损。7 岁以上的男孩在 1 年内 10 米步行/跑和 6MWT 的功能显著下降。80%的参与者在 9 岁时可以进行所有功能测试。在 7-12.9 岁的儿童中，TFT 似乎比 6MWT 更敏感，更能预测疾病进展。
讨论
这项研究深入了解了 DMD 中关键功能终点的当代自然病史。肌肉神经 58:631-638，2018。
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