Introduction

Clinical trials targeting younger cohorts of boys with Duchenne muscular dystrophy are necessary as earlier intervention may maximize treatment effect. Boys with Duchenne muscular dystrophy often have gross motor delays very early in life, and although they gain skills, they are on a lower trajectory than typical peers. Quantifying the natural rate of motor maturation in Duchenne muscular dystrophy from an early age permits identification of deviations from the expected trajectory related to treatment effects.

Methods

The purpose of our study was to define the natural history in boys aged from ≥3 to <8 years using the North Star Ambulatory Assessment (NSAA), 100-meter timed test (100m), 10-meter walk/run (10m), time to rise (Rise), and 4-stair climb (4SC). Assessments were completed as standard of care during regularly scheduled clinic visits.

Results

One hundred sixty-two boys with DMD aged 3.1 to 7.9 years on glucocorticoids were evaluated using one or more of the following tests as appropriate for age: NSAA (N = 158; 3.1-7.9 years), 100m (N = 131; 3.4-7.9 years), 10m (N = 162; 3.1-7.9 years), Rise (N = 160; 3.1-7.9 years), and 4SC (N = 153; 3.1-7.9 years). Longitudinal data are presented by age in a subcohort (N = 64).

Conclusions

Our study documents the baseline function of boys with DMD who are being treated with corticosteroids. These data will be useful to compare ongoing and future therapeutic intervention(s) for DMD.

中文翻译：

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使用 NSAA、100m 和定时功能测试的类固醇治疗杜氏肌营养不良症小男孩的自然史

介绍

有必要针对年轻的杜氏肌营养不良症男孩进行临床试验，因为早期干预可以最大限度地提高治疗效果。患有杜氏肌营养不良症的男孩通常在生命早期就有明显的运动发育迟缓，尽管他们获得了技能，但与典型的同龄人相比，他们的发展轨迹较低。从小就量化杜氏肌营养不良症的自然运动成熟率，可以识别与治疗效果相关的预期轨迹的偏差。

方法

我们研究的目的是使用北极星动态评估 (NSAA)、100 米计时测试 (100m)、10 米步行/跑步 (10m)、上升时间 (Rise) 和 4 级爬升 (4SC)。评估是在定期安排的诊所就诊期间作为护理标准完成的。

结果

162 名年龄在 3.1 至 7.9 岁之间接受糖皮质激素治疗的 DMD 男孩根据年龄使用以下一项或多项测试进行评估：NSAA（N = 158；3.1-7.9 岁），100m（N = 131；3.4- 7.9 年）、10m（N = 162；3.1-7.9 年）、Rise（N = 160；3.1-7.9 年）和 4SC（N = 153；3.1-7.9 年）。纵向数据在子队列中按年龄呈现（N = 64）。

结论

我们的研究记录了正在接受皮质类固醇治疗的 DMD 男孩的基线功能。这些数据将有助于比较正在进行和未来的 DMD 治疗干预。