AIM To investigate functional motor performance in a large cohort of young steroid-naïve males with Duchenne muscular dystrophy (DMD) and typically developing males, and to develop specific reference curves for both groups. Also, to describe associations between anthropometric values and functional motor outcomes. METHOD Cross-sectional data of 196 steroid-naïve males with DMD aged 4 to 8 years and 497 typically developing males aged 2 years 6 months to 8 years were included. Both groups were evaluated with the time to rise from the floor test, 10-metre walk/run test, 6-minute walk test, and North Star Ambulatory Assessment. Reference curves with centiles 5%, 10%, 25%, 50%, 75%, 90%, and 95% were estimated using quantile regression. RESULTS Males with DMD scored significantly worse on all functional motor outcomes than age-matched typically developing males (p < 0.001): 89% to 95% of the males with DMD scored below the 5th centile of the typically developing males. No or weak correlations exist between anthropometric values and functional motor outcomes. INTERPRETATION The estimated reference curves can support consultation with families of young males with DMD and can support the evaluation of treatment for reaching motor skills and functional motor outcomes compared with typically developing males.

中文翻译：

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患有杜氏肌营养不良症的年轻未接受类固醇治疗的男性运动功能结果的参考曲线。

目的 研究一大群患有杜氏肌营养不良症 (DMD) 且未接受过类固醇治疗的年轻男性和典型发育中男性的功能性运动表现，并为两组制定具体的参考曲线。此外，还描述了人体测量值和功能性运动结果之间的关联。方法 纳入了 196 名年龄为 4 至 8 岁、未接受类固醇治疗的 DMD 男性和 497 名年龄为 2 岁 6 个月至 8 岁的典型发育男性的横断面数据。两组均接受了从地板起身时间测试、10米步行/跑步测试、6分钟步行测试和北极星动态评估等评估。使用分位数回归估计百分位数为 5%、10%、25%、50%、75%、90% 和 95% 的参考曲线。结果 患有 DMD 的男性在所有功能性运动结果方面的得分均明显低于年龄匹配的典型发育男性 (p < 0.001)：89% 至 95% 的 DMD 男性得分低于典型发育男性的第 5 个百分位。人体测量值和功能运动结果之间不存在或存在微弱的相关性。解释 估计的参考曲线可以支持与患有 DMD 的年轻男性的家庭进行协商，并且可以支持与典型发育男性相比，对达到运动技能和功能性运动结果的治疗进行评估。