目的 探讨皮质类固醇治疗下Duchenne型肌营养不良(DMD)儿童运动功能指标的纵向变化,为DMD儿童临床干预提供科学依据。 方法 选取2017年10月—2024年7月在深圳市儿童医院康复科就诊的46例3～14岁DMD男童,分为学龄前期组和学龄期组,收集基本信息、运动功能测试(MFM)和计时功能测试(TFTs),TFTs包括10m步行/跑(10MWR)、仰卧站起(RFF)和上4级台阶(4SC)。采用Spearman秩相关分析和配对t检验分析MFM-32与TFTs间的相关性及12个月后的变化。 结果 MFM-D1区与TFTs呈强负相关(r=-0.665～-0.614,P＜0.01)。学龄前期组随访前后D1区得分、D3区得分、总分、10MWR和4SC测试结果比较,差异均有统计学意义(t=2.782、2.963、3.639、-2.138、-2.381,P＜0.05);学龄期组随访前后D1区得分、总分、10MWR和RFF的差异均有统计学意义(t=-4.433、-3.427、3.150、2.128,P＜0.05)。 结论 12个月后,学龄前DMD患儿运动功能总体呈上升趋势,学龄期患儿总体呈下降趋势。MFM-32和TFTs是反映DMD儿童运动功能变化的有效工具,建议临床上将两者纳入评估指标。
Abstract
Objective To investigate longitudinal changes in motor function outcomes in children with Duchenne muscular dystrophy (DMD) under corticosteroid treatment, in order to provide evidence for clinical management. Methods This longitudinal study included 46 male DMD patients (aged 3 - 14 years) treated at Rehabilitation Department of Shenzhen Children′s Hospital from October 2017 to July 2024.Participants were stratified into preschool and school-age groups.Assessments included basic information, Motor Function Measure-32 (MFM-32) and Timed Function Tests (TFTs).TFTs included the 10-meter walk/run test (10MWR), rise from floor (RFF), and four-step climb (4SC).Spearman′s correlation and paired t-tests were used to analyze the relationships between MFM-32 domains and TFTs at baseline and 12-month follow-up. Results MFM-D1 domain scores showed a strong negative correlation with TFTs (r =-0.665 to -0.614, P < 0.01).In the preschool group, statistically significant differences were observed in the MFM-D1 (t=2.782), D3 (t=2.963), total score (t=3.639), 10MWR (t=-2.138) and 4SC (t=-2.381) (all P<0.05).School-age group demonstrated significant changes in MFM-D1 (t=-4.433), total score (t=-3.427), 10MWR (t=3.15) and RFF (t=2.128) (all P<0.05). Conclusions After 12 months,motor function in the preschool children with DMD shows an overall upward trend, while the school-age children with DMD exhibited a decline. MFM-32 and TFTs are sensitive tools for monitoring motor function progression in DMD.Combined implementation is recommended for comprehensive clinical assessment.
关键词
杜氏肌营养不良症 /
运动功能评估 /
儿童
Key words
Duchenne muscular dystrophy /
motor function measure /
children
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基金
深圳市科技计划项(JCYJ20220530160004010)
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