Shenzhen Children's Hospital, Shenzhen, China.
Dev Neurorehabil. 2022 Aug;25(6):370-377. doi: 10.1080/17518423.2021.2020352. Epub 2021 Dec 29.
This study aimed to examine the responsiveness and minimal clinically important difference of the Motor Function Measure 32-Chinese version (MFM 32-CN) in children and adolescents with Duchenne muscular dystrophy (DMD).
This observational, prospective cohort study assessed changes in motor function over 12 months using the MFM 32-CN in 108 pediatric patients with DMD.
Moderate to high internal responsiveness was found for MFM total, D1, and D2 scores (SRM = 0.46-0.83). Sufficient external responsiveness was observed in all MFM scores, as determined by a good correlation with the Patient Global Impression of Change (PGIC) ratings (r = 0.54-0.72, < .01). The estimated MCID values derived from different anchor-based methods ranged from 4.7 to 6.0, with a median of 5.0 points (%).
The MFM 32-CN demonstrates sufficient internal and external responsiveness as a measure of motor function in children and adolescents with DMD. The present study established the MCID of MFM-32 in pediatric patients with DMD.
本研究旨在探讨杜氏肌营养不良症(DMD)患儿和青少年使用中文版 32 项肌营养不良量表(MFM 32-CN)的反应度和最小临床重要差值。
本观察性前瞻性队列研究在 108 例 DMD 儿科患者中使用 MFM 32-CN 评估了 12 个月内运动功能的变化。
MFM 总分、D1 和 D2 评分的内部反应度为中高度(SRM=0.46-0.83)。所有 MFM 评分的外部反应度均足够,与患者总体变化印象(PGIC)评分的相关性良好(r=0.54-0.72,<0.01)。不同基于锚定的方法得出的估计 MCID 值范围为 4.7 至 6.0,中位数为 5.0 分(%)。
MFM 32-CN 作为 DMD 患儿和青少年运动功能的测量工具,具有足够的内部和外部反应度。本研究确定了 DMD 儿科患者 MFM-32 的 MCID。