Service de Médecine Physique et de Réadaptation, CHRU de Brest, Hôpital Morvan, Brest, France.
Pôle Information Médicale, Évaluation, Recherche, Hospices Civils de Lyon, Lyon, France.
Dev Med Child Neurol. 2018 Feb;60(2):185-191. doi: 10.1111/dmcn.13590. Epub 2017 Oct 9.
To monitor the evolution of the motor function of ambulatory patients with Duchenne muscular dystrophy (DMD) treated by corticosteroids for 2 years in comparison with untreated patients.
This observational, multicentre cohort study explores the evolution of the motor function measure (MFM) over a 24-month period for 29 ambulant corticosteroids-treated and 45 ambulant untreated patients with DMD.
Significant differences were found between mean MFM scores in corticosteroids-treated and untreated groups for domain 1 of the MFM (standing position and transfers; D1), domain 2 of the MFM (axial and proximal motor function; D2), and domain 3 of the MFM (distal motor function; D3). Subscores were between 0 months and 6 months, and 0 months and 24 months. For the D1 subscore specifically, there was a significant increase in the corticosteroids-treated group (mean±standard deviation [SD] slope of change=12.6±15.5%/y), while a decrease was observed in the untreated group (-17.8±17.7%/y) between 0 months and 6 months (p<0.001). Sensitivity to change as assessed by standardized response means was high between 12 months and 24 months for D1 of both corticosteroids-treated and untreated groups (1.0 and 1.2 respectively), and low for D2 and D3 of both treated and untreated groups.
Patients with DMD treated by corticosteroids present a different course of the disease as assessed by MFM, confirming the sensitivity to change of the MFM in this population.
Corticosteroids have a quantitative impact on muscle strength 6 to 24 months after starting treatment. Motor function measure is a valid outcome measure in Duchenne muscular dystrophy patients under corticosteroid treatment.
比较 2 年糖皮质激素治疗与未治疗的杜氏肌营养不良症（DMD）门诊患者的运动功能演变。
这项观察性、多中心队列研究探讨了 29 名接受糖皮质激素治疗的门诊 DMD 患者和 45 名未治疗的门诊 DMD 患者在 24 个月期间运动功能测量（MFM）的演变。
糖皮质激素治疗组和未治疗组在 MFM 的第 1 域（站立位和转移；D1）、第 2 域（轴和近端运动功能；D2）和第 3 域（远端运动功能；D3）的平均 MFM 评分存在显著差异。亚评分在 0 个月至 6 个月和 0 个月至 24 个月之间。特别是 D1 亚评分，糖皮质激素治疗组有显著增加（平均±标准偏差[SD]变化斜率=12.6±15.5%/y），而未治疗组则在 0 个月至 6 个月之间下降（-17.8±17.7%/y）（p<0.001）。12 个月至 24 个月时，糖皮质激素治疗组和未治疗组的 D1 变化的标准化反应均值的敏感性均较高（分别为 1.0 和 1.2），而治疗组和未治疗组的 D2 和 D3 的敏感性均较低。
用 MFM 评估，接受糖皮质激素治疗的 DMD 患者表现出不同的疾病过程，证实了 MFM 在该人群中的变化敏感性。
糖皮质激素治疗开始后 6 至 24 个月对肌肉力量有定量影响。MFM 是评估接受皮质激素治疗的杜氏肌营养不良症患者的有效结局指标。
