Glanzman Allan M, O'Hagen Jessica M, McDermott Michael P, Martens William B, Flickinger Jean, Riley Susan, Quigley Janet, Montes Jacqueline, Dunaway Sally, Deng Liyong, Chung Wendy K, Tawil Rabi, Darras Basil T, De Vivo Darryl C, Kaufmann Petra, Finkel Richard S
Department of Physical Therapy, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, USA.
J Child Neurol. 2011 Dec;26(12):1499-507. doi: 10.1177/0883073811420294. Epub 2011 Sep 21.
The relationships between the Expanded Hammersmith Functional Motor Scale (HFMSE) and genotype and motor and respiratory outcomes were examined in patients with spinal muscular atrophy types II and III (n = 70). The correlation between the HFMSE and Gross Motor Function Measure was r = 0.98. Correlations between HFMSE and forced vital capacity (percentage of predicted normal) (n = 56) and a functional rating (n = 57) were r = 0.87 and r = 0.92, respectively. Correlations with strength were as follows: knee extension, r = 0.74 (n = 60); elbow flexion, r = 0.77 (n = 61); and knee flexion, r = 0.74 (n = 58). The HFMSE differentiated patients by SMN2 copy number (P = .0007); bi-level positive airway pressure use, <8 versus ≥8 hours/day (P < .0001); ambulatory status (P < .0001); and spinal muscular atrophy type (P < .0001). The HFMSE demonstrates significant associations with established measures of function, strength, and genotype, and discriminates patients based on function, diagnostic category, and bi-level positive airway pressure need. Time of administration averaged 12 minutes. The HFMSE is a valid, time-efficient outcome measure for clinical trials in spinal muscular atrophy types II and III.
在70例II型和III型脊髓性肌萎缩症患者中，研究了扩展哈默史密斯功能运动量表（HFMSE）与基因型、运动及呼吸结局之间的关系。HFMSE与粗大运动功能测量的相关性为r = 0.98。HFMSE与用力肺活量（预测正常值的百分比）（n = 56）和功能评级（n = 57）的相关性分别为r = 0.87和r = 0.92。与肌力的相关性如下：膝关节伸展，r = 0.74（n = 60）；肘关节屈曲，r = 0.77（n = 61）；膝关节屈曲，r = 0.74（n = 58）。HFMSE可根据SMN2拷贝数（P = 0.0007）、双水平气道正压通气使用情况（每天<8小时与≥8小时）（P < 0.0001）、行走状态（P < 0.0001）以及脊髓性肌萎缩症类型（P < 0.0001）对患者进行区分。HFMSE与已确立的功能、肌力和基因型测量方法存在显著关联，并可根据功能、诊断类别和双水平气道正压通气需求对患者进行鉴别。给药时间平均为12分钟。HFMSE是II型和III型脊髓性肌萎缩症临床试验中一种有效的、省时的结局指标。
