Haley Stephen M, Fragala-Pinkham Maria A, Ni Peng Sheng, Skrinar Alison M, Kaye Edward M
Health and Disability Research Institute, Boston University, Boston, MA 02215, USA.
Pediatr Neurol. 2004 Nov;31(5):333-41. doi: 10.1016/j.pediatrneurol.2004.05.002.
We developed normative profiles of physical functioning (mobility and self-care) in infancy up through 14 years of age with an expanded version of the Pediatric Evaluation of Disability Inventory. Mobility and self-care reference curves were based on the original Pediatric Evaluation of Disability Inventory standardization data (n = 412) and data from an additional cross-sectional, convenience sample (n = 373) via web-based survey, telephone or in-person interviews of parents. This new sample, which included children up through 14 years-of-age, was stratified for race, age, and sex, but was primarily limited geographically to the Northeast region of the United States. Goodness of fit of male, female, and combined sex (male and female) reference curves was examined. The mobility and self-care reference curves produced efficient and well-fitting estimates of conventional percentiles (3rd, 10th, 25th, 50th, 75th, 97th). Differences between males' and females' reference curves were negligible. This study highlights the use of these reference curves for determining the functional impact of Pompe disease, a lysosomal storage disorder that affects skeletal and cardiac muscle, restricting normal expression of mobility and self-care activities. This physical functioning instrument could also be used to evaluate the impact of muscle weakness in other neuromuscular disorders.
我们使用《儿童残疾评定量表》的扩展版本，制定了从婴儿期到14岁的身体功能（活动能力和自我护理）规范概况。活动能力和自我护理参考曲线基于《儿童残疾评定量表》的原始标准化数据（n = 412），以及通过对家长进行网络调查、电话或面对面访谈从一个额外的横断面便利样本（n = 373）中获取的数据。这个新样本包括14岁以下的儿童，按种族、年龄和性别进行了分层，但主要限于美国东北地区。对男性、女性和综合性别（男性和女性）参考曲线的拟合优度进行了检验。活动能力和自我护理参考曲线对传统百分位数（第3、10、25、50、75、97百分位数）产生了有效且拟合良好的估计。男性和女性参考曲线之间的差异可以忽略不计。本研究强调了使用这些参考曲线来确定庞贝病的功能影响，庞贝病是一种影响骨骼和心肌的溶酶体贮积症，会限制活动能力和自我护理活动的正常表现。这种身体功能评估工具也可用于评估其他神经肌肉疾病中肌肉无力的影响。
