Haley Stephen M, Fragala Maria A, Aseltine Robert, Ni Pengsheng, Skrinar Alison M
Center for Rehabilitation Effectiveness, Sargent College of Health and Rehabilitation Sciences, Boston University, 635 Commonwealth Avenue, Boston, MA 02215-1605, USA.
Pediatr Rehabil. 2003 Apr-Jun;6(2):77-84. doi: 10.1080/1363849031000139298.
The purpose of this study was to modify the Paediatric Evaluation of Disability Inventory (PEDI) to create a Pompe disease-specific disability instrument for use in clinical trials and natural history studies.
PEDI item content was revised to include self-care and mobility items appropriate for children and youth with Pompe disease. Data were collected on 30 individuals with Pompe disease (mean age 7.7+/-5.6 years; range 0.4-22.1 years) by parent proxy through telephone interviews. New items were merged with original PEDI items using Rasch rating scale methods.
The Pompe-PEDI extended the content range and scoring precision of the original PEDI. Construct validity was demonstrated and test-re-test reliability was excellent.
The Pompe-PEDI is a reliable and valid instrument to assess and monitor the functional changes of children and youth with Pompe disease.
本研究的目的是对儿童残疾评估量表（PEDI）进行修改，以创建一种用于庞贝病临床试验和自然史研究的特定于庞贝病的残疾评估工具。
修订PEDI项目内容，纳入适合庞贝病儿童和青少年的自我护理和活动能力项目。通过电话访谈，由家长代理对30名庞贝病患者（平均年龄7.7±5.6岁；范围0.4 - 22.1岁）收集数据。使用Rasch评分量表方法将新项目与原始PEDI项目合并。
庞贝病-PEDI扩展了原始PEDI的内容范围和评分精度。证明了结构效度，重测信度良好。
庞贝病-PEDI是一种可靠且有效的工具，可用于评估和监测庞贝病儿童和青少年的功能变化。
