Lowes Linda Pax, Le Reun Corinne M, Alfano Lindsay N, Reash Natalie F, Iammarino Megan A, Patel Shivangi, Audhya Ivana F
Center for Gene Therapy, Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Biostatistics, Sainte-Anne, Guadeloupe, France.
Dev Med Child Neurol. 2025 Jul;67(7):918-929. doi: 10.1111/dmcn.16198. Epub 2024 Dec 19.
To evaluate the psychometric properties and measurement quality of the Patient-Reported Outcomes Measurement Information System Parent Proxy (PROMIS PP) Mobility item bank (v1.0, 23 items) for children with Duchenne muscular dystrophy (DMD), through Rasch statistical analysis.
De-identified PROMIS PP Mobility items were completed by the caregivers of male patients with DMD, aged 4 to 12 years, as part of standard clinical care at the Nationwide Children's Hospital clinic; data were mined retrospectively from electronic health records. Rasch analysis was used to assess the internal functioning of the measure and items.
Overall, 151 observations were available for the Rasch analysis, equally split between patients aged 4 to 7 years and 8 to 12 years. After removing clinically irrelevant items and regrouping response options for specific items, the resulting 19-item measure demonstrated overall good fit to Rasch model expectations and the ability to discriminate between respondents with different mobility levels (Person Separation Index = 0.95, excellent reliability).
The customized PROMIS PP Mobility measure demonstrated good fit and may be a reliable option for mobility assessment in children with DMD. Rasch analysis can be used by other researchers to improve the sensitivity of patient-reported outcomes in their field of interest.
通过拉施统计分析，评估患者报告结局测量信息系统家长代理版（PROMIS PP）运动项目库（第1.0版，23个项目）用于杜氏肌营养不良症（DMD）患儿的心理测量特性和测量质量。
4至12岁男性DMD患者的照料者完成去识别化的PROMIS PP运动项目，作为全国儿童医院诊所标准临床护理的一部分；数据从电子健康记录中进行回顾性挖掘。采用拉施分析来评估该测量方法和项目的内部功能。
总体而言，有151份观察数据可用于拉施分析，4至7岁和8至12岁的患者各占一半。在去除临床无关项目并重新分组特定项目的回答选项后，最终得到的19个项目的测量方法总体上很好地符合拉施模型预期，并且能够区分不同运动水平的应答者（个人分离指数=0.95，可靠性极佳）。
定制的PROMIS PP运动测量方法显示出良好的拟合度，可能是评估DMD患儿运动能力的可靠选择。其他研究人员可以使用拉施分析来提高其感兴趣领域中患者报告结局的敏感性。
