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Updated: Jun 4, 2025
Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Psychometric evaluation of the PROMIS parent proxy mobility item bank for use in Duchenne muscular dystrophy
Linda Pax Lowes1, Corinne M Le Reun2, Lindsay N Alfano1
1Center for Gene Therapy, Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Insights
A customized Patient-Reported Outcomes Measurement Information System Parent Proxy (PROMIS PP) Mobility measure shows good psychometric properties for assessing mobility in boys with Duchenne muscular dystrophy (DMD). This reliable tool aids in evaluating functional status in pediatric DMD patients.
Area of Science:
- Pediatric Rheumatology
- Clinical Measurement
- Rehabilitation Science
Background:
- Duchenne muscular dystrophy (DMD) significantly impacts mobility in children.
- Accurate assessment of mobility is crucial for managing DMD.
- Existing measures may require refinement for this specific population.
Purpose of the Study:
- To evaluate the psychometric properties of the Patient-Reported Outcomes Measurement Information System Parent Proxy (PROMIS PP) Mobility item bank (v1.0) for children with DMD.
- To assess the measurement quality of a customized PROMIS PP Mobility measure using Rasch analysis.
- To determine the reliability and validity of the PROMIS PP Mobility measure in a pediatric DMD cohort.
Main Methods:
- Retrospective analysis of de-identified PROMIS PP Mobility data from caregivers of male patients with DMD (aged 4-12 years).
- Data collected from electronic health records at Nationwide Children's Hospital.
- Rasch statistical analysis employed to evaluate item and measure functioning.
Main Results:
- 151 observations were analyzed, with equal distribution across age groups (4-7 and 8-12 years).
- A 19-item customized measure demonstrated good fit to Rasch model expectations after item removal and response option regrouping.
- The measure exhibited excellent reliability (Person Separation Index = 0.95) and discriminated well between different mobility levels.
Conclusions:
- The customized PROMIS PP Mobility measure is a reliable and valid tool for assessing mobility in children with DMD.
- Rasch analysis proved effective in refining the measure for this population.
- This approach can enhance the sensitivity of patient-reported outcomes in research and clinical practice for pediatric neuromuscular conditions.
Aim:
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.
Method:
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.
Results:
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).
Interpretation:
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.
