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Abstract Details

The Pediatric Evaluation of Disability Inventory Computer Adaptive Test Captures Impairments in Alexander Disease
Child Neurology and Developmental Neurology
P4 - Poster Session 4 (5:30 PM-6:30 PM)
7-051
To determine the utility of the Pediatric Evaluation of Disability Inventory Computer Adaptive Test (PEDI-CAT) in quantifying functional abilities in Alexander Disease (AxD) patients.
The PEDI-CAT Content-Balanced “Comprehensive” assessment is a computerized tool designed for children with physical, cognitive, and behavioral impairments. We explored the feasibility of the PEDI-CAT as a potential observer-reported outcome measure for an imminent AxD clinical trial.

Participants in an AxD Outcomes Study completed a group of multidisciplinary evaluations, including the Comprehensive assessment of the PEDI-CAT, administered on an iPad and answered by a proxy (parent/guardian) for subjects <21 years. Four domains were captured: daily activities (activities of daily living), mobility (motor skills), social (interactions with others), and responsibility (independence). AxD scores were compared to normative scores (for age-matched controls, mean 50, standard deviation [SD] 10) and percentile ranges generated by the program. The Gross Motor Function Measure-88 (GMFM-88) captures gross motor skills across 5 dimensions (lying/rolling, sitting, crawling/kneeling, and standing/walking/running/jumping) and was administered during the same visit. GMFM-88 and mobility scaled scores (which are not age-related but provide data across a continuum for those unlikely to attain age-expected development) were compared using the Pearson’s correlation coefficient.

Proxies of 38 AxD patients completed the PEDI-CAT. Mean normative scores were lower than age-matched youth: activities 26.1 (SD 25.6), mobility 5.0 (SD 49.8), social 27.9 (23.9), and responsibility 30.5 (SD 20.6). The percentages of patients scoring <5th percentile by domain were: activities 65.79%, mobility 76.32%, social 57.89%, and responsibility 57.89%. Post-hoc analyses revealed a significant correlation between PEDI-CAT mobility scaled scores and GMFM-88 (p<0.001).

PEDI-CAT scores in AxD capture increased impairments in daily skills compared to age-matched youth. Mobility scores are highly correlated with the GMFM-88 in AxD, lending concurrent validity to the use of the PEDI-CAT in assessing gross motor function.
Authors/Disclosures
Hannah R. Cooper (Children'S Hospital of Philadelphia)
PRESENTER
Ms. Cooper has nothing to disclose.
Stacy Cusack (Children's Hospital of Philadlephia) No disclosure on file
No disclosure on file
No disclosure on file
Tracy Kornafel Tracy Kornafel has nothing to disclose.
No disclosure on file
No disclosure on file
Amy T. Waldman, MD (Children's Hospital of Philadelphia) Dr. Waldman has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for SwanBio. An immediate family member of Dr. Waldman has or had stock in Pfizer. The institution of Dr. Waldman has received research support from Ionis Pharmaceuticals. The institution of Dr. Waldman has received research support from Roche/Genentech. The institution of Dr. Waldman has received research support from Ionis Pharmaceuticals. The institution of Dr. Waldman has received research support from Calico. Dr. Waldman has received publishing royalties from a publication relating to health care. Dr. Waldman has received publishing royalties from a publication relating to health care.