Performance outcomes of the <scp>PEDI</scp> ‐ <scp>CAT</scp> for assessing functional ability in the population with leukodystrophy

AIM: To describe the use of the Pediatric Evaluation of Disability Index-Computer Adapted Test (PEDI-CAT), a parent-reported outcome, and determine functional performance in a cohort with leukodystrophy. METHOD: This was a cross-sectional observational study. Ninety-nine caregivers (of 46 females and 53 males, mean age = 7 years 11 months, age range = 2 months-34 years) completed the PEDI-CAT (Aicardi-Goutières syndrome n = 52, TUBB4A-related leukodystrophies n = 22, Pelizaeus-Merzbacher disease n = 7, POLR3-related leukodystrophy n = 18). Concurrent validity was compared between the PEDI-CAT mobility domain and the 88-item Gross Motor Function Measure (GMFM-88), and the PEDI-CAT daily activities domain with the visual motor integration (VMI) and grasping subtests of the Peabody Developmental Motor Scales, Second Edition (PDMS-2). RESULTS: Strong correlations were observed between the PEDI-CAT mobility domain and the GMFM-88 (Spearman's rank correlation coefficient: ρ = 0.93, 95% confidence interval [CI] = 0.90-0.95, p < 0.001), and the PEDI-CAT daily activities domain and the PDMS-2 VMI and grasping section (both Spearman's rank correlation coefficient: ρ = 0.94, 95% CI = 0.91-0.96, p < 0.001). INTERPRETATION: The PEDI-CAT is a simple, feasible tool for caregiver assessment of function in patients with leukodystrophies; a strong correlation with established assessment tools was achieved. These findings support the use of computerized adaptive tools as a faster way to stratify functional abilities in the population with leukodystrophy.