Abstract
As adaptive skills (AS) are dynamic and may indicate the success of an intervention, they are a common domain measured in clinical trials. Typical interview tools for measuring AS are time-consuming, and questionnaire measures often lead to inconsistent information. The present study was designed to evaluate the feasibility, validity and test-retest performance of the Pediatric Evaluation of Disability Inventory Computer Adaptive Test (PEDI-CAT) in Fragile X syndrome (FXS). The PEDI-CAT is administered via tablet and uses the item response theory to efficiently determine the items administered. The PEDI-CAT was administered to 42 individuals with FXS (27 males; 15 females) aged 1.6–50.9 years (M = 14.9; SD = 11.2), followed by the Vineland-3 (VABS-3) interview for comparison. Administration was efficient (M = 21.7 min; SD = 9.5; range 8–45 min; mode = 19). Males and females did not significantly differ on the PEDI-CAT domains, except for daily activities (t(40) = −2.22, p = 0.037). Floor effects were significant for both measures, although the PEDI-CAT showed more floor effects in the mobility (35.7%) and social-cognitive (50%) domains. PEDI-CAT daily activities, mobility, social-cognitive and responsibility domains were all significantly correlated with most of the VABS-3 domains (all rho > 0.5; p < 0.01). Test-rest of the PEDI-CAT was comparable to the VABS-3. Results suggest that the PEDI-CAT is efficient, and minimal training is needed to administer it; however, it lacks specificity and shares a high rate of floor effects with the VABS-3.
Highlights
Fragile X syndrome (FXS) is the most common known inherited cause of intellectual disability (ID) and autism spectrum disorder with an estimated frequency of about 1:4000–5000 [1] affecting all racial and ethnic groups worldwide
The purpose of this study was to explore the results of a computer-adaptive measure of adaptive behaviors (PEDI-computerized adaptive testing (CAT)) [14] in individuals with FXS compared to the Vineland-3
Missing data across all measures was primarily due to limitations on the length of assessments tolerated by participants with FXS, as well as the availability of measures at the beginning of the study
Summary
Fragile X syndrome (FXS) is the most common known inherited cause of intellectual disability (ID) and autism spectrum disorder with an estimated frequency of about 1:4000–5000 [1] affecting all racial and ethnic groups worldwide. Extensive study of the neurobiology and synaptic mechanisms of the disease in cellular and animal models has identified many neuronal targets for treatment and made possible enormous progress in preclinical and clinical translational works in FXS over the past decade [2,3]. Clinical trials of several new treatments targeted to the underlying disease have been initiated, and some new drugs targeting glutamate or GABA pathways have shown promising results in both open-label and early phase studies [4,5]. Multiple complex issues have led to challenges replicating results in larger placebo-controlled phase 2b and 3 trials [6,7], including the lack of outcome. Brain Sci. 2020, 10, 351 measures validated in FXS [8]. Adaptive functioning measures may better reflect the overall functioning of individuals with FXS compared to cognitive measures, given challenges with the administration and interpretation of standardized cognitive tests in much of the FXS population, as well as the effects of anxiety and avoidance on test performances [10]
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