title: Characterization of potential outcome measures for future clinical trials in fragile X syndrome.
abstract: Clinical trials targeting recently elucidated synaptic defects in fragile X syndrome (FXS) will require outcome measures capable of assessing short-term changes in cognitive functioning. Potentially useful measures for FXS were evaluated here in a test-retest setting in males and females with FXS (N = 46). Good reproducibility, determined by an interclass correlation (ICC) or weighted kappa (kappa) of 0.7-0.9 was seen for RBANS List and Story Memory, NEPSY Tower, Woodcock-Johnson Spatial Relations and the commissions score from the Carolina Fragile X Project Continuous Performance Test (CPT). This study demonstrates the feasibility of generating test profiles containing reliability data, ability levels required for test performance, and refusal rates to assist with choice of outcome measures in FXS and other cohorts with cognitive disability.
authors: Department of Pediatrics, Rush University Medical Center, 1725 W. Harrison St., Suite 718, Chicago, IL, USA. elizabeth_m_berry-kravis@rush.edu
authors: Elizabeth
authors: E
authors: Berry-Kravis
authors: Allison
authors: A
authors: Sumis
authors: Ok-Kyung
authors: OK
authors: Kim
authors: Rebecca
authors: R
authors: Lara
authors: Joanne
authors: J
authors: Wuu
mesh_terms: D000293:Adolescent; D000328:Adult; D001921:Brain / Q000503:physiopathology; D002648:Child; D002675:Child, Preschool; D003072:Cognition Disorders / Q000175:diagnosis / Q000453:epidemiology; D005260:Female; D005600:Fragile X Syndrome / Q000453:epidemiology / Q000503:physiopathology / Q000628:therapy*; D006801:Humans; D008279:Magnetic Resonance Imaging; D008297:Male; D008875:Middle Aged; D009483:Neuropsychological Tests; D013028:Space Perception; D013569:Synapses / Q000502:physiology; D016896:Treatment Outcome