Characterization of potential outcome measures for future clinical trials in fragile X syndrome.
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.
Berry-Kravis
Elizabeth
E
Department of Pediatrics, Rush University Medical Center, 1725 W. Harrison St., Suite 718, Chicago, IL, USA. elizabeth_m_berry-kravis@rush.edu
Sumis
Allison
A
Kim
Ok-Kyung
OK
Lara
Rebecca
R
Wuu
Joanne
J
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