Abstract Details

Title The NIH Toolbox for Cognitive Surveillance in Duchenne Muscular Dystrophy

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) S18 - Neuromuscular and Clinical Neurophysiology (EMG): Neuroimaging, Outcome Measures, and Biomarkers (2:24 PM-2:36 PM)

Poster/Presentation
Number 008

Objective The objective of this prospective, cross-sectional study was to evaluate the feasibility of using the NIH Toolbox, a scientifically vigorous neuro-behavioral measure, for cognitive surveillance in Duchenne Muscular Dystrophy.

Background The unmet cognitive needs in Duchenne Muscular Dystrophy (DMD) continues to be high. We used parent-reported questionnaires and found that although more than 80% of parents reported concerns of attention-difficulty and speech delay, systematic evaluation by a neuropsychologist was not performed. Further, a neuromuscular provider survey found that psychosocial health was the most challenging for providers. Thus, resource-constraints and access issues continue to prevent the complete evaluation of an individual with DMD.

Design/Methods This is a prospective, cross-section study. We enrolled 30 subjects all of them who had mutations in the dystrophin gene. They underwent evaluation with age-appropriate NIH Toolbox Cognition Battery. The testing was completed within 35-40 minutes. Parents also completed study-related questionnaires.

Results Overall, the children with DMD scored one standard-deviation below age-corrected norms on the NIH Toolbox Cognition Battery. In particular, they scored 1.5 SD below age-corrected norms for fluid cognition which evaluates executive function, working memory, attention and processing speed. They scored 1 SD below age-corrected norms for crystallized cognition score, which evaluates vocabulary and reading. On standardized ratings scales, they demonstrated greater difficulty in executive functioning.

Conclusions The NIH Toolbox has discriminative capacity to detect relative cognitive strengths and weakness in Duchenne Muscular Dystrophy. It can be easily incorporated in an ambulatory setting for cognitive surveillance.

Authors/Disclosures
Mathula Thangarajh, MD, PhD, FAAN PRESENTER	Dr. Thangarajh has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. Dr. Thangarajh has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for NS Pharma.
Aaron Kaat	No disclosure on file
Genila Bibat, MD (Kennedy Krieger Institute Johns Hopkins Medical Center)	Dr. Bibat has nothing to disclose.
	No disclosure on file
Kathryn R. Wagner, MD, PhD (The Kennedy Krieger Institute)	Dr. Wagner has received personal compensation for serving as an employee of F. Hoffmann-La Roche Ltd. Dr. Wagner has stock in F. Hoffmann-La Roche.

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