Challenges in measuring the effects of pharmacological interventions on cognitive and adaptive functioning in individuals with Down syndrome: A systematic review.

Journal Article (Journal Article;Review;Systematic Review)

We systematically reviewed the measures used in pharmaceutical trials in children/adults with Down syndrome without dementia. Our purpose was to identify developmentally appropriate outcome measures capable of detecting changes in cognitive and adaptive functioning in this population. Eleven studies were included and used diverse outcome measures across the domains of language, memory, attention, behavior, and executive/adaptive functioning. Our results highlight the challenges in selecting measures capable of capturing improvements in pharmaceutical trials in individuals with DS. We offer suggestions to enhance future research, including: conducting studies with larger samples of participants with a range of developmental abilities; modifying existing/developing novel outcome measures; incorporating advances from related areas and DS observational studies; and considering alternative analytic techniques to characterize treatment effects.

Full Text

Duke Authors

Cited Authors

  • Keeling, LA; Spiridigliozzi, GA; Hart, SJ; Baker, JA; Jones, HN; Kishnani, PS

Published Date

  • November 2017

Published In

Volume / Issue

  • 173 / 11

Start / End Page

  • 3058 - 3066

PubMed ID

  • 28857390

Electronic International Standard Serial Number (EISSN)

  • 1552-4833

Digital Object Identifier (DOI)

  • 10.1002/ajmg.a.38416


  • eng

Conference Location

  • United States