Outcome measures for clinical trials in down syndrome

Anna J. Esbensen; Stephen R. Hooper; Deborah Fidler; Sigan L. Hartley; Jamie Edgin; Xavier Liogier D'Ardhuy; George Capone; Frances A. Conners; Carolyn B. Mervis; Leonard Abbeduto; Michael Rafii; Sharon J. Krinsky-Mchale; Tiina Urv; Elisabeth Dykens; Anna Esbenson; Sigan Hartlay; Seth Keller; Sara Weir

doi:10.1352/1944-7558-122.3.247

Outcome measures for clinical trials in down syndrome

Anna J. Esbensen, Stephen R. Hooper, Deborah Fidler, Sigan L. Hartley, Jamie Edgin, Xavier Liogier D'Ardhuy, George Capone, Frances A. Conners, Carolyn B. Mervis, Leonard Abbeduto, Michael Rafii, Sharon J. Krinsky-Mchale, Tiina Urv, Elisabeth Dykens, Anna Esbenson, Sigan Hartlay, Seth Keller, Sara Weir

Research output: Contribution to journal › Article › peer-review

110 Scopus citations

Abstract

Increasingly individuals with intellectual and developmental disabilities, including Down syndrome, are being targeted for clinical trials. However, a challenge exists in effectively evaluating the outcomes of these new pharmacological interventions. Few empirically evaluated, psychometrically sound outcome measures appropriate for use in clinical trials with individuals with Down syndrome have been identified. To address this challenge, the National Institutes of Health (NIH) assembled leading clinicians and scientists to review existing measures and identify those that currently are appropriate for trials; those that may be appropriate after expansion of age range addition of easier items, and/or downward extension of psychometric norms; and areas where new measures need to be developed. This article focuses on measures in the areas of cognition and behavior.

Original language	English (US)
Pages (from-to)	247-281
Number of pages	35
Journal	American Journal on Intellectual and Developmental Disabilities
Volume	122
Issue number	3
DOIs	https://doi.org/10.1352/1944-7558-122.3.247
State	Published - May 2017

Keywords

Assessment
Behavior
Clinical trials
Cognition
Down syndrome
Intellectual disability

ASJC Scopus subject areas

General Medicine

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10.1352/1944-7558-122.3.247

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Esbensen, A. J., Hooper, S. R., Fidler, D., Hartley, S. L., Edgin, J., D'Ardhuy, X. L., Capone, G., Conners, F. A., Mervis, C. B., Abbeduto, L., Rafii, M., Krinsky-Mchale, S. J., Urv, T., Dykens, E., Esbenson, A., Hartlay, S., Keller, S., & Weir, S. (2017). Outcome measures for clinical trials in down syndrome. American Journal on Intellectual and Developmental Disabilities, 122(3), 247-281. https://doi.org/10.1352/1944-7558-122.3.247

Esbensen, AJ, Hooper, SR, Fidler, D, Hartley, SL, Edgin, J, D'Ardhuy, XL, Capone, G, Conners, FA, Mervis, CB, Abbeduto, L, Rafii, M, Krinsky-Mchale, SJ, Urv, T, Dykens, E, Esbenson, A, Hartlay, S, Keller, S & Weir, S 2017, 'Outcome measures for clinical trials in down syndrome', American Journal on Intellectual and Developmental Disabilities, vol. 122, no. 3, pp. 247-281. https://doi.org/10.1352/1944-7558-122.3.247

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doi = "10.1352/1944-7558-122.3.247",

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AU - D'Ardhuy, Xavier Liogier

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Outcome measures for clinical trials in down syndrome

Abstract

Keywords

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