Considerations for measuring individual outcomes across contexts in Down syndrome: Implications for research and clinical trials.

4区 医学 Q3 Medicine
Anna J Esbensen, Emily K Schworer, Deborah J Fidler, Angela John Thurman
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引用次数: 0

Abstract

Individuals with Down syndrome (DS) are increasingly involved in clinical trials that target developmental outcomes, like cognition and behavior. The increased focus on treatment in DS has led to ongoing discussions regarding the selection of outcome measures using syndrome-informed criteria. This discourse is warranted as clinical trials can fail if the outcome measures selected are inappropriate for individuals with DS or do not take into account the behavioral phenotype commonly associated with DS. This review focuses on the challenges present in the measurement of outcomes in DS, with a specific focus on considerations made in evaluating cognitive, language, and behavioral/psychopathology outcomes. This review also provides a summary of recommendations for assessment of outcomes in these domains as well as recommendations for future research. The impact of physical health and assessment psychometrics on the measurement of outcomes is also reviewed.

测量唐氏综合症患者不同情况下的个体结果的考虑因素:对研究和临床试验的影响。
唐氏综合征(DS)患者越来越多地参与到以认知和行为等发育结果为目标的临床试验中。对唐氏综合症治疗的日益关注引发了有关使用综合症相关标准选择结果测量方法的持续讨论。这种讨论是有必要的,因为如果选择的结果指标不适合 DS 患者,或者没有考虑到 DS 常见的行为表型,临床试验就可能失败。本综述将重点放在 DS 患者疗效测量中存在的挑战上,尤其关注评估认知、语言和行为/心理病理学疗效时的注意事项。本综述还对这些领域的结果评估建议以及未来研究建议进行了总结。此外,还综述了身体健康和评估心理测量学对结果测量的影响。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
CiteScore
1.10
自引率
0.00%
发文量
12
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