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卡巴拉汀治疗唐氏综合征青少年的安全性和有效性:一项为期20周的初步开放标签研究。

Safety and efficacy of rivastigmine in adolescents with Down syndrome: a preliminary 20-week, open-label study.

作者信息

Heller James H, Spiridigliozzi Gail A, Crissman Blythe G, Sullivan Jennifer A, Eells Rebecca L, Li Jennifer S, Doraiswamy P Murali, Krishnan K Ranga, Kishnani Priya S

机构信息

Department of Surgery, Duke University Medical Center, Durham, North Carolina 27710, USA.

出版信息

J Child Adolesc Psychopharmacol. 2006 Dec;16(6):755-65. doi: 10.1089/cap.2006.16.755.

DOI:10.1089/cap.2006.16.755
PMID:17201619
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC3129997/
Abstract

Individuals with Down syndrome (DS) exhibit a cholinergic deficiency similar to that found in Alzheimer's disease. Cholinesterase inhibitors, used to treat Alzheimer's disease, may improve cognitive function in individuals with DS. This is the first investigation of the safety and efficacy of rivastigmine (an acetyl and butyryl cholinesterase inhibitor) on specific cognitive domains in pediatric DS. Eleven subjects with DS (ages 10-17 years) were treated with a liquid formulation of rivastigmine. Four subjects experienced no adverse events (AEs). Seven subjects reported AEs that were mild, transient and consistent with adverse events typically noted with cholinesterase inhibitors. Significant improvements were found in overall adaptive function (Vineland Adaptive Behavior Scales and Clinician's Interview-Based Impression of Change), attention (Leiter Attention Sustained tests A and B), memory (NEPSY: Narrative and Immediate Memory for Names subtests) and language (Test of Verbal Expression and Reasoning and Clinical Evaluation of Language Fundamentals-Preschool) domains. Improved language performance was found across all functional levels. These results underscore the need for larger, controlled studies employing a carefully constructed test battery capable of measuring the full scope of performance across multiple domains and a wide range of functional levels.

摘要

唐氏综合征(DS)患者表现出与阿尔茨海默病相似的胆碱能缺乏。用于治疗阿尔茨海默病的胆碱酯酶抑制剂可能会改善DS患者的认知功能。这是首次关于利伐斯的明(一种乙酰胆碱酯酶和丁酰胆碱酯酶抑制剂)对儿童DS特定认知领域安全性和有效性的研究。11名DS患者(年龄10 - 17岁)接受了利伐斯的明液体制剂治疗。4名患者未出现不良事件(AE)。7名患者报告了轻度、短暂的AE,且与胆碱酯酶抑制剂通常出现的不良事件一致。在总体适应功能(文兰适应行为量表和基于临床医生访谈的变化印象)、注意力(莱特持续性注意力测试A和B)、记忆(NEPSY:叙事和名字即时记忆子测试)和语言(言语表达与推理测试和语言基础学前临床评估)领域发现了显著改善。在所有功能水平上都发现了语言表现的改善。这些结果强调需要进行更大规模的对照研究,采用精心构建的测试组合,能够测量多个领域和广泛功能水平上的全面表现。

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本文引用的文献

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Rivastigmine in the treatment of dementia in Alzheimer's disease in adults with Down syndrome.卡巴拉汀用于治疗患有唐氏综合征的成年阿尔茨海默病患者的痴呆症。
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Rivastigmine for dementia associated with Parkinson's disease.卡巴拉汀用于帕金森病所致痴呆。
N Engl J Med. 2004 Dec 9;351(24):2509-18. doi: 10.1056/NEJMoa041470.
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The neuropsychology of Down syndrome: evidence for hippocampal dysfunction.唐氏综合征的神经心理学:海马体功能障碍的证据。
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