Donepezil for Treatment of Cognitive Dysfunction in Children With Down Syndrome Aged 10-17
Donepezil for Treatment of Cognitive Dysfunction in Children With Down Syndrome Aged 10-17
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DOI：
10.1002/ajmg.a.33730
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发表时间：
2010-12-01
影响因子：
2
通讯作者：
McRae, Thomas
中科院分区：
文献类型：
--
作者：
Kishnani, Priya S.;Heller, James H.;McRae, Thomas
这项为期10周的随机、双盲、安慰剂对照多中心研究的目的是评估多奈哌齐治疗唐氏综合征(DS)儿童认知功能障碍的有效性和安全性。干预措施包括对患有轻中度DS的儿童(10-17岁)给予多奈哌齐(2.5-10 mg/天)。主要测量内容为Vineland-II适应行为量表(VABS-II)、父母/照顾者评定表(PCRF)、9个子域标准化分数和标准安全措施的总和。次级测量包括以下领域及其各自子领域的VABS-II/PCRF分数：沟通(接受性、表现性和书面性)；日常生活技能(个人、家庭和社区)；社会化(人际关系、游戏和休闲时间以及应对技能)，以及言语表达和推理测试的分数，这是一种基于学科表现的表达语言测试。在基线时，129名参与者被分配给多奈哌齐或安慰剂治疗。在双盲阶段，两组的9个亚域标准化分数的VABSII/PCRF值之和称为v分数，较基线显著改善(P<；0.0001)，组间无显著差异。这项试验没有证明多奈哌齐与安慰剂相比对患有DS的儿童和青少年有任何益处，尽管多奈哌齐似乎耐受性很好。(C)2010年Wiley-Liss公司
The objective of this 10-week, randomized, double-blind, placebo-controlled multicenter study was to assess the efficacy and safety of donepezil for the treatment of cognitive dysfunction exhibited by children with Down syndrome (DS). Intervention comprised donepezil (2.5-10 mg/day) in children (aged 10-17 years) with DS of mild-to-moderate severity. The primary measures were the Vineland-II Adaptive Behavior Scales (VABS-II) Parent/Caregiver Rating Form (PCRF) the sum of nine subdomain standardized scores and standard safety measures. Secondary measures included the VABS-II/PCRF scores on the following domains and their respective individual subdomains: Communication (receptive, expressive, and written); Daily Living Skills (personal, domestic, and community); Socialization (interpersonal relationships, play and leisure time, and coping skills), and scores on the Test of Verbal Expression and Reasoning, a subject-performance-based measure of expressive language. At baseline, 129 participants were assigned treatment with donepezil or placebo. During the double-blind phase, VABS II/PCRF sum of the nine subdomain standardized scores, called v-scores, improved significantly from baseline in both groups (P < 0.0001), with no significant between-group differences. This trial failed to demonstrate any benefit for donepezil versus placebo in children and adolescents with DS, although donepezil appeared to be well tolerated. (C) 2010 Wiley-Liss, Inc.