Heller James H, Spiridigliozzi Gail A, Crissman Blythe G, McKillop Jane Anne, Yamamoto Haru, Kishnani Priya S
Department of Surgery, Duke University Medical Center, Durham, North Carolina 27710, USA.
J Child Adolesc Psychopharmacol. 2010 Dec;20(6):517-20. doi: 10.1089/cap.2009.0099.
Following the completion of a 20-week, open-label study of the safety and efficacy of liquid rivastigmine for adolescents with Down syndrome, 5 of the 10 adolescents in the clinical trial continued long-term rivastigmine therapy and 5 did not. After an average period of 38 months, all 10 subjects returned for a follow-up assessment to determine the safety and efficacy of long-term rivastigmine use. Rivastigmine was well tolerated and overall health appeared to be unaffected by long-term rivastigmine use. Performance change on cognitive and language measures administered at the termination of the open-label clinical trial was compared between the two groups. No between-group difference in median performance change across the long-term period was found, suggesting that the long-term use of rivastigmine does not improve cognitive and language performance. However, two subjects demonstrated remarkable improvement in adaptive function over the long-term period. Both subjects had received long-term rivastigmine therapy. The discussion addresses the challenge of assessing cognitive change in clinical trials using adolescents with Down syndrome as subjects and the use of group versus individual data to evaluate the relevance of medication effects.
在完成一项针对唐氏综合征青少年的20周开放性液体卡巴拉汀安全性和有效性研究后，临床试验中的10名青少年中有5名继续接受长期卡巴拉汀治疗，5名未接受。平均38个月后，所有10名受试者返回进行随访评估，以确定长期使用卡巴拉汀的安全性和有效性。卡巴拉汀耐受性良好，长期使用卡巴拉汀似乎对整体健康没有影响。比较了两组在开放性临床试验结束时进行的认知和语言测量的表现变化。在长期期间，两组之间未发现中位数表现变化的组间差异，这表明长期使用卡巴拉汀并不能改善认知和语言表现。然而，两名受试者在长期期间适应性功能有显著改善。两名受试者均接受了长期卡巴拉汀治疗。讨论涉及以唐氏综合征青少年为受试者的临床试验中评估认知变化的挑战，以及使用组数据与个体数据来评估药物效果的相关性。
