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脆性 X 综合征患者使用米诺环素治疗的副作用及疗效评估方法的探索。

Side effects of minocycline treatment in patients with fragile X syndrome and exploration of outcome measures.

机构信息

M.I.N.D. Institute, University of California at Davis Medical Center, USA.

出版信息

Am J Intellect Dev Disabil. 2010 Sep;115(5):433-43. doi: 10.1352/1944-7558-115.5.433.

Abstract

Minocycline can rescue the dendritic spine and synaptic structural abnormalities in the fragile X knock-out mouse. This is a review and preliminary survey to document side effects and potential outcome measures for minocycline use in the treatment of individuals with fragile X syndrome. We surveyed 50 patients with fragile X syndrome who received minocycline for at least 2 weeks and found that the most common reported side effect is gastrointestinal difficulty, including loss of appetite. The families reported an improvement in language and behavioral areas. Outcome measures in the design of future randomized clinical trials should include both behavioral and language measures. As with any other treatments, we emphasize that randomized clinical trials are needed to determine the efficacy of minocycline in fragile X syndrome.

摘要

米诺环素可挽救脆性 X 敲除小鼠的树突棘和突触结构异常。本文旨在综述并初步调查米诺环素治疗脆性 X 综合征患者的副作用和潜在的结果评估方法。我们调查了 50 名接受米诺环素治疗至少 2 周的脆性 X 综合征患者,发现最常见的报告副作用是胃肠道困难,包括食欲不振。家庭报告语言和行为方面有所改善。未来随机临床试验的设计中应包括行为和语言评估方法。与任何其他治疗方法一样,我们强调需要进行随机临床试验以确定米诺环素治疗脆性 X 综合征的疗效。

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