低剂量舍曲林治疗脆性X综合征幼儿的随机、双盲、安慰剂对照试验
A Randomized, Double-Blind, Placebo-Controlled Trial of Low-Dose Sertraline in Young Children With Fragile X Syndrome.
作者信息
Greiss Hess Laura, Fitzpatrick Sarah E, Nguyen Danh V, Chen Yanjun, Gaul Kimberly N, Schneider Andrea, Lemons Chitwood Kerrie, Eldeeb Marwa Abd Al Azaim, Polussa Jonathan, Hessl David, Rivera Susan, Hagerman Randi J
机构信息
*Medical Investigation of Neurodevelopmental Disorders (MIND) Institute, University of California, Davis Medical Center, Sacramento, CA; †Department of Occupational Therapy, Dominican University of California, San Rafael, CA; ‡Department of Neuroscience, The Ohio State University, Columbus, OH; §Department of Medicine, University of California, Irvine School of Medicine, Orange, CA; ‖Institute for Clinical and Translational Science, University of California, Irvine, CA; ¶Department of Psychology, University of California, Davis, Davis, CA; **Department of Pediatrics, University of California, Davis Medical Center, Sacramento, CA; ††Department of Special Education, California State University, Monterey Bay, CA; ‡‡Department of Psychiatry and Behavioral Sciences, University of California, Davis Medical Center, Sacramento, CA.
出版信息
J Dev Behav Pediatr. 2016 Oct;37(8):619-28. doi: 10.1097/DBP.0000000000000334.
OBJECTIVE
Observational studies and anecdotal reports suggest that sertraline, a selective serotonin reuptake inhibitor, may improve language development in young children with fragile X syndrome (FXS).
METHODS
The authors evaluated the efficacy of 6 months of treatment with low-dose sertraline in a randomized, double-blind, placebo-controlled trial in 52 children with FXS aged 2 to 6 years.
RESULTS
Eighty-one subjects were screened for eligibility, and 57 were randomized to sertraline (27) or placebo (30). Two subjects from the sertraline arm and 3 from the placebo arm discontinued. Intent-to-treat analysis showed no difference from placebo on the primary outcomes: the Mullen Scales of Early Learning (MSEL) expressive language (EL) age equivalent and Clinical Global Impression Scale-Improvement. However, analyses of secondary measures showed significant improvements, particularly in motor and visual perceptual abilities and social participation. Sertraline was well tolerated, with no difference in side effects between sertraline and placebo groups. No serious adverse events occurred.
CONCLUSION
This randomized controlled trial of 6 months of sertraline treatment showed no primary benefit with respect to early EL development and global clinical improvement. However, in secondary exploratory analyses, there were significant improvements seen on motor and visual perceptual subtests, the cognitive T score sum on the MSEL, and on one measure of social participation on the Sensory Processing Measure-Preschool. Furthermore, post hoc analysis found significant improvement in early EL development as measured by the MSEL among children with autism spectrum disorder on sertraline. Treatment appears safe for this 6-month period in young children with FXS, but the authors do not know the long-term side effects of this treatment. These results warrant further studies of sertraline in young children with FXS using refined outcome measures as well as longer term follow-up studies to address long-term side effects of low-dose sertraline in early childhood.
目的
观察性研究和轶事报告表明，选择性5-羟色胺再摄取抑制剂舍曲林可能改善脆性X综合征（FXS）幼儿的语言发育。
方法
作者在一项随机、双盲、安慰剂对照试验中，评估了低剂量舍曲林治疗6个月对52名2至6岁FXS儿童的疗效。
结果
对81名受试者进行了资格筛查，57名被随机分为舍曲林组（27名）或安慰剂组（30名）。舍曲林组有2名受试者、安慰剂组有3名受试者退出。意向性分析显示，在主要结局指标上与安慰剂无差异：早期学习穆伦量表（MSEL）表达性语言（EL）年龄当量和临床总体印象量表-改善情况。然而，次要指标分析显示有显著改善，尤其是在运动和视觉感知能力以及社会参与方面。舍曲林耐受性良好，舍曲林组和安慰剂组的副作用无差异。未发生严重不良事件。
结论
这项为期6个月的舍曲林治疗随机对照试验显示，在早期EL发育和总体临床改善方面没有主要益处。然而，在次要探索性分析中，运动和视觉感知子测试、MSEL上的认知T分数总和以及感觉统合量表-学前版的一项社会参与指标有显著改善。此外，事后分析发现，舍曲林治疗的自闭症谱系障碍儿童中，MSEL测量的早期EL发育有显著改善。对于FXS幼儿，这种治疗在6个月期间似乎是安全的，但作者不知道这种治疗的长期副作用。这些结果需要使用更精确的结局指标对FXS幼儿舍曲林进行进一步研究，以及进行长期随访研究，以解决幼儿期低剂量舍曲林的长期副作用问题。
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