A prospective open-label study of aripiprazole in fragile X syndrome
A prospective open-label study of aripiprazole in fragile X syndrome
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DOI：
10.1007/s00213-011-2194-7
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发表时间：
2011-07-01
影响因子：
3.4
通讯作者：
McDougle, Christopher J.
中科院分区：
文献类型：
--
作者：
Erickson, Craig A.;Stigler, Kimberly A.;McDougle, Christopher J.
脆性X综合征（FXS）是最常见的遗传性发育障碍形式，也是自闭症最常见的单基因原因。FXS患者经常表现出以攻击、自伤和严重发脾气为特征的易怒行为。尽管临床上经常使用非典型抗精神病药物来治疗这种行为，但迄今为止还没有系统性试验评估这些药物在FXS患者中的疗效和安全性。（平均年龄14.3岁），无合并精神活性药物。（平均剂量，9.8 mg/天）与治疗反应相关（定义为临床总体印象-改善量表评分大大改善或非常改善，且异常行为检查表改善≥ 25%-10/12例（87%）受试者的易怒子量表）。两名个体（13%）在研究完成前因不良事件停用阿立哌唑。1例停药是由于静坐不能、轻度流涎和轻度疲乏，另1例是由于中度疲乏和中度流涎。阿立哌唑治疗期间，包括体重或实验室指标的生命体征没有显着变化。结论阿立哌唑一般是安全的，耐受性良好，并与显着改善易激惹的行为。鉴于这些发现，有必要进行阿立哌唑治疗FXS的双盲、安慰剂对照研究。
Rationale Fragile X syndrome (FXS) is the most common inherited form of developmental disability and most common single gene cause of autism. Persons with FXS frequently exhibit irritable behavior marked by aggression, self-injury, and severe tantrums. Despite frequent clinical use of atypical antipsychotic drugs to target this behavioral cluster, no systematic trials to date have assessed the efficacy and safety of these drugs in persons with FXS.Methods We conducted a prospective open-label 12-week trial of aripiprazole in 12 persons aged 6-25 years (mean age, 14.3 years) with FXS who were free of concomitant psychoactive drugs.Results Aripiprazole use (mean dose, 9.8 mg/day) was associated with treatment response (defined by a Clinical Global Impressions-Improvement scale score of much improved or very much improved and a >= 25% improvement on the Aberrant Behavior Checklist-Irritability subscale) in 10 of 12 (87%) persons. Two individuals (13%) discontinued aripiprazole prior to study completion due to adverse events. One discontinuation was due to akathisia, mild drooling, and mild tiredness and the other due to moderate tiredness and moderate drooling. No significant changes in vital signs including weight or laboratory measures occurred during treatment with aripiprazole.Conclusions Aripiprazole was generally safe and well tolerated and was associated with significant improvement in irritable behavior. Given these findings, a double-blind, placebo-controlled study of aripiprazole in FXS is warranted.