Cincinnati Children's Hospital Medical Center, Department of Pediatrics, Cincinnati, Ohio, USA.
University of South Florida, Departments of Pediatrics and Psychiatry, Tampa, Florida, USA.
Mov Disord. 2018 Aug;33(8):1272-1280. doi: 10.1002/mds.27457. Epub 2018 Sep 7.
Dopamine D2 receptor antagonists used to treat Tourette syndrome may have inadequate responses or intolerable side effects. We present results of a 4-week randomized, double-blind, placebo-controlled crossover study evaluating the safety, tolerability, and efficacy of the D1 receptor antagonist ecopipam in children and adolescents with Tourette syndrome.
Forty youth aged 7 to 17 years with Tourette syndrome and a Yale Global Tic Severity Scale - total tic score of ≥20 were enrolled and randomized to either ecopipam (50 mg/day for weight of <34 kg, 100 mg/day for weight of >34 kg) or placebo for 30 days, followed by a 2-week washout and then crossed to the alternative treatment for 30 days. Stimulants and tic-suppressing medications were excluded. The primary outcome measure was the total tic score. Secondary outcomes included obsessive compulsive and attention deficit/hyperactivity disorder scales.
Relative to changes in placebo, reduction in total tic score was greater for ecopipam at 16 days (mean difference, -3.7; 95% CI, -6.5 to -0.9; P = 0.011) and 30 days (mean difference, -3.2; 95% CI, -6.1 to -0.3; P = 0.033). There were no weight gain, drug-induced dyskinesias, or changes in laboratory tests, electrocardiograms, vital signs, or comorbid symptoms. Dropout rate was 5% (2 of 40). Adverse events reported for both treatments were rated predominantly mild to moderate, with only 5 rated severe (2 for ecopipam and 3 for placebo).
Ecopipam reduced tics and was well tolerated. This placebo-controlled study of ecopipam supports further clinical trials in children and adolescents with Tourette syndrome. © 2018 International Parkinson and Movement Disorder Society.
用于治疗妥瑞氏综合征的多巴胺 D2 受体拮抗剂可能反应不足或无法耐受副作用。我们呈现了一项为期 4 周的随机、双盲、安慰剂对照交叉研究结果，该研究评估了 D1 受体拮抗剂依匹哌唑在患有妥瑞氏综合征的儿童和青少年中的安全性、耐受性和疗效。
40 名年龄在 7 至 17 岁之间、耶鲁整体抽动严重程度量表-总抽动评分≥20 的妥瑞氏综合征青年被纳入并随机分为依匹哌唑（体重<34kg 时 50mg/天，体重>34kg 时 100mg/天）或安慰剂组，各治疗 30 天，然后停药 2 周，再交叉治疗 30 天。排除了兴奋剂和抑制抽搐的药物。主要观察指标是总抽搐评分。次要观察指标包括强迫障碍和注意缺陷/多动障碍量表。
与安慰剂相比，依匹哌唑在 16 天（平均差异，-3.7；95%置信区间，-6.5 至 -0.9；P=0.011）和 30 天（平均差异，-3.2；95%置信区间，-6.1 至 -0.3；P=0.033）时总抽搐评分的降低更为显著。没有体重增加、药物引起的运动障碍，也没有实验室检查、心电图、生命体征或共病症状的变化。退出率为 5%（40 名中的 2 名）。两种治疗方法报告的不良事件主要为轻度至中度，仅有 5 项为重度（依匹哌唑 2 项，安慰剂 3 项）。
依匹哌唑减少了抽搐，且具有良好的耐受性。这项依匹哌唑的安慰剂对照研究支持进一步在患有妥瑞氏综合征的儿童和青少年中开展临床试验。© 2018 国际帕金森病和运动障碍学会。
