Neurology. 2006 Feb 14;66(3):366-72. doi: 10.1212/01.wnl.0000198586.85250.13.
Tetrabenazine (TBZ) selectively depletes central monoamines by reversibly binding to the type 2 vesicular monoamine transporter. Open-label reports indicate TBZ is effective in treating chorea.
To examine the safety, efficacy, and dose tolerability of TBZ for treating chorea in Huntington disease (HD).
The authors randomized 84 ambulatory patients with HD to receive TBZ (n = 54) or placebo (n = 30) for 12 weeks. TBZ was increased over 7 weeks up to a maximum of 100 mg/day or until the desired antichoreic effect occurred or intolerable adverse effects supervened. The primary outcome was the change from baseline in the chorea score of the Unified Huntington's Disease Rating Scale (UHDRS) RESULTS: TBZ treatment resulted in a reduction of 5.0 units in chorea severity compared with a reduction of 1.5 units on placebo treatment (adjusted mean effect size = -3.5 +/- 0.8 UHDRS units [mean +/- SE]; 95% CI: -5.2, -1.9; p < 0.0001). There was also a significant benefit on ratings of clinical global improvement. There were five study withdrawals in the TBZ group and five serious adverse events (SAEs) in four subjects (drowning suicide, complicated fall, restlessness/suicidal ideation, and breast cancer) compared with one withdrawal and no SAEs in the placebo group.
Tetrabenazine (TBZ), at adjusted dosages of up to 100 mg/day, effectively lessens chorea in ambulatory patients with Huntington disease. TBZ should be dosed individually based on ongoing assessment of possible adverse side effects.
丁苯那嗪（TBZ）通过与2型囊泡单胺转运体可逆性结合，选择性地消耗中枢单胺类神经递质。开放性报告表明，丁苯那嗪在治疗舞蹈症方面有效。
研究丁苯那嗪治疗亨廷顿病（HD）舞蹈症的安全性、有效性和剂量耐受性。
作者将84例能够自主活动的HD患者随机分为两组，分别接受丁苯那嗪治疗（n = 54）或安慰剂治疗（n = 30），为期12周。丁苯那嗪剂量在7周内逐渐增加，最大剂量为每日100 mg，直至出现预期的抗舞蹈症效果或出现无法耐受的不良反应。主要观察指标为统一亨廷顿病评定量表（UHDRS）中舞蹈症评分相对于基线的变化。结果：与安慰剂治疗使舞蹈症严重程度降低1.5个单位相比，丁苯那嗪治疗使舞蹈症严重程度降低了5.0个单位（校正平均效应量=-3.5±0.8 UHDRS单位[均值±标准误]；95%置信区间：-5.2，-1.9；p<0.0001）。在临床总体改善评分方面也有显著益处。丁苯那嗪组有5例患者退出研究，4例患者出现5起严重不良事件（溺水自杀、复杂跌倒、烦躁/自杀观念和乳腺癌），而安慰剂组有1例患者退出研究，无严重不良事件发生。
调整剂量至每日100 mg时，丁苯那嗪可有效减轻能够自主活动的HD患者的舞蹈症症状。应根据对可能出现的不良反应的持续评估对丁苯那嗪进行个体化给药。
