Singer H S, Wendlandt J, Krieger M, Giuliano J
Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Neurology. 2001 Mar 13;56(5):599-604. doi: 10.1212/wnl.56.5.599.
To investigate the effectiveness of baclofen for the treatment of tics in children with Tourette syndrome (TS).
Baclofen, which contains both gamma-aminobutyric acid (GABA) and phenylethylamine moieties, was suggested in an open-label protocol to be an effective treatment for TS. This is a double-blind, placebo-controlled study to investigate this medication in children with TS.
Subjects received, in a randomized sequence, 4-week medication cycles of baclofen (20 mg three times daily) and placebo with a 2-week intervening washout period between the cycles. Outcome measures included the Clinical Global Impression (CGI) scale, and the Yale Global Tic Severity Scale (YGTSS), the latter including subscales for total tics and overall impairment. Measures were assessed at baseline and on days 28, 42, and 70 of the study.
Ten children (seven boys and three girls, aged 8 to 14) with TS participated. Nine subjects completed the protocol; one dropped out for psychosocial reasons. No major side effects were reported. The mean change in CGI score (-0.9) after 4 weeks of baclofen treatment as compared with placebo treatment showed a significant improvement (95% CI, -1.7 to -0.1; p = 0.04). All subjects showed some amelioration in total YGTSS score during baclofen treatment. The mean change in total YGTSS score (-14.7) approached significance (95% CI, -30.3 to 0.9; p = 0.06). Examination of differences between baclofen and placebo treatment groups expressed as a percent change from baseline showed that baclofen had a statistically significant effect on both outcome measures. Subscales of the YGTSS showed that the reduction in total tic scores was primarily due to a reduction in the impairment score rather than a decrease in tics.
Children with TS may benefit from treatment with baclofen, although improvements may be related to factors other than tics. Larger studies directly comparing baclofen against other tic-suppressing agents are recommended.
探讨巴氯芬治疗抽动秽语综合征（TS）患儿抽动症状的有效性。
巴氯芬含有γ-氨基丁酸（GABA）和苯乙胺部分，在一项开放标签方案中被认为是治疗TS的有效药物。这是一项双盲、安慰剂对照研究，旨在调查该药对TS患儿的疗效。
受试者按随机顺序接受巴氯芬（每日三次，每次20毫克）和安慰剂的4周用药周期，周期之间有2周的洗脱期。疗效指标包括临床总体印象（CGI）量表和耶鲁全球抽动严重程度量表（YGTSS），后者包括总抽动和总体损害的子量表。在研究的基线以及第28、42和70天对指标进行评估。
10名TS患儿（7名男孩和3名女孩，年龄8至14岁）参与研究。9名受试者完成了方案；1名因社会心理原因退出。未报告重大副作用。与安慰剂治疗相比，巴氯芬治疗4周后CGI评分的平均变化（-0.9）显示有显著改善（95%CI，-1.7至-0.1；p = 0.04）。所有受试者在巴氯芬治疗期间总YGTSS评分均有一定改善。总YGTSS评分的平均变化（-14.7）接近显著水平（95%CI，-30.3至0.9；p = 0.06）。以相对于基线的百分比变化表示的巴氯芬与安慰剂治疗组之间差异的检验表明，巴氯芬对两个疗效指标均有统计学显著影响。YGTSS的子量表显示，总抽动评分的降低主要是由于损害评分的降低而非抽动次数的减少。
TS患儿可能从巴氯芬治疗中获益，尽管改善可能与抽动以外的因素有关。建议进行更大规模的研究，直接比较巴氯芬与其他抽动抑制药物。
