Baclofen treatment in Tourette syndrome - A double-blind, placebo-controlled, crossover trial

Objective: To investigate the effectiveness of baclofen for the treatment o f ties in children with Tourette syndrome (TS). Background: Baclofen, which contains both gamma -aminobutyric acid (GABA) and phenylethylamine moietie s, 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 m edication in children with TS. Methods: Subjects received, in a randomized sequence, 4-week medication cycles of baclofen (20 mg three times daily) an d placebo with a 2-week intervening washout period between the cycles. Outc ome measures included the Clinical Global Impression (CGI) scale, and the Y ale Global Tic Severity Scale (YGTSS), the latter including subscales for t otal ties and overall impairment. Measures were assessed at baseline and on days 28, 42, and 70 of the study. Results: Ten children (seven boys and th ree girls, aged 8 to 14) with TS participated. Nine subjects completed the protocol; one dropped out for psychosocial reasons. No major side effects w ere reported. The mean change in CGI score (-0.9) after 4 weeks of baclofen treatment as compared with placebo treatment showed a significant improvem ent (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 Y GTSS 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 stat istically significant effect on both outcome measures. Subscales of the YGT SS showed that the reduction in total tic scores was primarily due to a red uction in the impairment score rather than a decrease in ties. Conclusions: Children with TS may benefit from treatment with baclofen, although improv ements may be related to factors other than ties. Larger studies directly c omparing baclofen against other tie-suppressing agents are recommended.