Aasim Ali, Abdullah Shahid, Muhammad Usman, Asad Ullah Ansari, Labeeba Abdul Ghafoor, Csilla Dézsi, Viktor Gulyás-Oldal, Károly Szili, Muhammad Abdullah Ali, Usama Saleem, Muhammad Asad Shabbir, Armaghana Abdullah, Mukesh Kumar Sharma, Muhammad Talha
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Ecopipam is a selective dopamine D1/D5 receptor antagonist with a potentially distinct tolerability profile.</p><p><strong>Objectives: </strong>To evaluate the efficacy and safety of ecopipam in patients with Tourette syndrome using updated randomized evidence.</p><p><strong>Design: </strong>Systematic review and random-effects meta-analysis of randomized placebo-controlled trials.</p><p><strong>Data sources and methods: </strong>PubMed/MEDLINE, Embase, Scopus, ClinicalTrials.gov, reference lists, and conference sources were searched from database inception to May 31, 2026. The primary outcome was change in Yale Global Tic Severity Scale Total Tic Score. Headache and insomnia were evaluated as safety outcomes. Treatment estimates were pooled using random-effects models with Hartung-Knapp adjustment. Risk of bias was assessed using the Cochrane RoB 2 tool.</p><p><strong>Results: </strong>Three randomized trials involving 297 unique randomized participants were included. Ecopipam improved tic severity compared with placebo (mean difference, -3.72; 95% confidence interval, -5.96 to -1.47). The result remained significant after exclusion of the phase 3 randomized-withdrawal trial (mean difference, -3.33; 95% confidence interval, -4.85 to -1.81). No statistical heterogeneity was detected for efficacy outcomes, although the small number of trials limits interpretation. Headache was not significantly increased (risk ratio, 1.36; 95% confidence interval, 0.52-3.51). Insomnia showed an imprecise, nonsignificant association (risk ratio, 2.44; 95% confidence interval, 0.07-79.93; I<sup>2</sup>=62.2%).</p><p><strong>Conclusion: </strong>Short-term randomized evidence suggests that ecopipam reduces tic severity. 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Risk of bias was assessed using the Cochrane RoB 2 tool.</p><p><strong>Results: </strong>Three randomized trials involving 297 unique randomized participants were included. Ecopipam improved tic severity compared with placebo (mean difference, -3.72; 95% confidence interval, -5.96 to -1.47). The result remained significant after exclusion of the phase 3 randomized-withdrawal trial (mean difference, -3.33; 95% confidence interval, -4.85 to -1.81). No statistical heterogeneity was detected for efficacy outcomes, although the small number of trials limits interpretation. Headache was not significantly increased (risk ratio, 1.36; 95% confidence interval, 0.52-3.51). Insomnia showed an imprecise, nonsignificant association (risk ratio, 2.44; 95% confidence interval, 0.07-79.93; I<sup>2</sup>=62.2%).</p><p><strong>Conclusion: </strong>Short-term randomized evidence suggests that ecopipam reduces tic severity. 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引用次数: 0
摘要
背景：图雷特综合征是一种以持续的运动和声音抽搐为特征的神经发育障碍。多巴胺D2受体拮抗剂可以减轻抽动严重程度，但经常受到代谢、镇静和锥体外系不良反应的限制。Ecopipam是一种选择性多巴胺D1/D5受体拮抗剂，具有潜在的独特耐受性。目的：利用最新的随机证据评价ecopipam治疗抽动秽语综合征的疗效和安全性。设计：对随机安慰剂对照试验进行系统评价和随机效应荟萃分析。数据来源和方法：检索PubMed/MEDLINE、Embase、Scopus、ClinicalTrials.gov、参考文献列表和会议来源，检索时间为数据库建立至2026年5月31日。主要结局是耶鲁全球抽动严重程度量表总抽动评分的变化。头痛和失眠被评价为安全结局。使用Hartung-Knapp调整的随机效应模型汇总治疗估计。使用Cochrane RoB 2工具评估偏倚风险。结果：纳入了3项随机试验，涉及297名独特的随机受试者。与安慰剂相比，Ecopipam改善了抽动严重程度（平均差异为-3.72；95%可信区间为-5.96至-1.47）。在排除3期随机停药试验后，结果仍然显著（平均差值为-3.33；95%置信区间为-4.85至-1.81）。虽然少量试验限制了解释，但没有发现疗效结果的统计异质性。头痛没有明显增加（风险比1.36；95%可信区间0.52-3.51）。失眠表现出不精确的、不显著的关联（风险比2.44；95%可信区间0.07-79.93;I2=62.2%）。结论：短期随机证据提示ecopipam可减轻抽动严重程度。有关失眠与长期精神和代谢安全性的证据仍然不足，需要更大规模的独立试验和更长时间的随访。
Efficacy and safety of ecopipam for Tourette syndrome: An updated systematic review and meta-analysis of randomized controlled trials.
Background: Tourette syndrome is a neurodevelopmental disorder characterized by persistent motor and vocal tics. Dopamine D2 receptor antagonists can reduce tic severity but are frequently limited by metabolic, sedative, and extrapyramidal adverse effects. Ecopipam is a selective dopamine D1/D5 receptor antagonist with a potentially distinct tolerability profile.
Objectives: To evaluate the efficacy and safety of ecopipam in patients with Tourette syndrome using updated randomized evidence.
Design: Systematic review and random-effects meta-analysis of randomized placebo-controlled trials.
Data sources and methods: PubMed/MEDLINE, Embase, Scopus, ClinicalTrials.gov, reference lists, and conference sources were searched from database inception to May 31, 2026. The primary outcome was change in Yale Global Tic Severity Scale Total Tic Score. Headache and insomnia were evaluated as safety outcomes. Treatment estimates were pooled using random-effects models with Hartung-Knapp adjustment. Risk of bias was assessed using the Cochrane RoB 2 tool.
Results: Three randomized trials involving 297 unique randomized participants were included. Ecopipam improved tic severity compared with placebo (mean difference, -3.72; 95% confidence interval, -5.96 to -1.47). The result remained significant after exclusion of the phase 3 randomized-withdrawal trial (mean difference, -3.33; 95% confidence interval, -4.85 to -1.81). No statistical heterogeneity was detected for efficacy outcomes, although the small number of trials limits interpretation. Headache was not significantly increased (risk ratio, 1.36; 95% confidence interval, 0.52-3.51). Insomnia showed an imprecise, nonsignificant association (risk ratio, 2.44; 95% confidence interval, 0.07-79.93; I2=62.2%).
Conclusion: Short-term randomized evidence suggests that ecopipam reduces tic severity. Evidence concerning insomnia and long-term psychiatric and metabolic safety remains insufficient, requiring larger independent trials with longer follow-up.
期刊介绍：
Therapeutic Advances in Psychopharmacology delivers the highest quality peer-reviewed articles, reviews, and scholarly comment on pioneering efforts and innovative studies across all areas of psychopharmacology. The journal has a strong clinical and pharmacological focus and is aimed at clinicians and researchers in psychopharmacology, providing a forum in print and online for publishing the highest quality articles in this area.
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