目的 系統(tǒng)評價兒童抽動障礙隨機對照試驗的安慰劑效應，并探索其影響因素。方法 檢索中國學術期刊全文數(shù)據(jù)庫（CNKI）、維普中文期刊全文數(shù)據(jù)庫（VIP）、萬方數(shù)據(jù)庫、中國生物醫(yī)學文獻數(shù)據(jù)庫（CBM）、PubMed、Cochrane圖書館、Embase中關于兒童抽動障礙隨機對照試驗（RCT）中英文文獻，檢索時間均從各數(shù)據(jù)庫創(chuàng)建至2021年11月。使用R軟件對耶魯綜合抽動嚴重程度量表抽動總分（YGTSS-TTS）、耶魯綜合抽動嚴重程度量表（YGTSS）總分及基于其定義的有效率進行定量資料或定性資料的Meta分析，探索影響安慰劑效應的因素，并對影響因素進行亞組分析。結果 共納入11項RCTs，安慰劑組總樣本量606例。對于YGTSS-TTS變化均值及其有效率，Meta分析結果分別為7.2［95% CI（6.23，8.18）］和36%［95% CI（28%，45%）］；根據(jù)有效率的不同定義閾值（減分率≥25%、≥30%和≥50%）做亞組分析，其合并效應值分別為48%［95% CI（43%，53%）］、42%［95% CI（35%，49%）］和20%［95% CI（12%，29%）］。對于YGTSS總分及其有效率，Meta分析結果分別為13.89［95% CI（12.4，15.38）］和32%［95% CI（24%，41%）］；根據(jù)有效率的不同定義閾值（減分率≥30%和≥50%）做亞組分析，其合并效應值分別為40%［95% CI（34%，47%）］和22%［95% CI（14%，31%）］。對各結局指標分別進行了Meta回歸分析，結果提示疾病嚴重程度和療程，與安慰劑效應呈正相關。結論 兒童抽動障礙具有一定的安慰劑治療效應，其安慰劑效應可能與入組嚴重程度、療程等因素有關，其效應值隨著定義閾值的升高而降低。這些結果，可以為以安慰劑為對照的臨床試驗設計和樣本量估算，提供借鑒與參考。

Objective To systematically evaluate the placebo effect of randomized controlled trials of tic disorders in children and explore its influencing factors. Methods The Chinese and English literatures on children's tic disorder randomized controlled trial (RCT) in CNKI, VIP, Wanfang data, CBM, PubMed, Cochrane Library and Embase were searched from the establishment of each database to November 2021. Using R software, the total tic scores of Yale global tic severity scale (YGTSS-TTS), the total scores of Yale global tic severity scale (YGTSS) and the effective rate based on their definitions were analyzed by Meta-analysis of quantitative or qualitative data, to explore the factors affecting the placebo effect, and the influencing factors were analyzed by subgroup. Result A total of 11 RCTs were included, with a total sample size of 606 cases in the placebo group. For the mean change and effective rate of YGTSS-TTS, the results of Meta-analysis were 7.2 [95%CI (6.23, 8.18)] and 36% [95%CI (28%, 45%)] respectively; subgroup analysis was performed according to different defined thresholds of efficiency (score reduction rate ≥ 25%, ≥30% and ≥ 50%),the combined effect values were 48% [95%CI (43%, 53%)], 42% [95%CI (35%, 49%)] and 20% [95%CI (12%, 29%)] respectively. For the mean change and effective rate of total YGTSS score, the results of Meta-analysis were 13.89 [95%CI (12.4, 15.38)] and 32%[95%CI (24%, 41%)] respectively; subgroup analysis was performed according to different defined thresholds of efficiency (score reduction rate ≥ 30% and ≥ 50%),the combined effect values were 40% [95%CI (34%, 47%)] and 22% [95%CI (14%, 31%)]. Meta regression analysis was carried out for each outcome index and the results showed that the disease severity and course of treatment were positively correlated with the placebo effect. Conclusion Children with ic disorders have a certain placebo effect. The placebo effect may be related to factors such as enrollment severity and course of treatment. The effect size decreases with the increase of the defined threshold. These results can provide reference for placebo-controlled clinical trial design and sample size estimation.

R985;R969.4

“十三五”國家科技重大專項——重大新藥創(chuàng)制項目（2020ZX09201-008）