McGuire Joseph F, Karkanias George B, Bittman Richard M, Atkinson Sarah D, Munschauer Frederick E, Wanaski Stephen P, Cunniff Timothy M, Gilbert Donald L
Department of Psychiatry and Behavioral Sciences, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
Emalex Biosciences, Inc., Chicago, Illinois, USA.
J Child Adolesc Psychopharmacol. 2025 May 28. doi: 10.1089/cap.2025.0036.
Accurate assessment of treatment outcomes in patients with Tourette syndrome (TS) is essential for evidence-based clinical care. This report determined the minimal clinically important difference (MCID) on the Yale Global Tic Severity Scale (YGTSS) Total Tic Score (YGTSS-TTS) and YGTSS Impairment Scale (YGTSS-I), using the Clinical Global Impression of TS Severity (CGI-TS-S) and Improvement (CGI-TS-I) as anchors, in pediatric patients with TS receiving pharmacotherapy. Analyses used data from two clinical trials of ecopipam (a randomized controlled trial and its open-label extension). Receiver operating characteristic (ROC) analysis determined the percentage reduction in YGTSS scores that distinguished patients with improvement from those with no change or worsening on the CGI-TS-S and CGI-TS-I. Spearman's correlation, empirical cumulative distribution function, and probability distribution function analyses examined relationships between YGTSS-TTS and CGI-TS-S or CGI-TS-I. Overall, 133 patients (75.2% male; mean [SD] age, 12.7 [2.8]) were included; 63.2% had improvement on the CGI-TS-S, and 78.2% showed improvement on the CGI-TS-I. Percentage reduction in YGTSS scores that distinguished improvement from no change or worsening on the CGI-TS-S and CGI-TS-I ranged from 18.6%-33.3% (area under the ROC curve range, 0.71-0.81). Improvement on the YGTSS-TTS was correlated with posttreatment CGI-TS-S ( = -0.65; < 0.001) and CGI-TS-I ( = -0.61; < 0.001) scores. The MCID for YGTSS-TTS was achieved by 67% and 62% of patients with improvement on the CGI-TS-S and CGI-TS-I, respectively. This analysis is the first to determine the MCID for YGTSS in a pediatric population with TS receiving pharmacotherapy. Whether using CGI-TS-S or CGI-TS-I as the anchor, a 25% reduction in YGTSS scores was a generally appropriate minimum threshold to define clinically meaningful improvement in this population. Findings offer an objective threshold for classifying clinically meaningful improvement in children and adolescents receiving pharmacotherapy for TS in clinical practice.
准确评估抽动秽语综合征（TS）患者的治疗效果对于循证临床护理至关重要。本报告以抽动秽语综合征严重程度临床总体印象（CGI - TS - S）和改善情况（CGI - TS - I）为锚定指标，确定了接受药物治疗的小儿TS患者在耶鲁综合抽动严重程度量表（YGTSS）总抽动评分（YGTSS - TTS）和YGTSS损害量表（YGTSS - I）上的最小临床重要差异（MCID）。分析使用了来自依考必利两项临床试验的数据（一项随机对照试验及其开放标签扩展试验）。受试者操作特征（ROC）分析确定了YGTSS评分的降低百分比，该百分比可区分出在CGI - TS - S和CGI - TS - I上有改善的患者与无变化或病情恶化的患者。Spearman相关性分析、经验累积分布函数分析和概率分布函数分析研究了YGTSS - TTS与CGI - TS - S或CGI - TS - I之间的关系。总体而言，纳入了133例患者（75.2%为男性；平均[标准差]年龄为12.7[2.8]岁）；63.2%的患者在CGI - TS - S上有改善，78.2%的患者在CGI - TS - I上有改善。在CGI - TS - S和CGI - TS - I上，能够区分改善与无变化或病情恶化的YGTSS评分降低百分比范围为18.6% - 33.3%（ROC曲线下面积范围为0.71 - 0.81）。YGTSS - TTS的改善与治疗后CGI - TS - S（r = - 0.65；P < 0.001）和CGI - TS - I（r = - 0.61；P < 0.001）评分相关。在CGI - TS - S和CGI - TS - I上有改善的患者中，分别有67%和62%的患者达到了YGTSS - TTS的MCID。该分析首次确定了接受药物治疗的小儿TS患者群体中YGTSS的MCID。无论以CGI - TS - S还是CGI - TS - I作为锚定指标，YGTSS评分降低25%通常是定义该群体中有临床意义改善的合适最低阈值。研究结果为临床实践中接受TS药物治疗的儿童和青少年有临床意义的改善分类提供了一个客观阈值。
