Centre for Psychiatry Research, Department of Clinical Neuroscience, Karolinska Institutet, Stockholm, Sweden.
Stockholm Health Care Services, Region Stockholm, Stockholm, Sweden.
JAMA Netw Open. 2022 Aug 1;5(8):e2225614. doi: 10.1001/jamanetworkopen.2022.25614.
The availability of behavior therapy for individuals with Tourette syndrome (TS) and chronic tic disorder (CTD) is limited.
To determine the efficacy and cost-effectiveness of internet-delivered exposure and response prevention (ERP) for children and adolescents with TS or CTD.
DESIGN, SETTING, AND PARTICIPANTS: This single-masked, parallel group, superiority randomized clinical trial with nationwide recruitment was conducted at a research clinic in Stockholm, Sweden. Out of 615 individuals assessed for eligibility, 221 participants meeting diagnostic criteria for TS or CTD and aged 9 to 17 years were included in the study. Enrollment began in April 2019 and ended in April 2021. Data were analyzed between October 2021 and March 2022.
Participants were randomized to 10 weeks of therapist-supported internet-delivered ERP for tics (111 participants) or to therapist-supported internet-delivered education for tics (comparator group, 110 participants).
The primary outcome was change in tic severity from baseline to the 3-month follow-up as measured by the Total Tic Severity Score of the Yale Global Tic Severity Scale (YGTSS-TTSS). YGTSS-TTSS assessors were masked to treatment allocation. Treatment response was operationalized as a score of 1 ("Very much improved") or 2 ("Much improved") on the Clinical Global Impression-Improvement scale.
Data loss was minimal, with 216 of 221 participants (97.7%) providing primary outcome data. Among randomized participants (152 [68.8%] boys; mean [SD] age, 12.1 [2.3] years), tic severity improved significantly, with a mean reduction of 6.08 points on the YGTSS-TTSS in the ERP group (mean [SD] at baseline, 22.25 [5.60]; at 3-month follow-up, 16.17 [6.82]) and 5.29 in the comparator (mean [SD] at baseline, 23.01 [5.92]; at 3-month follow-up, 17.72 [7.11]). Intention-to-treat analyses showed that the 2 groups improved similarly over time (interaction effect, -0.53; 95% CI, -1.28 to 0.22; P = .17). Significantly more participants were classified as treatment responders in the ERP group (51 of 108 [47.2%]) than in the comparator group (31 of 108 [28.7%]) at the 3-month follow-up (odds ratio, 2.22; 95% CI, 1.27 to 3.90). ERP resulted in more treatment responders at little additional cost compared with structured education. The incremental cost per quality-adjusted life-year gained was below the Swedish willingness-to-pay threshold, at which ERP had a 66% to 76% probability of being cost-effective.
Both interventions were associated with clinically meaningful improvements in tic severity, but ERP led to higher response rates at little additional cost.
ClinicalTrials.gov identifier: NCT03916055.
对于患有妥瑞氏综合征（TS）和慢性抽动障碍（CTD）的个体，行为治疗的可及性有限。
确定互联网提供的暴露和反应预防（ERP）对患有 TS 或 CTD 的儿童和青少年的疗效和成本效益。
设计、设置和参与者：这是一项在瑞典斯德哥尔摩的研究诊所进行的、具有全国招募的、单盲、平行组、优势随机临床试验。在评估了 615 名符合条件的个体后，221 名符合 TS 或 CTD 诊断标准且年龄在 9 至 17 岁的参与者被纳入研究。招募于 2019 年 4 月开始，2021 年 4 月结束。数据分析于 2021 年 10 月至 2022 年 3 月进行。
参与者被随机分配到 10 周的由治疗师支持的互联网提供的 tic ERP（111 名参与者）或由治疗师支持的互联网提供的 tic 教育（对照组，110 名参与者）。
主要结局是从基线到 3 个月随访时耶鲁总体 tic 严重程度量表（YGTSS-TTSS）的 tic 严重程度变化。YGTSS-TTSS 评估者对治疗分配进行了盲法。治疗反应的定义为临床总体印象-改善量表上的得分为 1（“明显改善”）或 2（“明显改善”）。
数据丢失很少，221 名参与者中有 216 名（97.7%）提供了主要结局数据。在随机参与者中（152 名[68.8%]男孩；平均[SD]年龄，12.1[2.3]岁），tic 严重程度显著改善，ERP 组 YGTSS-TTSS 平均降低 6.08 分（基线平均[SD]，22.25[5.60]；3 个月随访时，16.17[6.82]），对照组降低 5.29 分（基线平均[SD]，23.01[5.92]；3 个月随访时，17.72[7.11]）。意向治疗分析显示，两组随时间推移改善相似（交互效应，-0.53；95%CI，-1.28 至 0.22；P = .17）。在 3 个月随访时，ERP 组（51 名[47.2%]）比对照组（31 名[28.7%]）中更多的参与者被归类为治疗应答者（优势比，2.22；95%CI，1.27 至 3.90）。与结构化教育相比，ERP 以较低的额外成本导致更多的治疗应答者。每获得一个质量调整生命年的增量成本低于瑞典支付意愿阈值，在此阈值下，ERP 具有 66%至 76%的成本效益概率。
两种干预措施都与 tic 严重程度的临床显著改善相关，但 ERP 以较低的额外成本导致了更高的应答率。
ClinicalTrials.gov 标识符：NCT03916055。
