美国杜克大学Linmarie Sikich团队研究了自闭症谱系障碍儿童和青少年的鼻内催产素治疗效果。相关论文发表在2021年10月13日出版的《新英格兰医学杂志》上。

实验性研究和小型临床试验表明，鼻内催产素治疗可减少自闭症谱系障碍患者的社交障碍。临床实践中，催产素被用于治疗许多患有自闭症谱系障碍的儿童。

研究组针对3至17岁患有自闭症谱系障碍的儿童和青少年进行了一项为期24周、安慰剂对照的鼻内催产素治疗的临床2期试验。将参与者按1：1随机分配，并根据年龄和语言流利程度进行分层，分别接受经鼻给予的催产素或安慰剂治疗，总目标剂量为每日48国际单位。主要结局为异常行为检查表改良的社会退缩行为量表（ABC mSW）从基线检查时的最小二乘平均值变化，该量表包括13个项目（0-39分，分数越高表明社会交往越少）。次要结局包括两个额外的社会功能指标和一个智商的简化指标。

在接受筛查的355名儿童和青少年中，共有290人登记。其中146名参与者被分配到催产素组，144名被分配到安慰剂组；分别有139名和138名完成了基线检查和至少一项基线检查后的ABC mSW评估，并被纳入改良意向治疗分析。催产素组的ABC mSW评分与基线相比的最小二乘平均变化为−3.7分，安慰剂组为−3.5分，组间差异不显著。两组间的次要结局均没有差异。两组不良事件的发生率和严重程度相似。

研究结果表明，对于患有自闭症谱系障碍的儿童和青少年，进行鼻内催产素治疗24周，与安慰剂相比，从基线测量社会或认知功能的最小二乘平均值变化方面，组间无显著差异。

附：英文原文

Title: Intranasal Oxytocin in Children and Adolescents with Autism Spectrum Disorder

Author: Linmarie Sikich, M.D.,, Alexander Kolevzon, M.D.,, Bryan H. King, M.D.,, Christopher J. McDougle, M.D.,, Kevin B. Sanders, M.D.,, Soo-Jeong Kim, M.D.,, Marina Spanos, Ph.D.,, Tara Chandrasekhar, M.D.,, M.D. Pilar Trelles, M.D.,, Carol M. Rockhill, M.D.,, Michelle L. Palumbo, M.D.,, Allyson Witters Cundiff, M.D.,, Alicia Montgomery, M.B., B.S.,, Paige Siper, Ph.D.,, Mendy Minjarez, Ph.D.,, Lisa A. Nowinski, Ph.D.,, Sarah Marler, M.A.,, Lauren C. Shuffrey, Ph.D.,, Cheryl Alderman, B.S.,, Jordana Weissman, B.A.,, Brooke Zappone, B.S.,, Jennifer E. Mullett, R.N.,, Hope Crosson, B.S.W.,, Natalie Hong, M.S.,, Stephen K. Siecinski, B.S.,, Stephanie N. Giamberardino, Sc.M.,, Sheng Luo, Ph.D.,, Lilin She, Ph.D.,, Manjushri Bhapkar, M.S.,, Russell Dean, M.S.,, Abby Scheer, M.S.I.S.,, Jacqueline L. Johnson, Dr.P.H.,, Simon G. Gregory, Ph.D.,, and Jeremy Veenstra-VanderWeele, M.D.

Issue&Volume: 2021-10-13

Abstract:

Background

Experimental studies and small clinical trials have suggested that treatment with intranasal oxytocin may reduce social impairment in persons with autism spectrum disorder. Oxytocin has been administered in clinical practice to many children with autism spectrum disorder.

Methods

We conducted a 24-week, placebo-controlled phase 2 trial of intranasal oxytocin therapy in children and adolescents 3 to 17 years of age with autism spectrum disorder. Participants were randomly assigned in a 1:1 ratio, with stratification according to age and verbal fluency, to receive oxytocin or placebo, administered intranasally, with a total target dose of 48 international units daily. The primary outcome was the least-squares mean change from baseline on the Aberrant Behavior Checklist modified Social Withdrawal subscale (ABC-mSW), which includes 13 items (scores range from 0 to 39, with higher scores indicating less social interaction). Secondary outcomes included two additional measures of social function and an abbreviated measure of IQ.

Results

Of the 355 children and adolescents who underwent screening, 290 were enrolled. A total of 146 participants were assigned to the oxytocin group and 144 to the placebo group; 139 and 138 participants, respectively, completed both the baseline and at least one postbaseline ABC-mSW assessments and were included in the modified intention-to-treat analyses. The least-squares mean change from baseline in the ABC-mSW score (primary outcome) was 3.7 in the oxytocin group and 3.5 in the placebo group (least-squares mean difference, 0.2; 95% confidence interval, 1.5 to 1.0; P=0.61). Secondary outcomes generally did not differ between the trial groups. The incidence and severity of adverse events were similar in the two groups.

Conclusions

This placebo-controlled trial of intranasal oxytocin therapy in children and adolescents with autism spectrum disorder showed no significant between-group differences in the least-squares mean change from baseline on measures of social or cognitive functioning over a period of 24 weeks.

DOI: 10.1056/NEJMoa2103583

Source: https://www.nejm.org/doi/full/10.1056/NEJMoa2103583