University of California, Los Angeles, USA.
Children's Hospital of Los Angeles, USA.
Autism. 2023 May;27(4):952-966. doi: 10.1177/13623613221121425. Epub 2022 Sep 9.
Children with autism spectrum disorder are prescribed a variety of medications that affect the central nervous system (psychotropic medications) to address behavior and mood. In clinical trials, individuals taking concomitant psychotropic medications often are excluded to maintain homogeneity of the sample and prevent contamination of biomarkers or clinical endpoints. However, this choice may significantly diminish the clinical representativeness of the sample. In a recent multisite study designed to identify biomarkers and behavioral endpoints for clinical trials (the Autism Biomarkers Consortium for Clinical Trials), school-age children with autism spectrum disorder were enrolled without excluding for medications, thus providing a unique opportunity to examine characteristics of psychotropic medication use in a research cohort and to guide future decisions on medication-related inclusion criteria. The aims of the current analysis were (1) to quantify the frequency and type of psychotropic medications reported in school-age children enrolled in the ABC-CT and (2) to examine behavioral features of children with autism spectrum disorder based on medication classes. Of the 280 children with autism spectrum disorder in the cohort, 42.5% were taking psychotropic medications, with polypharmacy in half of these children. The most commonly reported psychotropic medications included melatonin, stimulants, selective serotonin reuptake inhibitors, alpha agonists, and antipsychotics. Descriptive analysis showed that children taking antipsychotics displayed a trend toward greater overall impairment. Our findings suggest that exclusion of children taking concomitant psychotropic medications in trials could limit the clinical representativeness of the study population, perhaps even excluding children who may most benefit from new treatment options.
患有自闭症谱系障碍的儿童会被开各种影响中枢神经系统的药物（精神药物）来治疗行为和情绪问题。在临床试验中，为了保持样本的同质性并防止生物标志物或临床终点受到污染，通常会排除同时服用精神药物的个体。然而，这种选择可能会显著降低样本的临床代表性。在最近一项旨在确定生物标志物和临床试验终点的多中心研究（自闭症生物标志物联盟临床试验）中，患有自闭症谱系障碍的学龄儿童被纳入研究，而不排除药物因素，从而为研究队列中精神药物使用的特征提供了独特的机会，并为未来关于药物相关纳入标准的决策提供了指导。本分析的目的是：(1) 量化 ABC-CT 研究中入组的学龄儿童报告的精神药物的频率和类型；(2) 根据药物类别检查自闭症谱系障碍儿童的行为特征。在该队列中的 280 名自闭症谱系障碍儿童中，42.5%的儿童正在服用精神药物，其中一半的儿童同时服用多种药物。报告最多的精神药物包括褪黑素、兴奋剂、选择性 5-羟色胺再摄取抑制剂、α 受体激动剂和抗精神病药。描述性分析表明，服用抗精神病药的儿童整体受损程度呈上升趋势。我们的研究结果表明，在试验中排除同时服用精神药物的儿童可能会限制研究人群的临床代表性，甚至可能排除那些最有可能从新治疗方案中受益的儿童。
