Janssen Research & Development, LLC, 1125 Trenton Harbouron Road, Titusville, NJ 08560, USA.
Kaerus Bioscience Ltd, London, UK.
Child Adolesc Psychiatr Clin N Am. 2020 Apr;29(2):301-317. doi: 10.1016/j.chc.2019.12.004. Epub 2020 Jan 31.
There are no approved medications for autism spectrum disorder (ASD) core symptoms. However, given the significant clinical need, children and adults with ASD are prescribed medication off label for core or associated conditions, sometimes based on limited evidence for effectiveness. Recent developments in the understanding of biologic basis of ASD have led to novel targets with potential to impact core symptoms, and several clinical trials are underway. Heterogeneity in course of development, co-occurring conditions, and age-related treatment response variability hampers study outcomes. Novel measures and approaches to ASD clinical trial design will help in development of effective pharmacologic treatments.
目前尚无针对自闭症谱系障碍（ASD）核心症状的获批药物。然而，鉴于临床需求巨大，患有 ASD 的儿童和成人常常会根据有限的疗效证据，超适应证使用药物来治疗核心症状或相关病症。目前人们对 ASD 生物学基础的认识不断深入，为治疗核心症状带来了新的靶点，相关临床试验也正在进行中。但是，发育过程、共病和与年龄相关的治疗反应变异性的异质性会影响研究结果。新型 ASD 临床试验设计方法和措施有助于开发有效的药物治疗方法。
