Child and Adolescent Psychiatry Department, Center for Assessment and Diagnostic of Autism, Le Vinatier Hospital, Bron, France.
Centre of Excellence and Advanced Research on Childhood Neurodevelopmental Disorders, Child Neurology Division, Department of Pediatrics, All India Institute of Medical Sciences, New Delhi, India.
Eur Child Adolesc Psychiatry. 2022 Aug;31(8):1-10. doi: 10.1007/s00787-021-01772-z. Epub 2021 Apr 7.
The lack of consensual measures to monitor core change in Autism Spectrum Disorder (ASD) or response to interventions leads to difficulty to prove intervention efficacy on ASD core symptoms. There are no universally accepted outcome measures developed for measuring changes in core symptoms. However, the CARS (Childhood Autism Rating Scale) is one of the outcomes recommended in the EMA Guideline on the clinical development of medicinal products for the treatment of ASD. Unfortunately, there is currently no consensus on the response definition for CARS among individuals with ASD. The aim of this elicitation process was to determine an appropriate definition of a response on the CARS2 scale for interventions in patients with Autism Spectrum Disorder (ASD). An elicitation process was conducted following the Sheffield Elicitation Framework (SHELF). Five experts in the field of ASD and two experts in expert knowledge elicitation participated in an 1-day elicitation workshop. Experts in ASD were previously trained in the SHELF elicitation process and received a dossier of scientific evidence concerning the topic. The response definition was set as the mean clinically relevant improvement averaged over all patients, levels of functioning, age groups and clinicians. Based on the scientific evidence and expert judgment, a normal probability distribution was agreed to represent the state of knowledge of this response with expected value 4.03 and standard deviation 0.664. Considering the remaining uncertainty of the estimation and the available literature, a CARS-2 improvement of 4.5 points has been defined as a threshold to conclude to a response after an intervention. A CARS-2 improvement of 4.5 points could be used to evaluate interventions' meaningfulness in indivudals. This initial finding represents an important new benchmark and may aid decision makers in evaluating the efficacy of interventions in ASD.
缺乏共识的监测措施来监测自闭症谱系障碍（ASD）核心变化或干预反应，导致难以证明 ASD 核心症状的干预疗效。目前没有开发出普遍接受的用于衡量核心症状变化的结果测量方法。然而，CARS（儿童自闭症评定量表）是 EMA 关于治疗 ASD 的治疗药物临床开发指南中推荐的结果之一。不幸的是，目前 ASD 患者对 CARS 的反应定义没有共识。这项启发过程的目的是确定 CARS2 量表在自闭症谱系障碍（ASD）患者干预措施中的反应的适当定义。按照谢菲尔德启发框架（SHELF）进行了启发过程。五位 ASD 领域的专家和两位专家知识启发参加了为期一天的启发研讨会。 ASD 专家之前接受过 SHELF 启发过程的培训，并收到了一份关于该主题的科学证据档案。反应定义设定为所有患者、功能水平、年龄组和临床医生平均的临床相关改善的平均值。基于科学证据和专家判断，同意使用正态概率分布来表示这种反应的知识状态，预期值为 4.03，标准差为 0.664。考虑到估计的剩余不确定性和可用的文献，CARS-2 改善 4.5 点已被定义为干预后得出反应的阈值。CARS-2 改善 4.5 点可用于评估干预措施对个体的意义。这一初步发现代表了一个重要的新基准，可能有助于决策者评估 ASD 中干预措施的疗效。
