缺乏共识措施来监测自闭症谱系障碍(ASD)的核心变化或对干预措施的反应,导致难以证明对ASD核心症状的干预功效。没有开发用于测量核心症状变化的普遍接受的结果度量。然而,CARS(儿童自闭症评定量表)是EMA指南中关于ASD治疗药物临床开发的推荐结果之一.不幸的是,目前,ASD患者对CARS的反应定义尚未达成共识。此启发过程的目的是确定CARS2量表对自闭症谱系障碍(ASD)患者干预的反应的适当定义。遵循谢菲尔德启发框架(SHELF)进行了启发过程。ASD领域的五名专家和两名专业知识启发专家参加了为期1天的启发研讨会。ASD专家以前曾接受过SHELF启发过程的培训,并收到了有关该主题的科学证据档案。反应定义设定为所有患者的平均临床相关改善。功能水平,年龄组和临床医生。根据科学证据和专家判断,一个正态的概率分布被认为是表示知识状态的这种反应的期望值4.03和标准偏差0.664。考虑到估计的剩余不确定性和现有文献,CARS-2改善4.5分被定义为干预后判断缓解的阈值.CARS-2提高4.5分,可用于评估个体干预措施的意义。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干预措施的有效性。
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.