Measuring quality adjusted life years in children with autism spectrum disorders

描述（由申请人提供）：自闭症是一种行为状况，其特征是缺乏社会互动、语言和沟通以及游戏。自闭症儿童可能表现出一系列行为，包括严重的发脾气、不服从、破坏性和自残。他们可能需要较少的睡眠，并在夜间频繁醒来。成功的干预治疗ASD症状可以改善ASD患儿及其家庭成员的生活质量。早期行为干预最有可能改善自闭症儿童的症状，但成本昂贵，成本效益存在相当大的不确定性。目前还缺乏衡量自闭症儿童治疗干预措施成本效益的研究。本提案的主要目的是研究测量质量调整生命年（QALYS）的方法，用于分析治疗自闭症儿童的干预措施的成本效益。我们特别感兴趣的是，公共卫生服务处提出的用于描述QALYS的通用工具是否能捕捉到这些影响。我们的主要假设是，用于测量QALYS的通用仪器将对asd患儿中asd相关症状的变化敏感。我们还检验了家庭效应是否可以用通用的QALY仪器来测量。未能将家庭质量分析纳入治疗自闭症儿童的干预措施的成本-效果分析可能会影响成本-效果比。我们计划用参与自闭症治疗网络两个站点的自闭症儿童样本来检验我们的假设。该提案的调查人员制定了一项研究议程，以量化儿童和照顾者的健康与儿童残疾的关系。我们寻求通过以下方式进一步推进这一研究议程：1)评估自闭症儿童偏好加权健康结果（测量QALYS）的替代通用工具的敏感性；ii)确定是否应将护理者质量评估纳入asd儿童治疗干预措施的成本效益评估；iii)使用定性和定量方法评估用于测量asd儿童QALYS的通用工具的心理测量特性。这项研究的结果将改进方法，以准确评估治疗自闭症儿童的干预措施的成本效益，并有助于将实验结果转化为基于证据的决策政策。

DESCRIPTION (provided by applicant): Autism is a behavioral condition defined by deficient social interaction, language and communication, and play. Children with autism can exhibit a number of behaviors including severe tantrums, noncompliance, destructiveness, and self-injury. They may require less sleep and have frequent awakenings during the night. Successful interventions to treat the symptoms of ASD can improve the quality of life of children with ASDs as well as their family members. Early behavioral interventions have the most potential to improve symptoms in children with ASDs, yet are costly with considerable uncertainty regarding their cost-effectiveness. Research to measure the cost-effectiveness of interventions for the treatment of children with ASDs is lacking. The primary goal of this proposal is to investigate methods for measuring quality adjusted life years (QALYS) for cost-effectiveness analysis of interventions to treat children with ASDs. In particular, we are interested in whether generic instruments for describing QALYS as proposed by the Public Health Service can capture these effects. Our primary hypothesis is that generic instruments for measuring QALYS will be sensitive to variations in ASD-related symptoms among children with ASDs. We also examine whether family effects can be measured with generic QALY instruments. Failure to include family QALYS in cost-effectiveness analyses of interventions to treat children with ASDs can bias cost-effectiveness ratios. We plan to test our hypotheses using a sample of children with ASDs participating in two sites of the Autism Treatment Network. The investigators for this proposal have developed a research agenda to quantify child and caregiver health in relation to child disabilities. We seek to further this research agenda by i) Evaluating the sensitivity of alternative generic instruments for preference-weighting health outcomes (measuring QALYS) in children with ASDs; ii) Determining whether caregiver QALYS should be incorporated into cost-effectiveness evaluations of interventions to treat children with ASDs; and iii) Evaluating the psychometric properties of generic instruments for measuring QALYS in children with ASDs using qualitative and quantitative methods. Findings from the study will improve methods to accurately assess the cost-effectiveness of interventions to treat children with ASDs and assist in the translation of experimental findings into evidence-based policy for decision making. PUBLIC HEALTH RELEVANCE: Autism spectrum disorders (ASDs) are characterized by impairments in social skills, communication, and cognitive and behavioral functioning. Successful interventions for children with ASDs thus have the potential to not only affect outcomes of the child, but may include substantial health benefits for the family. Guidelines for evaluating the cost-effectiveness of services recommend using the cost per quality adjusted life year (QALY) gained. To date, no studies have attempted to evaluate whether instruments to measure QALYS in children with ASDs are sensitive to the condition. In addition, research is needed to account for family effects in cost- effectiveness analysis. Despite the potential for informing resource allocation to treatment of children with ASDs, methods and data for measuring QALYS in this population are lacking.