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，其定义为缺乏社会互动，语言和沟通以及游戏。自闭症儿童可能会表现出一些行为，包括严重的发脾气，不服从，破坏性和自我伤害。他们可能需要较少的睡眠，并在夜间频繁醒来。治疗ASD症状的成功干预措施可以改善ASD儿童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，但成本高昂，成本效益相当不确定。缺乏衡量自闭症儿童治疗干预措施成本效益的研究。该提案的主要目标是调查测量质量调整生命年（QQLS）的方法，用于治疗ASD儿童的干预措施的成本效益分析。特别是，我们感兴趣的是，是否通用的工具，描述由公共卫生服务所提出的QURTS可以捕捉到这些影响。我们的主要假设是，通用工具，用于测量QASTS将与ASD儿童的ASD相关症状的变化敏感。我们还研究了是否可以用通用的QALY工具来测量家庭效应。未能将家庭QOLS纳入ASD儿童治疗干预措施的成本效益分析可能会使成本效益比产生偏差。我们计划使用参与自闭症治疗网络两个站点的ASD儿童样本来测试我们的假设。该提案的调查人员制定了一项研究议程，以量化与儿童残疾有关的儿童和照顾者健康。我们寻求通过以下方式推进这一研究议程：i）评估替代通用工具对偏好加权健康结果的敏感性（ii）确定是否应将照顾者的QOHSS纳入治疗ASD儿童的干预措施的成本效益评估中;和iii）使用定性和定量方法评估用于测量ASD儿童QALTS的通用工具的心理测量学特性。该研究的结果将改进方法，以准确评估治疗ASD儿童的干预措施的成本效益，并协助将实验结果转化为基于证据的决策政策。 公共卫生相关性：自闭症谱系障碍（ASD）的特征是社交技能，沟通，认知和行为功能的障碍。因此，对自闭症儿童的成功干预不仅有可能影响儿童的结果，而且可能包括对家庭的实质性健康益处。评价服务成本效益的准则建议使用所获得的每质量调整生命年的成本。到目前为止，还没有研究试图评估测量ASD儿童QSHS的仪器是否对这种情况敏感。此外，还需要进行研究，以便在成本效益分析中考虑到家庭效应。尽管有可能为自闭症儿童的治疗提供信息资源分配，但缺乏测量这一人群中QASTS的方法和数据。

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.