Measuring quality adjusted life years in children with autism spectrum disorders

描述(由申请者提供)：自闭症是一种行为状况，定义为缺乏社交、语言和交流以及游戏。自闭症儿童会表现出许多行为，包括严重发脾气、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。对自闭症症状的成功干预可以改善自闭症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，其成本效益存在相当大的不确定性。缺乏衡量治疗自闭症儿童干预措施的成本效益的研究。这项建议的主要目标是研究衡量治疗自闭症儿童干预措施的质量调整寿命年(QALY)的方法。我们尤其感兴趣的是，公共卫生服务建议的用于描述QALY的通用工具是否能够反映这些影响。我们的主要假设是，用于测量QALY的通用工具将对ASD儿童中ASD相关症状的变化敏感。我们还检查了家庭效应是否可以用通用的QALY工具来衡量。没有将家庭QALY纳入治疗自闭症儿童的干预措施的成本-效果分析，可能会使成本-效果比产生偏差。我们计划使用参加自闭症治疗网络两个站点的自闭症儿童样本来验证我们的假设。这项提案的调查人员已经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。我们试图通过以下方式推动这项研究议程：i)评估用于衡量自闭症儿童的偏爱加权健康结果(测量QALY)的替代通用工具的敏感性；ii)确定是否应将照顾者QALY纳入治疗自闭症儿童的干预措施的成本效益评估；以及iii)使用定性和定量方法评估用于测量患有自闭症儿童的QALY的通用工具的心理测量学特性。这项研究的结果将改进准确评估治疗自闭症儿童的干预措施的成本效益的方法，并有助于将实验结果转化为决策的循证政策。 公共卫生相关性：自闭症谱系障碍(ASD)的特征是社交技能、沟通以及认知和行为功能的损害。因此，对患有自闭症儿童的成功干预不仅有可能影响儿童的结局，而且可能会给家庭带来实质性的健康益处。评估服务成本效益的指南建议使用每质量调整寿命年(QALY)的成本。到目前为止，还没有研究试图评估测量患有自闭症儿童的QALY的工具是否对这种情况敏感。此外，还需要进行研究，在成本效益分析中考虑家庭影响。尽管有可能为自闭症儿童的治疗提供资源分配，但在这一人群中缺乏衡量QALY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.