Preference-based health-related quality-of-life outcomes in children with autism spectrum disorders: a comparison of generic instruments.
Preference-based health-related quality-of-life outcomes in children with autism spectrum disorders: a comparison of generic instruments.
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DOI：
10.2165/11597200-000000000-00000
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发表时间：
2012-08-01
影响因子：
4.4
通讯作者：
Kuhlthau KA
中科院分区：
文献类型：
--
作者：
Tilford JM;Payakachat N;Kovacs E;Pyne JM;Brouwer W;Nick TG;Bellando J;Kuhlthau KA
对自闭症谱系障碍儿童的药物和其他治疗方法进行成本效益分析，有可能通过向公共和私人支付者展示治疗的价值来改善获得服务的机会，但对儿童质量调整生命年（QALYs）的测量方法研究不足。没有使用每个QALY指标的成本对这一人群进行成本效益分析。本研究描述了asd儿童健康相关生活质量（HRQoL）结果，并比较了两种基于偏好的通用工具相对于asd相关条件和症状的敏感性。研究设计是横断面的，前瞻性收集的结果数据与回顾性评估的临床信息相关。研究对象是从自闭症治疗网络（ATN）的两个地点招募的：阿肯色州小石城的一个发育中心和纽约州哥伦比亚大学医学中心的一个精神病门诊诊所。通过多学科小组评估符合DSM-IV标准的ASD儿童被要求参加临床登记。作为ATN的一部分，同意联系自闭症儿童的家庭参与未来的研究，形成了研究的抽样框架。如果患有自闭症谱系障碍的孩子年龄在4到17岁之间，并且家庭照顾者会说英语，那么这个家庭也被包括在内。通过邮件联系符合条件的家庭，看他们是否有兴趣参与这项研究，N=150完成调查。HRQoL结果使用健康效用指数标记III （HUI-3）和自我管理的幸福质量量表（QWB-SA）进行描述，该量表由家庭照顾者代理获得。儿童被诊断患有自闭症（76%）、广泛性发育障碍（PDD-NOS）（15%）和阿斯伯格障碍（9%）。平均HUI-3和QWB-SA评分分别为0.68 （SD=0.21，范围0.07 ~ 1）和0.59 （SD=0.16，范围0.18 ~ 1）。HUI-3评分与适应行为（ρ=0.52; p<0.001）和认知功能（ρ=0.36; p<0.001）等临床变量显著相关。QWB-SA评分与适应行为（ρ=0.25; p<0.001）和认知功能（ρ=0.17; p<0.005）呈弱相关。在所有临床测量中，HUI3的变化评分都大于QWB-SA。在认知功能测试的第一到第三个四分位数中，HUI3的得分增加了0.21分（95% CI: 0.14-0.29），而QWB-SA的得分为0.05分（95% CI: - 0.01-0.11）。在所有临床测量中，与QWB-SA相比，HUI3的校正R2也更高。与QWB-SA评分相比，HUI-3对用于表征自闭症儿童的临床指标更为敏感。研究结果为比较其他方法和工具获得的分数提供了一个基准。研究人员应考虑将HUI-3纳入临床试验和其他纵向研究，以建立证据基础，描述向这一重要人群提供服务的成本效益。
Cost-effectiveness analysis of pharmaceutical and other treatments for children with autism spectrum disorders (ASDs) has the potential to improve access to services by demonstrating the value of treatment to public and private payers, but methods for measuring quality adjusted life years (QALYs) in children are understudied. No cost-effectiveness analyses have been undertaken in this population using the cost per QALY metric. This study describes health-related quality of life (HRQoL) outcomes in children with ASDs and compares the sensitivity of two generic preference-based instruments relative to ASD-related conditions and symptoms. The study design was cross-sectional with prospectively collected outcome data that was correlated with retrospectively assessed clinical information. Subjects were recruited from two sites of the Autism Treatment Network (ATN): a developmental center in Little Rock, Arkansas, and an outpatient psychiatric clinic at Columbia University Medical Center in New York, NY. Children that met DSM-IV criteria for an ASD by a multi-disciplinary team evaluation were asked to participate in a clinical registry. Families of children with an ASD that agreed to be contacted about participation in future research studies as part of the ATN formed the sampling frame for the study. Families were included if the child with the ASD was between 4 and 17 years of age and the family caregiver spoke English. Eligible families were contacted by mail to see if they would be interested in participating in the study with N=150 completing surveys. HRQoL outcomes were described using the Health Utilities Index Mark III (HUI-3) and the self-administered Quality of Well-Being scale (QWB-SA) obtained by proxy via the family caregiver. Children were diagnosed as having autistic disorder (76%), pervasive developmental disorder (PDD-NOS) (15%), and Asperger's disorder (9%). Average HUI-3 and QWB-SA scores were 0.68 (SD=0.21, range of 0.07 to 1) and 0.59 (SD=0.16, range of 0.18 to 1) respectively. The HUI-3 score was significantly correlated with clinical variables including adaptive behavior (ρ=0.52; p<0.001) and cognitive functioning (ρ=0.36; p<0.001). The QWB-SA score had weak correlation with adaptive behavior (ρ=0.25; p<0.001) and cognitive functioning (ρ=0.17; p<0.005). Change scores for the HUI3 were larger than the QWB-SA for all clinical measures. Scores for the HUI3 increased 0.21 (95% CI: 0.14–0.29) points across the first to third quartile of the cognitive functioning measure compared to 0.05 (95% CI: −0.01–0.11) for the QWB-SA. Adjusted R2's also were higher for the HUI3 compared to the QWB-SA across all clinical measures. The HUI-3 was more sensitive to clinical measures used to characterize children with autism compared to the QWB-SA score. The findings provide a benchmark to compare scores obtained by alternative methods and instruments. Researchers should consider incorporating the HUI-3 in clinical trials and other longitudinal research studies to build the evidence base for describing the cost-effectiveness of services provided to this important population.
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影响因子：
3.9
作者：
Gotham, Katherine;Pickles, Andrew;Lord, Catherine
通讯作者：
Lord, Catherine
影响因子：
3
作者：
KAPLAN, RM;ANDERSON, JP;ORENSTEIN, D
通讯作者：
ORENSTEIN, D
影响因子：
--
作者：
Buysse, Corinne M. P.;Raat, Hein;Joosten, Koen F. M.
通讯作者：
Joosten, Koen F. M.
影响因子：
3
作者：
Feeny, D;Furlong, W;Boyle, M
通讯作者：
Boyle, M
影响因子：
5.4
作者：
Marra, CA;Woolcott, JC;Anis, AH
通讯作者：
Anis, AH