, Volume 30, Issue 8, pp 661–679 | Cite as

Preference-Based Health-Related Quality-of-Life Outcomes in Children with Autism Spectrum Disorders

A Comparison of Generic Instruments
  • J. Mick Tilford
  • Nalin Payakachat
  • Erica Kovacs
  • Jeffrey M. Pyne
  • Werner Brouwer
  • Todd G. Nick
  • Jayne Bellando
  • Karen A. Kuhlthau
Original Research Article



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 QALYs in children are under-studied. No cost-effectiveness analyses have been undertaken in this population using the cost-per-QALY metric.


This study describes health-related quality-of-life (HR-QOL) 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 were correlated with retrospectively assessed clinical information. Subjects were recruited from two sites of the Autism Treatment Network (ATN) in the US: a developmental centre in Little Rock, Arkansas, and an outpatient psychiatric clinic at Columbia University Medical Center in New York. Children that met Diagnostic and Statistical Manual of Mental Disorders, Fourth Edition (DSM-IV) criteria for an ASD by a multidisciplinary 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 150 completing surveys. HR-QOL outcomes were described using the Health Utilities Index (HUI) 3 and the Quality of Well-Being Self-Administered (QWB-SA) scale obtained by proxy via the family caregiver.


Children were diagnosed as having autistic disorder (76%), pervasive developmental disorder-not otherwise specified [PDD-NOS] (15%), and Asperger’s disorder (9%). Average HUI3 and QWB-SA scores were 0.68 (SD 0.21, range 0.07–1) and 0.59 (SD 0.16, range 0.18–1), respectively. The HUI3 score was significantly correlated with clinical variables including adaptive behaviour (ρ=0.52;p<0.001) and cognitive functioning (ρ=0.36;p<0.001). The QWB-SA score had weak correlation with adaptive behaviour (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 points (95% CI 0.14, 0.29) across the first to the third quartile of the cognitive functioning measure compared with 0.05 (95% CI −0.01, 0.11) for the QWB-SA. Adjusted R2 values also were higher for the HUI3 compared with the QWB-SA across all clinical measures.


The HUI3 was more sensitive to clinical measures used to characterize children with autism compared with the QWB-SA score. The findings provide a benchmark to compare scores obtained by alternative methods and instruments. Researchers should consider incorporating the HUI3 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.



The project was supported by a grant (no. R01MH089466) from the National Institute of Mental Health with JMT and KAK serving as principal investigators. The content of this article is solely the responsibility of the authors and does not necessarily represent the official views of the National Institute of Mental Health or the National Institutes of Health. The authors acknowledge the members of the ATN for use of the data. The data for the study were collected as part of the ATN, a programme of Autism Speaks. Further support came from a cooperative agreement (UA3 MC 11054) from the U.S. Department of Health and Human Services, Health Resources and Services Administration, Maternal and Child Health Research Program, to the Massachusetts General Hospital. The work described in this article represents the independent efforts of the authors with no restrictions from the funding source or the ATN. None of the authors of this study reported a conflict of interest associated with the preparation of the manuscript. Maria Melguizo, Nupur Chowdhury, Rebecca Rieger and Latunja Sockwell provided excellent research assistance.

JMT, NP, JMP, and KAK conceptualized the original study with input from WB, TGN, JB and EK. JMT was the principal author of the manuscript with input from WB, NP, TGN, JMP and the co-authors. Data collection was the work of JMT, NP, JB and EK. TGN, JMT and NP led the data analysis. JMT serves as the guarantor for the overall content of the paper.

This paper is part of a theme issue co-edited by Lisa Prosser, University of Michigan, USA, and no external funding was used to support the publication of this theme issue.


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Copyright information

© Springer International Publishing AG 2012

Authors and Affiliations

  • J. Mick Tilford
    • 1
    • 2
    • 3
  • Nalin Payakachat
    • 2
  • Erica Kovacs
    • 4
  • Jeffrey M. Pyne
    • 5
    • 6
  • Werner Brouwer
    • 7
  • Todd G. Nick
    • 3
  • Jayne Bellando
    • 3
  • Karen A. Kuhlthau
    • 8
    • 9
  1. 1.Department of Health Policy and ManagementUniversity of Arkansas for Medical SciencesLittle RockUSA
  2. 2.Department of Pharmacy PracticeUniversity of Arkansas for Medical SciencesLittle RockUSA
  3. 3.Department of PediatricsUniversity of Arkansas for Medical SciencesLittle RockUSA
  4. 4.Department of PsychiatryColumbia University Medical CenterNew YorkUSA
  5. 5.Center for Mental Healthcare and Outcomes ResearchCentral Arkansas Veterans Healthcare SystemLittle RockUSA
  6. 6.Psychiatric Research InstituteUniversity of Arkansas for Medical SciencesLittle RockUSA
  7. 7.Department of Health Policy and ManagementErasmus UniversityRotterdamThe Netherlands
  8. 8.Department of PediatricsHarvard Medical SchoolBostonUSA
  9. 9.Center for Adolescent Health PolicyMassachusetts General HospitalBostonUSA

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