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Quality of Life Research
Published in: Quality of Life Research 2/2015

Open Access 01-02-2015

Developing a utility index for the Aberrant Behavior Checklist (ABC-C) for fragile X syndrome

Authors: Cicely Kerr, Katie Breheny, Andrew Lloyd, John Brazier, Donald B. Bailey Jr, Elizabeth Berry-Kravis, Jonathan Cohen, Jennifer Petrillo

Published in: Quality of Life Research | Issue 2/2015

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Abstract

Purpose

This study aimed to develop a utility index (the ABC-UI) from the Aberrant Behavior Checklist-Community (ABC-C), for use in quantifying the benefit of emerging treatments for fragile X syndrome (FXS).

Methods

The ABC-C is a proxy-completed assessment of behaviour and is a widely used measure in FXS. A subset of ABC-C items across seven dimensions was identified to include in health state descriptions. This item reduction process was based on item performance, factor analysis and Rasch analysis performed on an observational study dataset, and consultation with five clinical experts and a methodological expert. Dimensions were combined into health states using an orthogonal design and valued using time trade-off (TTO), with lead-time TTO methods used where TTO indicated a state valued as worse than dead. Preference weights were estimated using mean, individual level, ordinary least squares and random-effects maximum likelihood estimation [RE (MLE)] regression models.

Results

A representative sample of the UK general public (n = 349; mean age 35.8 years, 58.2 % female) each valued 12 health states. Mean observed values ranged from 0.92 to 0.16 for best to worst health states. The RE (MLE) model performed best based on number of significant coefficients and mean absolute error of 0.018. Mean utilities predicted by the model covered a similar range to that observed.

Conclusions

The ABC-UI estimates a wide range of utilities from patient-level FXS ABC-C data, allowing estimation of FXS health-related quality of life impact for economic evaluation from an established FXS clinical trial instrument.
Literature
1.
Hessl, D., Dyer-Friedman, J., Glaser, B., Wisbeck, J., Barajas, R. G., Taylor, A., et al. (2001). The influence of environmental and genetic factors on behavior problems and autistic symptoms in boys and girls with fragile X syndrome. Pediatrics, 108(5), e88.PubMedCrossRef
2.
Crawford, D. C., Acuña, J. M., & Sherman, S. L. (2001). FMR1 and the fragile X syndrome: Human genome epidemiology review. Genetics in Medicine, 3(5), 359–371.PubMedCrossRef
3.
Hagerman, R. J., Berry-Kravis, E., Kaufmann, W. E., Ono, M. Y., Tartaglia, N., Lachiewicz, A., et al. (2009). Advances in the treatment of fragile X syndrome. Pediatrics, 123(1), 378–390.PubMedCentralPubMedCrossRef
4.
Finestack, L. H., Richmond, E. K., & Abbeduto, L. (2009). Language development in individuals with fragile X syndrome. Topics in Language Disorders, 29(2), 133.PubMedCentralPubMedCrossRef
5.
Cordeiro, L., Ballinger, E., Hagerman, R., & Hessl, D. (2011). Clinical assessment of DSM-IV anxiety disorders in fragile X syndrome: Prevalence and characterization. Journal of Neurodevelopmental Disorders, 3(1), 57–67.PubMedCentralPubMedCrossRef
6.
Kaufmann, W. E., Cortell, R., Kau, A. S. M., Bukelis, I., Tierney, E., Gray, R. M., et al. (2004). Autism spectrum disorder in fragile X syndrome: communication, social interaction, and specific behaviors. American Journal of Medical Genetics Part A, 129(3), 225–234.CrossRef
7.
Bailey, D. B, Jr, Raspa, M., Bishop, E., Mitra, D., Martin, S., Wheeler, A., et al. (2012). Health and economic consequences of fragile X syndrome for caregivers. Journal of Developmental and Behavioral Pediatrics, 33(9), 705–712.PubMedCrossRef
8.
Hartley, S. L., Seltzer, M. M., Raspa, M., Olmstead, M., Bishop, E., & Bailey, D. B, Jr. (2011). Exploring the adult life of men and women with fragile X syndrome: Results from a national survey. American Journal of Intellectual and Developmental Disabilities, 116(1), 16–35.CrossRef
9.
Gross, C., Berry-Kravis, E. M., & Bassell, G. J. (2012). Therapeutic strategies in fragile X syndrome: Dysregulated mGluR signaling and beyond. Neuropsychopharmacology, 37(1), 178–195.PubMedCentralPubMedCrossRef
10.
Wijetunge, L. S., Chattarji, S., Wyllie, D. J., & Kind, P. C. (2013). Fragile X Syndrome: From targets to treatments. Neuropharmacology, 68, 83–96.PubMedCrossRef
11.
Aman, M. G., Singh, N. N., Stewart, A. W., & Field, C. J. (1985). The Aberrant Behavior Checklist: A behavior rating scale for the assessment of treatment effects. American Journal of Mental Deficiency, 89(5), 485–491.PubMed
12.
Erickson, C. A., Stigler, K. A., Wink, L. K., Mullett, J. E., Kohn, A., Posey, D. J., et al. (2011). A prospective open-label study of aripiprazole in fragile X syndrome. Psychopharmacology, 216(1), 85–90.PubMedCrossRef
13.
Berry-Kravis, E., Sumis, A., Hervey, C., Nelson, M., Porges, S. W., Weng, N., et al. (2008). Open-label treatment trial of lithium to target the underlying defect in fragile X syndrome. Journal of Developmental and Behavioral Pediatrics, 29(4), 293–302.PubMedCrossRef
14.
Jacquemont, S., Curie, A., des Portes, V., Torrioli, M. G., Berry-Kravis, E., Hagerman, R. J., et al. (2011). Epigenetic modification of the FMR1 gene in fragile X syndrome is associated with differential response to the mGluR5 antagonist AFQ056. Science Translational Medicine, 3, 64ra1.PubMedCrossRef
15.
Paribello, C., Tao, L., Folino, A., Berry-Kravis, E., Tranfaglia, M., Ethell, I., et al. (2010). Open-label add-on treatment trial of minocycline in fragile X syndrome. BMC Neurology, 10(1), 91.PubMedCentralPubMedCrossRef
16.
Sansone, S.M., Widaman, K.F., Hall, S.S., Reiss, A.L., Lightbody, A., Kaufmann, W.E., Berry-Kravis, E., Lachiewicz, A., Brown, E.C., Hessl, D. (2011). Psychometric study of the Aberrant Behavior Checklist in fragile X syndrome and implications for targeted treatment. Journal of Autism and Developmental Disorders, 1–16.
17.
18.
Drummond, M., Jönsson, B., & Rutten, F. (1997). The role of economic evaluation in the pricing and reimbursement of medicines. Health Policy, 40(3), 199–215.PubMedCrossRef
19.
Gold, M. (1996). Panel on cost-effectiveness in health and medicine. Medical care, 34 (12).
20.
21.
Dolan, P. (1996). Modelling valuations for health states: The effect of duration. Health Policy, 38(3), 189–203.PubMedCrossRef
22.
Brazier, J., Roberts, J., & Deverill, M. (2002). The estimation of a preference-based measure of health from the SF-36. Journal of health economics, 21(2), 271–292.PubMedCrossRef
23.
Feeny, D., Furlong, W., Boyle, M., & Torrance, G. W. (1995). Multi-attribute health status classification systems. Health Utilities Index. Pharmacoeconomics, 7(6), 490.PubMedCrossRef
24.
Tilford, J. M., Payakachat, N., Kovacs, E., Pyne, J. M., Brouwer, W., Nick, T. G., et al. (2012). Preference-based health-related quality-of-life outcomes in children with autism spectrum disorders: A comparison of generic instruments. Pharmacoeconomics, 30(8), 661–679.PubMedCentralPubMedCrossRef
25.
26.
Papaioannou, D., Brazier, J., & Parry, G. (2011). How valid and responsive are generic health status measures, such as EQ-5D and SF-36, in schizophrenia? A systematic review. Value in Health, 14(6), 907–920.PubMedCentralPubMedCrossRef
27.
Tosh, J., Brazier, J., Evans, P., & Longworth, L. (2012). A review of generic preference-based measures of health-related quality of life in visual disorders. Value in Health, 15(1), 118–127.PubMedCentralPubMedCrossRef
28.
Brazier, J. E., Rowen, D., Mavranezouli, I., Tsuchiya, A., Young, T., Yang, Y., et al. (2012). Developing and testing methods for deriving preference-based measures of health from condition specific measures (and other patient based measures of outcome). Health Technology Assessment, 16(32), 1366–5278.CrossRef
29.
Young, T. A., Yang, Y., Brazier, J. E., & Tsuchiya, A. (2011). The use of Rasch analysis in reducing a large condition-specific instrument for preference valuation the case of moving from AQLQ to AQL-5D. Medical Decision Making, 31(1), 195–210.PubMedCrossRef
30.
Wheeler, A., Raspa, M., Bann, C., Bishop, E., Hessl, D., Sacco, P., et al. (2014). Anxiety, attention problems, hyperactivity, and the Aberrant Behavior Checklist in fragile X syndrome. American Journal of Medical Genetics, Part A, 164(1), 141–155.CrossRef
31.
Esbensen, A. J., Rojahn, J., Aman, M. G., & Ruedrich, S. (2003). Reliability and validity of an assessment instrument for anxiety, depression, and mood among individuals with mental retardation. Journal of Autism and Developmental Disorders, 33(6), 617–629.PubMedCrossRef
32.
Gadow, K. D., & Sprafkin, J. (2010). Child and adolescent symptom inventory-4R. Stony Brook, NY: Checkmate Plus.
33.
Gadow, K. D., Sprafkin, J., & Weiss, M. (2004). Adult inventory-4. Stony Brook, NY: Checkmate Plus.
34.
35.
36.
37.
Rowen, D., Brazier, J., Young, T., Gaugris, S., Craig, B. M., King, M. T., et al. (2011). Deriving a preference-based measure for cancer using the EORTC QLQ-C30. Value in Health, 14, 721–731.PubMedCrossRef
38.
Yang, Y., Brazier, J. E., Tsuchiya, A., & Young, T. A. (2011). Estimating a preference-based index for a 5-dimensional health state classification for asthma derived from the asthma quality of life questionnaire. Medical Decision Making, 31, 281–291.PubMedCrossRef
39.
Sinha, Y., Silove, N., Hayen, A., Williams, K. (2011) Auditory integration training and other sound therapies for autism spectrum disorders [ASD]. Cochrane Database of Systematic Reviews, 12.
40.
Aman, M. G. (2012). Aberrant Behavior Checklist: Current identity and future developments. Clinical & Experimental Pharmacology, 2, e114.CrossRef
41.
Wille, N., Badia, X., Bonsel, G., Burström, K., Cavrini, G., Devlin, N., et al. (2010). Development of the EQ-5D-Y: A child-friendly version of the EQ-5D. Quality of Life Research, 19(6), 875–886.PubMedCentralPubMedCrossRef
42.
Coucill, W., Bryan, S., Bentham, P., Buckley, A., & Laight, A. (2001). EQ-5D in patients with dementia an investigation of inter-rater agreement. Medical Care, 39(8), 760–771.PubMedCrossRef
43.
Torrance, G. W., Thomas, W. H., & Sackett, D. L. (1972). A utility maximization model for evaluation of health care programs. Health Services Research, 7(2), 118.PubMedCentralPubMed
44.
Devlin, N. J., Tsuchiya, A., Buckingham, K., & Tilling, C. (2011). A uniform time trade off method for states better and worse than dead: Feasibility study of the lead time approach. Health Economics, 20(3), 348–361.PubMedCrossRef
45.
Ratcliffe, J., Stevens, K., Flynn, T., Brazier, J., & Sawyer, M. (2011). Whose values in health? An empirical comparison of the application of adolescent and adult values for the CHU-9D and AQOL-6D in the Australian adolescent general population. Value in Health, 15(5), 730–736.CrossRef
Metadata
Title
Developing a utility index for the Aberrant Behavior Checklist (ABC-C) for fragile X syndrome
Authors
Cicely Kerr
Katie Breheny
Andrew Lloyd
John Brazier
Donald B. Bailey Jr
Elizabeth Berry-Kravis
Jonathan Cohen
Jennifer Petrillo
Publication date
01-02-2015
Publisher
Springer International Publishing
Published in
Quality of Life Research / Issue 2/2015
Print ISSN: 0962-9343
Electronic ISSN: 1573-2649
DOI
https://doi.org/10.1007/s11136-014-0759-8

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