caa a22 4500 606180419 CHVBK 20210128100806.0 cr unu---uuuuu 210128e20150201xx s 000 0 eng 10.1007/s11136-014-0759-8 doi (NATIONALLICENCE)springer-10.1007/s11136-014-0759-8 Developing a utility index for the Aberrant Behavior Checklist (ABC-C) for fragile X syndrome [Elektronische Daten] [Cicely Kerr, Katie Breheny, Andrew Lloyd, John Brazier, Donald Bailey Jr, Elizabeth Berry-Kravis, Jonathan Cohen, Jennifer Petrillo] 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 35.8years, 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. The Author(s), 2014 Fragile X syndrome nationallicence Aberrant Behavior Checklist nationallicence TTO nationallicence Lead-time nationallicence Utility index nationallicence Kerr Cicely ICON Patient Reported Outcomes, Seacourt Tower, West Way, OX2 0JJ, Oxford, UK aut Breheny Katie ICON Patient Reported Outcomes, Seacourt Tower, West Way, OX2 0JJ, Oxford, UK aut Lloyd Andrew ICON Patient Reported Outcomes, Seacourt Tower, West Way, OX2 0JJ, Oxford, UK aut Brazier John School of Health and Related Research (ScHARR), The University of Sheffield, Sheffield, UK aut Bailey Jr Donald RTI International, Research Triangle Park, NC, USA aut Berry-Kravis Elizabeth Departments of Pediatrics, Neurological Sciences, and Biochemistry, Rush University Medical Center, Chicago, IL, USA aut Cohen Jonathan Fragile X Alliance Inc, Melbourne, Australia aut Petrillo Jennifer Novartis AG, Basel, Switzerland aut Quality of Life Research Springer International Publishing 24/2(2015-02-01), 305-314 0962-9343 24:2<305 2015 24 11136 https://doi.org/10.1007/s11136-014-0759-8 text/html Onlinezugriff via DOI BK010053 XK010053 XK010000 Metadata rights reserved Springer special CC-BY-NC licence nationallicence 1 research-article jats NATIONALLICENCE NATIONALLICENCE NL-springer NATIONALLICENCE 856 40 https://doi.org/10.1007/s11136-014-0759-8 text/html Onlinezugriff via DOI NATIONALLICENCE 700 1- Kerr Cicely ICON Patient Reported Outcomes, Seacourt Tower, West Way, OX2 0JJ, Oxford, UK aut NATIONALLICENCE 700 1- Breheny Katie ICON Patient Reported Outcomes, Seacourt Tower, West Way, OX2 0JJ, Oxford, UK aut NATIONALLICENCE 700 1- Lloyd Andrew ICON Patient Reported Outcomes, Seacourt Tower, West Way, OX2 0JJ, Oxford, UK aut NATIONALLICENCE 700 1- Brazier John School of Health and Related Research (ScHARR), The University of Sheffield, Sheffield, UK aut NATIONALLICENCE 700 1- Bailey Jr Donald RTI International, Research Triangle Park, NC, USA aut NATIONALLICENCE 700 1- Berry-Kravis Elizabeth Departments of Pediatrics, Neurological Sciences, and Biochemistry, Rush University Medical Center, Chicago, IL, USA aut NATIONALLICENCE 700 1- Cohen Jonathan Fragile X Alliance Inc, Melbourne, Australia aut NATIONALLICENCE 700 1- Petrillo Jennifer Novartis AG, Basel, Switzerland aut NATIONALLICENCE 773 0- Quality of Life Research Springer International Publishing 24/2(2015-02-01), 305-314 0962-9343 24:2<305 2015 24 11136