Development of the multi-attribute Adolescent Health Utility Measure (AHUM)
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RESEARCH
Open Access
Development of the multi-attribute Adolescent Health Utility Measure (AHUM) Kathleen M Beusterien1*, Jean-Ezra Yeung1, Francis Pang2 and John Brazier3
Abstract Objective: Obtain utilities (preferences) for a generalizable set of health states experienced by older children and adolescents who receive therapy for chronic health conditions. Methods: A health state classification system, the Adolescent Health Utility Measure (AHUM), was developed based on generic health status measures and input from children with Hunter syndrome and their caregivers. The AHUM contains six dimensions with 4–7 severity levels: self-care, pain, mobility, strenuous activities, self-image, and health perceptions. Using the time trade off (TTO) approach, a UK population sample provided utilities for 62 of 16,800 AHUM states. A mixed effects model was used to estimate utilities for the AHUM states. The AHUM was applied to trial NCT00069641 of idursulfase for Hunter syndrome and its extension (NCT00630747). Results: Observations (i.e., utilities) totaled 3,744 (12*312 participants), with between 43 to 60 for each health state except for the best and worst states which had 312 observations. The mean utilities for the best and worst AHUM states were 0.99 and 0.41, respectively. The random effects model was statistically significant (p < 0.0001; adjusted R2 = 0.361; RMSE = 0.194). When AHUM utilities were applied to the idursulfase trial, mean utilities in the idursulfase weekly and placebo groups improved +0.087 and +0.006, respectively, from baseline to week 53. In the extension, when all patients received idursulfase, the utilities in the treatment group remained stable and the placebo group improved +0.039. Discussion: The AHUM health state classification system may be used in future research to enable calculation of quality-adjust life expectancy for applicable health conditions.
Introduction An important innovation in cost effectiveness analysis in health care has been the development of the quality adjusted life year (QALY) that combines longevity with quality of life. It achieves this by assigning a preference value (utility weight) to each health state experienced by a patient on a scale where 0.0 reflects being dead and 1.0 reflects full health [1]. Evaluation of the incremental cost per QALY gained, or cost-utility analyses, is now a standard type of cost-effectiveness analysis [2,3]. The last decade has seen increasing use made of generic utility measures that are assigned preference weights based on the general population perspective, as recommended by the US Panel on Cost-Effectiveness in Health and Medicine and used by the National Institute for * Correspondence: [email protected] 1 Oxford Outcomes Inc, an ICON plc Company, 7315 Wisconsin Ave. Ste 250 W, Bethesda, MD 20814, UK Full list of author information is available at the end of the article
Health and Clinical Excellence (NICE) [4,5]. Examples of such measures include the EQ-5D and the SF-6D [6,7]. These are multi-attribute scales
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