title: Development of the multi-attribute Adolescent Health Utility Measure (AHUM).
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.
  
  
  CONCLUSIONS
  The AHUM health state classification system may be used in future research to enable calculation of quality-adjust life expectancy for applicable health conditions.
authors.affiliation: Oxford Outcomes Inc, an ICON plc Company, 7315 Wisconsin Ave, Ste 250 W, Bethesda, MD 20814, USA. kathy.beusterien@oxfordoutcomes.com
authors.forename: Kathleen M
authors.initials: KM
authors.lastname: Beusterien
authors.forename: Jean-Ezra
authors.initials: JE
authors.lastname: Yeung
authors.forename: Francis
authors.initials: F
authors.lastname: Pang
authors.forename: John
authors.initials: J
authors.lastname: Brazier
mesh_terms: D000203:Activities of Daily Living / Q000523:psychology; D000293:Adolescent; D000295:Adolescent Medicine / Q000295:instrumentation*; D000328:Adult; D002908:Chronic Disease / Q000523:psychology / Q000628:therapy; D015897:Comorbidity; D003365:Costs and Cost Analysis; D005191:Family Characteristics; D005260:Female; D006302:Health Services Research; D006305:Health Status Indicators; D006801:Humans; D007066:Iduronate Sulfatase / Q000627:therapeutic use; D008297:Male; D008875:Middle Aged; D015233:Models, Statistical; D016532:Mucopolysaccharidosis II / Q000150:complications / Q000188:drug therapy; D011247:Pregnancy; D036301:Qualitative Research; D011788:Quality of Life; D019057:Quality-Adjusted Life Years; D015203:Reproducibility of Results; D012647:Self-Assessment; D012923:Social Class; D011795:Surveys and Questionnaires; D013997:Time Factors; D006113:United Kingdom; D026712:Value of Life
chemical_list: D007066:Iduronate Sulfatase