title: Deriving a Preference-Based Measure for Myelofibrosis from the EORTC QLQ-C30 and the MF-SAF.
abstract: BACKGROUND
  Utility values are required for economic evaluation using cost-utility analyses. Often, generic measures such as the EuroQol five-dimensional questionnaire are used, but this may not appropriately reflect the health-related quality of life of patients with cancer including myelofibrosis.
  
  
  OBJECTIVE
  To derive a condition-specific preference-based measure for myelofibrosis using appropriate existing measures, the Myelofibrosis-Symptom Assessment Form and the European Organisation for Research and Treatment of Cancer Quality of Life 30 Questionnaire.
  
  
  METHODS
  Data from the Controlled Myelofibrosis Study with Oral JAK Inhibitor Treatment trial (n = 309) were used to derive the health state classification system. Psychometric and factor analyses were used to determine the dimensions of the classification system. Psychometric and Rasch analyses were then used to select an item to represent each dimension. Item selection was validated with experts. A selection of health states was valued by members of the general population using time trade-off. Finally, health state values were modeled using regression analysis to produce utility values for every state.
  
  
  RESULTS
  The Myelofibrosis 8 dimensions has eight dimensions: physical functioning, emotional functioning, fatigue, itchiness, pain under ribs on the left side, abdominal discomfort, bone or muscle pain, and night sweats. Regression models were estimated using time trade-off data from 246 members of the general population valuing a total of 33 states. The best performing model was a random effects maximum likelihood model producing utility values ranging from 0.089 to 1.
  
  
  CONCLUSIONS
  The Myelofibrosis 8 dimensions is a condition-specific preference-based measure for myelofibrosis. This measure can be used to generate utility values for myelofibrosis for any data set containing the Myelofibrosis-Symptom Assessment Form and the European Organisation for Research and Treatment of Cancer Quality of Life 30 Questionnaire data.
authors.affiliation: Health Economics and Decision Science, School of Health and Related Research, The University of Sheffield, Sheffield, UK. Electronic address: c.mukuria@sheffield.ac.uk.
authors.forename: Clara
authors.initials: C
authors.lastname: Mukuria
authors.affiliation: Health Economics and Decision Science, School of Health and Related Research, The University of Sheffield, Sheffield, UK.
authors.forename: Donna
authors.initials: D
authors.lastname: Rowen
authors.affiliation: Health Economics and Decision Science, School of Health and Related Research, The University of Sheffield, Sheffield, UK.
authors.forename: John E
authors.initials: JE
authors.lastname: Brazier
authors.affiliation: Health Economics and Decision Science, School of Health and Related Research, The University of Sheffield, Sheffield, UK.
authors.forename: Tracey A
authors.initials: TA
authors.lastname: Young
authors.affiliation: ICON plc, Dublin, Ireland.
authors.forename: Beenish
authors.initials: B
authors.lastname: Nafees
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D017326:Clinical Trials, Phase III as Topic; D005163:Factor Analysis, Statistical; D005260:Female; D006302:Health Services Research; D006304:Health Status; D006801:Humans; D053612:Janus Kinases / Q000037:antagonists & inhibitors / Q000378:metabolism; D008297:Male; D008875:Middle Aged; D015233:Models, Statistical; D015337:Multicenter Studies as Topic; D057240:Patient Preference; D011237:Predictive Value of Tests; D055728:Primary Myelofibrosis / Q000175:diagnosis / Q000188:drug therapy* / Q000503:physiopathology / Q000523:psychology; D011494:Protein Kinases / Q000009:adverse effects / Q000627:therapeutic use*; D011594:Psychometrics; D011788:Quality of Life; D019057:Quality-Adjusted Life Years; D016032:Randomized Controlled Trials as Topic; D011795:Surveys and Questionnaires; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D011494:Protein Kinases; D053612:Janus Kinases
keywords: EORTC QLQ-C30; MF-SAF; myelofibrosis; preference-based measure