title: Population preference values for health states in relapsed or refractory B-precursor acute lymphoblastic leukemia in the United Kingdom.
abstract: BACKGROUND
  To date, reliable and comprehensive health-related quality of life data for patients with relapsed or refractory B-precursor acute lymphoblastic leukemia (ALL) have not been collected in clinical trials of the disease, and no utility studies have been published. The purpose of this study was to define and validate health states experienced by adults with relapsed/refractory B-precursor ALL, and to assign utility values to these health states using time-trade off methodology.
  
  
  METHODS
  This study was conducted in the UK in three phases. In the first phase, five health state descriptions were developed based on a recent clinical trial. The second phase validated the health state descriptions with clinicians and patients with experience of relapsed/refractory B-precursor ALL. The third phase involved prospective health state valuation using time-trade off methodology in a sample of the general public. The study was approved by the UK National Health Service Research Ethics Committee.
  
  
  RESULTS
  In total, 123 participants were recruited and included in the final analysis; all participants gave written, informed consent. Complete remission was the most preferred health state (mean utility [SEM], 0.86 [0.01]), followed by complete remission with partial hematological recovery (with minimal risk of bleeding or developing infection) (0.75 [0.02]); aplastic bone marrow (0.59 [0.02]); partial remission (0.50 [0.03]); and progressive disease (0.30 [0.04]).
  
  
  CONCLUSIONS
  This is the first study to report utility values for health states associated with relapsed/refractory B-precursor ALL. It was designed and conducted to align with NICE guidance on alternative methods for generating health state utility values when EQ-5D data are either unavailable or inappropriate. These utilities can be applied in future cost-effectiveness analyses of treatment for relapsed/refractory B-precursor ALL.
authors.affiliation: PRMA Consulting, Fleet, UK. MAristides@prmaconsulting.com.
authors.forename: Mike
authors.initials: M
authors.lastname: Aristides
authors.affiliation: Amgen GHE, Thousand Oaks, USA.
authors.forename: Arie
authors.initials: A
authors.lastname: Barlev
authors.affiliation: Amgen GHE, Thousand Oaks, USA.
authors.forename: Beth
authors.initials: B
authors.lastname: Barber
authors.affiliation: PRMA Consulting, Fleet, UK.
authors.forename: Merel
authors.initials: M
authors.lastname: Gijsen
authors.affiliation: PRMA Consulting, Fleet, UK.
authors.forename: Casey
authors.initials: C
authors.lastname: Quinn
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D005260:Female; D006304:Health Status; D006801:Humans; D008297:Male; D008875:Middle Aged; D057240:Patient Preference / Q000523:psychology*; D054198:Precursor Cell Lymphoblastic Leukemia-Lymphoma / Q000523:psychology* / Q000628:therapy*; D011446:Prospective Studies; D011594:Psychometrics / Q000295:instrumentation*; D011788:Quality of Life / Q000523:psychology*; D006113:United Kingdom; D055815:Young Adult