摘要：Health state (HS) utility values for patients with acute myeloid leukemia (AML), a hematological malignancy, are not available in the United Kingdom (UK). This study aims to develop clinically sound HSs for previously untreated patients with AML and to assign utility values based on preferences of the general UK population. This study was conducted in the UK and comprised 2 stages. During the first stage, AML HSs were drafted based on evidence from a literature review of AML clinical and health-related quality-of-life studies (published January 2000–June 2016) and patient-reported outcome measures previously used in this population. A panel of UK hematologists with AML experience validated the clinical relevance and accuracy of the HSs. During the second stage, validated HSs were valued in an elicitation survey with a representative UK population sample using the time trade-off (TTO) method. Descriptive statistics and bivariate tests were obtained and performed. A total of eight HSs were developed and clinically validated, including treatment with chemotherapy, consolidation therapy, transplant, graft-vs-host disease (GvHD), remission, relapse, refractory, and functionally cured. In total, 125 adults participated (mean age, 49.6 years [range, 18–87 years], 52.8% female). Mean (95% confidence interval [CI]) TTO preference values (n = 120), ranked from lowest (worst HS) to highest (best HS) were as follows: refractory − 0.11 (− 0.21 to − 0.01), relapse 0.10 (0.00–0.20), transplant 0.28 (0.20–0.37), treatment with chemotherapy 0.36 (0.28–0.43), GvHD 0.43 (0.36–0.50), consolidation 0.46 (0.40–0.53), remission 0.62 (0.57–0.67), and functionally cured 0.76 (0.72–0.79). Mean (95% CI) visual analog scale preference values followed the same rank order, ranging from 0.15 (0.13–0.17) for refractory to 0.71 (0.68–0.73) for functionally cured. To our knowledge, this is the first study to report utility values for AML from the UK societal perspective. Participants were able to distinguish differences in severity among AML HSs, and preference values were consistent with clinical perception of HS severity. HS preference values observed in this study may be useful in future evaluations of treatment benefit, including cost-effectiveness analyses and improved patient well-being.
关键词：Acute myeloid leukemia ; Health-related quality of life ; Time trade-off ; Health states ; Utility values
