Social preferences for health states associated with acute myeloid leukemia for patients undergoing treatment in the United Kingdom

Abstract

BackgroundHealth 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.MethodsThis 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.ResultsA 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.ConclusionsTo 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.

American Society of Hematology 2020 guidelines for treating newly diagnosed acute myeloid leukemia in older adults

“…62,64,[85][86][87][88][89][90][91][92][93][94][95][96][97] Eighteen additional studies were reviewed but excluded from the meta-analyses due to lack of data on the outcomes prioritized by the expert panel. [98][99][100][101][102][103][104][105][106][107][108][109][110][111][112][113][114][115][116] Given the challenges in randomizing patients to intensive or less-intensive treatments, most of the included studies were observational. 62,85,86,93,95,96 Two were randomized clinical trials (RCTs).…”

“…One study provided evidence regarding acceptability of antileukemic therapy, suggesting that the value of the health state of receiving therapy is 0.50. 116 The importance of patient-reported outcome research is recognized now more than ever, but there remains a paucity of information on how older AML patients view their therapeutic options and goals of treatment. Existing data illustrate that patients recognize the importance of therapeutic milestones such as CR.…”

“…There were 3 studies addressing patients' values and preferences regarding the outcomes of interest. 116,119,120 These showed that patients placed a high value on achieving CR (health state median, 0.70, on a scale from 0 to 1, where 0 is dead and 1 is totally healthy) 115 and consider relapse an outcome with a negative value (health state and utility ranged from 0.10 to 0.50 across studies). 116,119,120 There were 3 studies providing evidence regarding costs 54,135,136 and 1 providing evidence relevant to equity.…”

“…116,119,120 These showed that patients placed a high value on achieving CR (health state median, 0.70, on a scale from 0 to 1, where 0 is dead and 1 is totally healthy) 115 and consider relapse an outcome with a negative value (health state and utility ranged from 0.10 to 0.50 across studies). 116,119,120 There were 3 studies providing evidence regarding costs 54,135,136 and 1 providing evidence relevant to equity. 137 Because these 2 factors did not have an important bearing on the recommendation, their results are presented only in the EtD.…”

“…These showed that patients placed a high value on being in CR (utility, 0.88, on a scale from 0 to 1, where 0 is dead and 1 is totally healthy) 120 and consider relapse an outcome with a negative value (health state and utility ranged from 0.10 to 0.50 across studies). 116,119,120 In 2 of these studies, the researchers also explored how patients accept postremission therapy and reported that the median health state attributed to consolidation therapy ranged from 0.47 to 0.70. 116,119 There was no research evidence regarding costs and feasibility.…”