Estimating utility values for non-alcoholic steatohepatitis health states: a discrete choice experiment.
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SUBMITTER: Aggio D
PROVIDER: S-EPMC10842270 | biostudies-literature | 2024 Feb
REPOSITORIES: biostudies-literature
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Estimating utility values for non-alcoholic steatohepatitis health states: a discrete choice experiment.
Aggio Daniel D Gallop Katy K Wittrup-Jensen Villum V Farsani Soulmaz Fazeli SF Lloyd Andrew J AJ
Journal of comparative effectiveness research 20240116 2
<b>Background:</b> This study estimated utility values for non-alcoholic steatohepatitis (NASH). Previous studies have assumed that health-related quality of life does not vary between the early stages of NASH. <b>Materials & Methods:</b> Discrete choice experiment (DCE) surveys estimated the value of avoiding fibrosis progression. Patients also completed the EQ-5D-5L. Marginal rates of substitution estimated utility change associated with fibrosis progression. <b>Results:</b> DCE surveys were c ...[more]
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Project description:BackgroundAtypical hemolytic uremic syndrome is a rare disease caused by complement dysregulation that can lead to progressive kidney damage or death if untreated. Owing to its rarity, the impact of atypical hemolytic uremic syndrome and available therapies (eculizumab and ravulizumab) on patients' health-related quality of life is difficult to describe, but such data are required for an economic evaluation.ObjectiveThe objective of this study was to estimate utility values for atypical hemolytic uremic syndrome-related attributes in five countries for an economic evaluation.MethodsUsing discrete choice experiment surveys, key atypical hemolytic uremic syndrome-related attributes (life expectancy, administration frequency, risk of meningitis, need for hospitalization, and risk of kidney impairment) were evaluated in adult general population samples from Australia, Canada, the Netherlands, Sweden, and the UK. Survey choice sets were constructed using a published orthogonal array. A mixed-effects logit model estimated preference strength for each attribute. Utilities were estimated using marginal substitution rates between overall survival and other attributes, weighted against average life expectancy.ResultsAcross all countries (N = 2382), utility weights revealed a consistent pattern: participants were averse to the risk of kidney impairment (disutility/utility weight range: -0.185 to -0.158), risk of meningitis (-0.041 to -0.032), and the need for hospitalization (-0.063 to -0.048), but preferred 8-weekly vs 2-weekly infusions over 1 h (0.013-0.039).ConclusionsAlthough all attributes played a role in determining treatment preferences, the largest drivers were life expectancy and risk of kidney impairment. Participants favored 8-weekly dosing (corresponding to ravulizumab administration frequency) vs 2-weekly dosing. The discrete choice experiment was designed such that estimated (dis)utility weights can be used in future cost-effectiveness models in atypical hemolytic uremic syndrome.
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Project description:ObjectivesThe importance of adolescent engagement in health decisions and public health programs such as immunisation is becoming increasingly recognised. Understanding adolescent preferences and further identifying barriers and facilitators for immunisation acceptance is critical to the success of adolescent immunisation programs. This study applied a discrete choice experiment (DCE) to assess vaccination preferences in adolescents.MethodsThis study was conducted as a cross-sectional, national online survey in Australian adolescents. The DCE survey evaluated adolescent vaccination preferences. Six attributes were assessed including disease severity, target for protection, price, location of vaccination provision, potential side effects and vaccine delivery method. A mixed logit model was used to analyse DCE data.ResultsThis survey was conducted between December 2014 and January 2015. Of 800 adolescents aged 15 to 19 years, stronger preferences were observed overall for: vaccination in the case of a life threatening illness (p<0.001), lower price vaccinations (p<0.001), mild but common side effects (p = 0.004), delivery via a skin patch (p<0.001) and being administered by a family practitioner (p<0.001). Participants suggested that they and their families would be willing to pay AU$394.28 (95%CI: AU$348.40 to AU$446.92) more for a vaccine targeting a life threatening illness than a mild-moderate illness, AU$37.94 (95%CI: AU$19.22 to AU$57.39) more for being vaccinated at a family practitioner clinic than a council immunisation clinic, AU$23.01 (95%CI: AU$7.12 to AU$39.24) more for common but mild and resolving side effects compared to rare but serious side effects, and AU$51.80 (95%CI: AU$30.42 to AU$73.70) more for delivery via a skin patch than injection.ConclusionsConsideration of adolescent preferences may result in improved acceptance of, engagement in and uptake of immunisation programs targeted for this age group.
| S-EPMC5528895 | biostudies-literature
Project description:ImportanceA glaucoma-specific instrument for estimating utilities across the spectrum of glaucoma severity is currently lacking, hindering the assessment of the cost-effectiveness of glaucoma treatments.ObjectiveTo develop and validate the preference-based Glaucoma Utility Instrument (Glau-U) and to ascertain the association between Glau-U utilities and severity of glaucoma and vision impairment.Design, setting, and participantsThis cross-sectional study was conducted in 2 stages at the Singapore National Eye Centre glaucoma clinics. Stage 1 focused on the identification and pretesting of the Glau-U attributes and was carried out between June 2009 and May 2016. Stage 2 involved the development and administration of the discrete choice experiment (DCE) survey and tasks and was conducted between May 7, 2018, and December 11, 2019. Stage 2 participants were English- or Mandarin-speaking Singaporean citizens or permanent residents of Chinese, Malay, or Indian ethnicity who were 40 years or older and had a clinical diagnosis of glaucoma in at least 1 eye.ExposuresGlau-U comprised 6 quality-of-life attributes: activities of daily living, lighting and glare, movement, eye discomfort, other effects of glaucoma, and social and emotional effects. The descriptions or response options for these attributes were no difficulty or never, some difficulty or sometimes, or severe difficulty or often.Main outcomes and measuresUtility weights for Glau-U were developed using a DCE questionnaire, which was interviewer administered to participants. Mixed logit regression determined utility weights for each health state. Glau-U utility weights across better- or worse-eye glaucoma and vision impairment severity were calculated using 1-way analysis of variance. Correlations between Glau-U utilities and better- or worse-eye visual fields and EuroQol 5-Dimension utilities were ascertained to assess convergent and divergent validity.ResultsOf the 304 participants (mean [SD] age, 68.3 [8.7] years; 182 men [59.9%]), 281 (92.4%) had no vision impairment in the better eye, 13 (4.3%) had mild impairment, and 10 (3.3%) had moderate to severe vision impairment. Mean (SD) Glau-U utilities decreased as better-eye glaucoma severity increased (none: 0.73 [0.21]; mild: 0.66 [0.21]; moderate: 0.66 [0.20]; severe: 0.60 [0.28]; and advanced or end-stage: 0.22 [0.38]; P < .001), representing reductions of 20.7% to 76.1% in quality-adjusted life-years compared with a health state that included preperimetric glaucoma. Mean (SD) Glau-U utilities also decreased as better-eye vision impairment worsened from 0.67 (0.23) for none to 0.58 (0.32) for mild to 0.46 (0.29) for moderate to severe vision impairment. Glau-U utilities demonstrated moderate correlations with better-eye (r = 0.34; P < .001) and worse-eye (r = 0.33; P < .001) mean deviation scores and low correlations with EuroQol 5-Dimension utilities (r = 0.22; P < .001), supporting convergent and divergent validity.Conclusions and relevanceUse of Glau-U revealed large decrements in utility that were associated with late-stage glaucoma, suggesting that this new instrument may be useful for cost-effectiveness analyses of interventions and informing resource allocation policies for glaucoma and vision loss.
| S-EPMC8227456 | biostudies-literature
Project description:This paper is based upon the "Charles Lieber Satellite Symposia" organized by Manuela G. Neuman at the Research Society on Alcoholism (RSA) Annual Meetings, 2013 and 2014. The present review includes pre-clinical, translational and clinical research that characterize alcoholic liver disease (ALD) and non-alcoholic steatohepatitis (NASH). In addition, a literature search in the discussed area was performed. Strong clinical and experimental evidence lead to recognition of the key toxic role of alcohol in the pathogenesis of ALD. The liver biopsy can confirm the etiology of NASH or alcoholic steatohepatitis (ASH) and assess structural alterations of cells, their organelles, as well as inflammatory activity. Three histological stages of ALD are simple steatosis, ASH, and chronic hepatitis with hepatic fibrosis or cirrhosis. These latter stages may also be associated with a number of cellular and histological changes, including the presence of Mallory's hyaline, megamitochondria, or perivenular and perisinusoidal fibrosis. Genetic polymorphisms of ethanol metabolizing enzymes such as cytochrome p450 (CYP) 2E1 activation may change the severity of ASH and NASH. Alcohol mediated hepatocarcinogenesis, immune response to alcohol in ASH, as well as the role of other risk factors such as its co-morbidities with chronic viral hepatitis in the presence or absence of human immunodeficiency virus are discussed. Dysregulation of hepatic methylation, as result of ethanol exposure, in hepatocytes transfected with hepatitis C virus (HCV), illustrates an impaired interferon signaling. The hepatotoxic effects of ethanol undermine the contribution of malnutrition to the liver injury. Dietary interventions such as micro and macronutrients, as well as changes to the microbiota are suggested. The clinical aspects of NASH, as part of metabolic syndrome in the aging population, are offered. The integrative symposia investigate different aspects of alcohol-induced liver damage and possible repair. We aim to (1) determine the immuno-pathology of alcohol-induced liver damage, (2) examine the role of genetics in the development of ASH, (3) propose diagnostic markers of ASH and NASH, (4) examine age differences, (5) develop common research tools to study alcohol-induced effects in clinical and pre-clinical studies, and (6) focus on factors that aggravate severity of organ-damage. The intention of these symposia is to advance the international profile of the biological research on alcoholism. We also wish to further our mission of leading the forum to progress the science and practice of translational research in alcoholism.
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Project description:IntroductionType 2 diabetes mellitus (T2DM) is a chronic condition associated with substantial clinical and economic burden. As multiple therapeutic options are available, patient preferences on treatment characteristics are key in T2DM therapeutic decision-making. This study aimed to determine the preferences of US patients with T2DM for therapies recommended for first pharmacologic intensification after metformin.MethodsAs part of a discrete choice experiment, an online survey was designed using literature review and qualitative interview findings. Eligibility was met by US patients with T2DM who were aged 18 years or older with an HbA1c ≥ 6.5%. Anonymized therapy profiles were created from six antidiabetic therapies including oral and injectable semaglutide, dulaglutide, empagliflozin, sitagliptin, and thiazolidinediones.ResultsEligible patients (n = 500) had a mean HbA1c of 7.4%, and a mean BMI of 32.0 kg/m2, the majority of which (72.2%) were injectable-naïve. The treatment characteristic with greatest importance was mode and frequency of administration (35.5%), followed by body weight change (29.2%), cardiovascular event risk (19.1%), hypoglycemic event risk (9.9%), and HbA1c change (6.5%). An oral semaglutide-like profile was preferred by 91.9-70.1% of respondents depending on the comparator agent, and preference was significant in each comparison (p < 0.05); an injectable semaglutide-like profile was preferred by 89.3-55.7% of respondents in each comparison depending on the comparator agent.ConclusionPatients with T2DM in the USA are significantly more likely to prefer oral or injectable semaglutide-like profiles over those of key comparators from the glucagon-like peptide 1 receptor agonist, sodium-glucose cotransporter 2 inhibitor, dipeptidyl peptidase 4 inhibitor, and thiazolidinedione classes.
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Project description:Healthcare systems are increasingly considering widespread implementation of rapid genomic testing of critically ill children, but evidence on the value of the benefits generated is lacking. This information is key for an optimal implementation into healthcare systems. A discrete choice experiment survey was designed to elicit preferences and values for rapid genomic testing in critically ill children. The survey was administered to members of the Australian public and families with lived experience of rapid genomic testing. A Bayesian D-efficient explicit partial profiles design was used, and data were analysed using a panel error component mixed logit model. Preference heterogeneity was explored using a latent class model and fractional logistic regressions. The public (n = 522) and families with lived experiences (n = 25) demonstrated strong preferences for higher diagnostic yield and clinical utility, faster result turnaround times, and lower cost. Society on average would be willing to pay an additional AU$9510 (US$6657) for rapid (2 weeks results turnaround time) and AU$11,000 (US$7700) for ultra-rapid genomic testing (2 days turnaround time) relative to standard diagnostic care. Corresponding estimates among those with lived experiences were AU$10,225 (US$7158) and AU$11,500 (US$8050), respectively. Our work provides further evidence that rapid genomic testing for critically ill children with rare conditions generates substantial utility. The findings can be used to inform cost-benefit analyses as part of broader healthcare system implementation.
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Project description:ObjectiveTo establish which attributes of conservative treatments for prostate cancer are most important to men.DesignDiscrete choice experiment.SettingTwo London hospitals.Participants129 men with non-metastatic prostate cancer, mean age 70 years; 69 of 118 (58%) with T stage 1 or 2 cancer at diagnosis.Main outcome measuresMen's preferences for, and trade-offs between, the attributes of diarrhoea, hot flushes, ability to maintain an erection, breast swelling or tenderness, physical energy, sex drive, life expectancy, and out of pocket expenses.ResultsThe men's responses to changes in attributes were all statistically significant. When asked to assume a starting life expectancy of five years, the men were willing to make trade-offs between life expectancy and side effects. On average, they were most willing to give up life expectancy to avoid limitations in physical energy (mean three months) and least willing to trade life expectancy to avoid hot flushes (mean 0.6 months to move from a moderate to mild level or from mild to none).ConclusionsMen with prostate cancer are willing to participate in a relatively complex exercise that weighs up the advantages and disadvantages of various conservative treatments for their condition. They were willing to trade off some life expectancy to be relieved of the burden of troublesome side effects such as limitations in physical energy.
| S-EPMC341386 | biostudies-literature
Project description:While metabolic syndrome and alcohol consumption are the two main causes of chronic liver disease, one of the two conditions is often predominant, with the other acting as a cofactor of morbimortality. It has been shown that obesity and alcohol act synergistically to increase the risk of fibrosis progression, hepatic carcinogenesis and mortality, while genetic polymorphisms can strongly influence disease progression. Based on common pathogenic pathways, there are several potential targets that could be used to treat both diseases; based on the prevalence and incidence of these diseases, new therapies and clinical trials are needed urgently.
| S-EPMC7267467 | biostudies-literature
Project description:BackgroundAscertaining preferences for SARS-CoV-2 testing and incorporating findings into the design and implementation of strategies for delivering testing services may enhance testing uptake and engagement, a prerequisite to reducing onward transmission.ObjectiveThis study aims to determine important drivers of decisions to obtain a SARS-CoV-2 test in the context of increasing community transmission.MethodsWe used a discrete choice experiment to assess preferences for SARS-CoV-2 test type, specimen type, testing venue, and results turnaround time. Participants (n=4793) from the US national longitudinal Communities, Households and SARS-CoV-2 Epidemiology (CHASING) COVID Cohort Study completed our online survey from July 30 to September 8, 2020. We estimated the relative importance of testing method attributes and part-worth utilities of attribute levels, and simulated the uptake of an optimized testing scenario relative to the current typical testing scenario of polymerase chain reaction (PCR) via nasopharyngeal swab in a provider's office or urgent care clinic with results in >5 days.ResultsTest result turnaround time had the highest relative importance (30.4%), followed by test type (28.3%), specimen type (26.2%), and venue (15.0%). In simulations, immediate or same-day test results, both PCR and serology, or oral specimens substantially increased testing uptake over the current typical testing option. Simulated uptake of a hypothetical testing scenario of PCR and serology via a saliva sample at a pharmacy with same-day results was 97.7%, compared to 0.6% for the current typical testing scenario, with 1.8% opting for no test.ConclusionsTesting strategies that offer both PCR and serology with noninvasive methods and rapid turnaround time would likely have the most uptake and engagement among residents in communities with increasing community transmission of SARS-CoV-2.
| S-EPMC7781587 | biostudies-literature
Project description:IntroductionA decade ago, the first national valuation study of the EQ-5D-Y-3L (Y-3L) involved a discrete choice experiment (DCE) that asked 4155 US adult respondents to complete 40 paired comparisons, choosing between two dying children. Instead of choosing between dying children, the respondents in this novel protocol are asked whether 'being in a coma' is better or worse than experiencing 'health problems' (ie, experience scale) and how they would relieve health problems (ie, kaizen tasks). Our aims are to compare the preference evidence of the paired comparison and kaizen tasks and to conduct a DCE for the valuation of Y-3L profiles on an experience scale.Methods and analysisUnder this protocol, we will conduct an online survey that collects preference evidence from 600 US adult respondents on the health of a 10-year-old child for a week. Across all scenarios, each child will be described as either being 'in a coma' or having 'health problems', namely five three-level attributes (Y-3L). In this DCE, each respondent will be randomly assigned to one of four D-efficient blocks, including five coma comparisons (ie, Y-3L vs coma), 10 paired comparisons (Y-3L vs Y-3L) and 10 kaizen tasks (preference paths). In addition to comparing evidence by task (aim 2), the analysis plan includes the estimation of main-effects conditional logit models to create a Y-3L value set on an 'experience scale' where positive (negative) experiences have positive (negative) values (0 is 'being in a coma' and 1 is full health).Ethics and disseminationThe institutional review board (IRB) (Advarra) determined that this project (Pro00072276) is exempt from IRB oversight based on DHHS 45 CFR 46.104(d)(2) and is not subject to requirements for continuing review. The results will be prepared for publication in peer-reviewed journals and presented at scientific meetings. The data and code will be made available on reasonable request.
| S-EPMC10603523 | biostudies-literature