Quantifying Patient Risk Threshold in Managing Pancreatic Intraductal Papillary Mucinous Neoplasms.

OBJECTIVE: We aimed to better understand patients' treatment preferences and quantify the level of cancer risk at which treatment preferences change (risk threshold) to inform better counseling of patients with intraductal papillary mucinous neoplasms (IPMNs). SUMMARY BACKGROUND DATA: The complexity of IPMN management provides an opportunity to align treatment with individual preference. METHODS: We surveyed a sample of healthy volunteers simulating a common scenario: undergoing an imaging study that incidentally identifies an IPMN. In the scenario, the estimated risk of cancer in the IPMN was 5%. Patients were asked their treatment preference (surgery or surveillance), to quantify the level of cancer risk in the IPMN at which their treatment preference would change (i.e. risk threshold), and their level of cancer anxiety as measured on a 5-point Likert scale. We examined associations between participant characteristics, treatment preferences, and risk threshold using multivariable linear regression. RESULTS: The median risk threshold among the 520 participants was 25% (IQR 2.3-50%). The risk threshold had a bimodal distribution: 40% of participants had a risk threshold between 0-10% and 47% had a risk threshold above 30%. When informed that the risk of cancer was 5%, 62% of participants (n=323) preferred surveillance, and the remaining 38% (n=197) preferred surgery. After adjusting for potential confounders, participants who expressed "worry" or "extreme worry" about the malignancy risk of IPMN had significantly lower risk thresholds than participants who were "not at all worried" (Coefficient -12, 95%CI -21 to -2, P=0.015 and Coefficient -18, 95%CI -29 to -8, P<0.001, respectively). CONCLUSIONS: Participants varied in treatment preference and risk threshold of incidentally identified IPMNs. Given the uncertainty in estimating the true malignant potential of IPMNs, a better understanding of a patient's risk threshold, as influenced by patient concern about malignancy, will help inform the shared decision-making process.

Public Perspectives on Multi-Cancer Early Detection: A Qualitative Study.

BACKGROUND: Multi-cancer early detection tests (MCEDs) have the potential to identify over 50 types of cancer from a blood sample, possibly transforming cancer screening paradigms. Studies on the safety and effectiveness of MCEDs are underway, but there is a paucity of research exploring public views on MCEDs. We sought to explore public perspectives and understanding on the use of MCEDs in patient care. METHODS: We conducted a cross-sectional, qualitative study using one-on-one, semi-structured interviews. Residents of the United States aged 45-70 years old were recruited through a survey panel and purposively sampled to maximize racial diversity. Interviews explored understanding of MCEDs and perspectives on their use. Interviews were analyzed using thematic analysis with deductive coding and semi-quantification. RESULTS: Among 27 participants, mean age was 62 years (range 48-70) and most (63%) were non-white. Most participants had completed at least one cancer screening (89%). Participants had a positive impression of MCEDs (85%) and found the concept easy to understand (88%). They were enthusiastic about the convenience of MCEDs (30%) and thought they would improve "cancer outcomes" by looking for multiple cancers (70%) and facilitating early detection (33%). Participants emphasized the need to balance these benefits against potential harms, including inaccuracy (96%), cost (92%), test-related anxiety (56%), and lack of evidence of effectiveness (22%). Participants favored that MCEDs be delivered in primary care (93%). Participants worried that the potential benefits of MCEDs might not be equitably distributed (44%). CONCLUSIONS: Members of the US public in this study expressed an interest in using MCEDs but had concerns regarding cost, accuracy, and potential inequitable access to the tests. Findings suggest that MCEDs that are found to be safe and effective will be acceptable to patients as a part of primary care, and underscore public interest in improving this technology.

Uncertainty Profiles and Treatment Preferences for Intraductal Papillary Mucinous Neoplasms.

INTRODUCTION: Intraductal papillary mucinous neoplasms (IPMNs) are pancreatic premalignant lesions frequently detected incidentally. Choosing between surgery and surveillance for IPMNs is rooted in uncertainty. We characterized patient preferences in IPMN management, and examined associations with patients' uncertainty profiles (risk perception, risk attitude, and uncertainty tolerance). METHODS: We conducted a cross-sectional survey drawn from a national opt-in panel. We simulated an encounter following an incidental computed tomography scan finding of an IPMN with a 5% cancer risk. We elicited participants' preferred treatment (surgery versus surveillance). Participant cancer risk perception, risk attitude (risk seeking versus risk averse), and uncertainty tolerance (comfort with the unknown) were determined using validated measures. Multivariate regression models assessed for independent predictors of treatment preference and risk perception. RESULTS: The sample included 520 participants, ages 40-70, racially representative of the US population. Participants preferred surveillance (n = 331, 64%) over surgery (n = 189, 36%). Patients were significantly more likely to prefer surgery as their cancer risk perception increased (absolute difference = 12% from 1.0 standard deviation below to 1.0 standard deviation above the mean, 95% CI 3.5-20.2). Treatment preference was not significantly associated with risk attitude (P = 0.068) or uncertainty tolerance (P = 0.755). However, initial cancer risk perception was significantly associated with both uncertainty tolerance (P = 0.013) and baseline cancer anxiety (risk perception 16.4% versus 65%, not worried at all versus extremely worried, P < 0.001). CONCLUSIONS: Patient preference varies widely for IPMN and is significantly associated with cancer risk perception, which is, in turn, significantly associated with uncertainty tolerance and cancer anxiety. These findings argue for the preference-sensitive nature of IPMN treatment decisions.

Cost Comparison and Spending on Tobacco Products: Evidence from A Nationally Representative Sample of Adult E-Cigarette Users.

INTRODUCTION: Over 20 states and local jurisdictions in the U.S. have imposed e-cigarette taxes. It is important to evaluate how adult vapers, including those who also smoke respond to e-cigarette taxation. The purpose of this study is to examine factors associated with adult vapers' cost comparison between e-cigarettes and cigarettes (i.e., which one is perceived to be more expensive) and expenditures (i.e., dollars spent) on these two products. METHODS: We recruited a nationally representative sample of 801 adult e-cigarette users in the U.S., who participated in an online survey in April-May 2023. Nested-ordered logit and ordinary least squares regressions were used in analysis. RESULTS: On average, adult vapers spent $82.22 per month on e-cigarettes, and dual users spent $118.77 per month on cigarettes. More frequent e-cigarette use, lower state-level e-cigarette taxes, age and exclusive use of rechargeable tank systems with e-liquids were associated with considering e-cigarettes as less expensive than cigarettes. Exclusive use of tank systems was associated with lower e-cigarette spending. Adults who used e-cigarettes more frequently preferred to report weekly (versus monthly) spending on e-cigarettes; among dual users, everyday smokers were more likely to think of weekly (versus monthly) spending on cigarettes compared to someday smokers. CONCLUSIONS: Among US adult vapers, tobacco use frequency and e-cigarette device type are closely related to cost measures; e-cigarette taxes are associated with cost comparison between e-cigarettes and cigarettes, suggesting potential financial disincentive for vaping. Policymaker may consider imposing differential taxes by e-cigarette product types due to their different costs to consumers. IMPLICATIONS: This study provides supportive evidence that e-cigarette taxes may impact how vapers compare the costs of e-cigarettes relative to cigarettes. Given different expenditures and cost comparisons by e-cigarette device types, tax and policy design may need to take into consideration of e-cigarette device types. We also found that indoor tobacco use restrictions in shared housing may reduce vaping, and future research is needed. The associations between socio-demographics and cost comparison and expenditures reflect known patterns in price responsiveness and vaping prevalence. Cost comparison and expenditures are related measures, which may further reflect product affordability.

Experiences of treatment decision-making among older newly diagnosed adults with acute myeloid leukemia: a qualitative descriptive study.

PURPOSE: Treatment decision-making for older adults with acute myeloid leukemia (AML) is complex and preference-sensitive. We sought to understand the patient experience of treatment decision-making to identify specific challenges in shared decision-making to improve clinical care and to inform the development of directed interventions. METHODS: We conducted in-depth interviews with newly diagnosed older (≥ 60 years) adults with AML and their caregivers following a semi-structured interview guide at a public safety net academic hospital. Interviews were digitally recorded, and qualitative thematic analysis was employed to synthesize findings. RESULTS: Eighteen in-depth interviews were conducted. Age ranged from 62 to 78 years. Patients received intermediate- (50%) or high-intensity (44%) chemotherapy or best supportive care only (6%). Six themes of patient experiences emerged from the analysis: patients (1) felt overwhelmed and in shock at diagnosis, (2) felt powerless to make decisions, (3) felt rushed and unprepared to make a treatment decision, (4) desired to follow oncologist recommendations for treatment, (5) balanced multiple competing factors during treatment decision-making, and (6) desired for ongoing engagement into their care planning. Patients reported many treatment outcomes that were important in treatment decision-making. CONCLUSIONS: Older adults with newly diagnosed AML feel devastated and in shock at their diagnosis which appears to contribute to a feeling of being overwhelmed, unprepared, and rushed into treatment decisions. Because no one factor dominated treatment decision-making for all patients, the use of strategies to elicit individual patient preferences is critical to inform treatment decisions. Interventions are needed to reduce distress and increase a sense of participation in treatment decision-making.

Using best-worst scaling to inform policy decisions in Africa: a literature review.

BACKGROUND: Stakeholder engagement in policy decision-making is critical to inform required trade-offs, especially in low-and-middle income settings, such as many African countries. Discrete-choice experiments are now commonly used to engage stakeholders in policy decisions, but other methods such as best-worst scaling (BWS), a theory-driven prioritization technique, could be equally important. We sought to document and explore applications of BWS to assess stakeholder priorities in the African context to bring attention to BWS as a method and to assess how and why it is being used to inform policy. METHODS: We conducted a literature review of published applications of BWS for prioritization in Africa. RESULTS: Our study identified 35 studies, with the majority published in the past four years. BWS has most commonly been used in agriculture (43%) and health (34%), although its broad applicability is demonstrated through use in fields influencing social and economic determinants of health, including business, environment, and transportation. Published studies from eastern, western, southern, and northern Africa include a broad range of sample sizes, design choices, and analytical approaches. Most studies are of high quality and high policy relevance. Several studies cited benefits of using BWS, with many of those citing potential limitations rather than observed limitations in their study. CONCLUSIONS: Growing use of the method across the African continent demonstrates its feasibility and utility, recommending it for consideration among researchers, program implementers, policy makers, and funders when conducting preference research to influence policy and improve health systems. REGISTRATION: The review was registered on PROSPERO (CRD42020209745).

Prioritization of ethical concerns regarding HIV molecular epidemiology by public health practitioners and researchers.

BACKGROUND: HIV molecular epidemiology (HIV ME) can support the early detection of emerging clusters of new HIV infections by combining HIV sequence data routinely obtained during the clinical treatment of people living with HIV with behavioral, geographic, and sociodemographic information. While information about emerging clusters promises to facilitate HIV prevention and treatment efforts, the use of this data also raises several ethical concerns. We sought to assess how those working on the frontlines of HIV ME, specifically public health practitioners (PHPs) and researchers, prioritized these issues. METHODS: Ethical issues were identified through literature review, qualitative in-depth interviews, and stakeholder engagement. PHPs and researchers using HIV ME prioritized the issues using best-worst scaling (BWS). A balanced incomplete block design was used to generate 11 choice tasks each consisting of a sub-set of 5 ethical concerns. In each task, respondents were asked to assess the most and least concerning issue. Data were analyzed using conditional logit, with a Swait-Louviere test of poolability. Latent class analysis was then used to explore preference heterogeneity. RESULTS: In total, 57 respondents completed the BWS experiment May-June 2023 with the Swait-Louviere test indicating that researchers and PHPs could be pooled (p = 0.512). Latent class analysis identified two classes, those highlighting "Harms" (n = 29) (prioritizing concerns about potential risk of legal prosecution, individual harm, and group stigma) and those highlighting "Utility" (n = 28) (prioritizing concerns about limited evidence, resource allocation, non-disclosure of data use for HIV ME, and the potential to infer the directionality of HIV transmission). There were no differences in the characteristics of members across classes. CONCLUSIONS: The ethical issues of HIV ME vary in importance among stakeholders, reflecting different perspectives on the potential impact and usefulness of the data. Knowing these differences exist can directly inform the focus of future deliberations about the policies and practices of HIV ME in the United States.

Increasing Access to Buprenorphine for Opioid Use Disorder in Primary Care: an Assessment of Provider Incentives.

BACKGROUND: Primary care providers (PCPs) are essential to increasing access to office-based buprenorphine medication treatment for opioid use disorder (B-MOUD). Barriers to B-MOUD prescribing are well-documented, but there is little information regarding incentives to overcome these barriers. OBJECTIVE: To identify optimal incentives for PCPs to promote B-MOUD prescribing and compare incentive preferences across provider and practice characteristics. DESIGN: We surveyed PCPs using best-worst scaling (BWS) to prioritize seven potential incentives for B-MOUD prescribing (monetary compensation, paid vacation, protected time, professional development, reduced workload, service recognition, clinical resources). We then used a direct elicitation approach to determine preferred incentive levels (e.g., monetary thresholds) and types (e.g., specific clinical resources). PARTICIPANTS: Primary care physicians and advanced practice providers (APPs) at a large Department of Veterans Affairs healthcare system. MAIN MEASURES: B-MOUD prescribing incentive preferences and relative preference levels using descriptive statistics and conditional logistic regression with relative importance scale transformation (coefficients sum to 100, higher coefficient=greater importance). KEY RESULTS: Fifty-three PCPs responded (73% response), including 47% APPs and 36% from community-based clinics. Reduced workload (relative importance score=26.8), protected time (18.7), and clinical resources (16.8) were significantly more preferred (Ps < 0.001) than professional development (10.5), paid vacation (10.3), or service recognition (1.5). Relative importance of monetary compensation varied between physicians (12.6) and APPs (17.5) and between PCPs located at a medical center (11.4) versus community clinic (22.3). APPs were more responsive than physicians to compensation increases of $5000 and $12,000 but less responsive to $25,000; trends were similar for medical center versus community clinic PCPs. The most frequently requested clinical resource was on-demand consult access to an addiction specialist. CONCLUSIONS: Interventions promoting workload reductions, protected time, and clinical resources could increase access to B-MOUD in primary care. Monetary incentives may be additionally needed to improve B-MOUD prescribing among APPs and within community clinics.

A Roadmap for Increasing the Usefulness and Impact of Patient-Preference Studies in Decision Making in Health: A Good Practices Report of an ISPOR Task Force.

Many qualitative and quantitative methods are readily available to study patient preferences in health. These methods are now being used to inform a wide variety of decisions, and there is a growing body of evidence showing studies of patient preferences can be used for decision making in a wide variety of contexts. This ISPOR Task Force report synthesizes current good practices for increasing the usefulness and impact of patient-preference studies in decision making. We provide the ISPOR Roadmap for Patient Preferences in Decision Making that invites patient-preference researchers to work with decision makers, patients and patient groups, and other stakeholders to ensure that studies are useful and impactful. The ISPOR Roadmap consists of 5 key elements: (1) context, (2) purpose, (3) population, (4) method, and (5) impact. In this report, we define these 5 elements and provide good practices on how patient-preference researchers and others can actively contribute to increasing the usefulness and impact of patient-preference studies in decision making. We also present a set of key questions that can support researchers and other stakeholders (eg, funders, reviewers, readers) to assess efforts that promote the ongoing impact (both intended and unintended) of a particular preference study and additional studies in the future.

Diagnostic experiences of Duchenne families and their preferences for newborn screening: A mixed-methods study.

Duchenne muscular dystrophy is the most common form of muscular dystrophy diagnosed in childhood but is not routinely screened for prenatally or at birth in the United States. We sought to characterize the diagnostic experiences of families and describe their preferences for newborn screening (NBS). We conducted a registry-based survey of families with Duchenne and Becker muscular dystrophy that included open- and closed-ended questions regarding the journey to a diagnosis, preferences for when to learn of a diagnosis, and how knowledge of a diagnosis would impact life decisions. Open-ended responses were analyzed thematically, and closed-ended responses were analyzed descriptively. Sixty-five families completed the survey. The average ages of first concern and diagnosis were 2 and 4 years, respectively. One-third of families (30%) indicated that they would prefer to receive a diagnosis in the newborn period irrespective of treatment options available, and nearly all of the remaining families (93%) indicated that they would want to learn about a diagnosis if there were treatments that worked well during the newborn period. All families (100%) indicated that a diagnosis in the newborn period would impact life decisions. We identified three overarching themes, which described the stages of the diagnostic journey, including having concerns about the child, seeking answers, and receiving the diagnosis. NBS can facilitate improved health outcomes through early access to care, and inform families on major health and nonhealth decisions. The preferences and experiences of families and other stakeholders should be considered when determining the potential value and benefit of expanding NBS programs.

Assessment of Fall-Related Emergency Medical Service Calls and Transports after a Community-Level Fall-Prevention Initiative.

Background: Getting effective fall prevention into the homes of medically and physically vulnerable individuals is a critical public health challenge. Community paramedicine is emerging globally as a new model of care that allows emergency medical service units to evaluate and treat patients in non-emergency contexts for prevention efforts and chronic care management. The promise of community paramedicine as a delivery system for fall prevention that scales to community-level improvements in outcomes is compelling but untested.Objective: To study the impact of a community paramedic program's optimization of a fall prevention system entailing a clinical pathway and learning health system (called Community-FIT) on community-level fall-related emergency medical service utilization rates.Methods: We used an implementation science framework and quality improvement methods to design and optimize a fall prevention model of care that can be embedded within community paramedic operations. The model was implemented and optimized in an emergency medical service agency servicing a Midwestern city in the United States (∼35,000 residents). Primary outcome measures included relative risk reduction in the number of community-level fall-related 9-1-1 calls and fall-related hospital transports. Interrupted time series analysis was used to evaluate relative risk reduction from a 12-month baseline period (September 2016 - August 2017) to a 12-month post-implementation period (September 2018-August 2019).Results: Community paramedic home visits increased from 25 in 2017, to 236 in 2018, to 517 in 2019, indicating a large increase in the number of households that benefited from the efforts. A relative risk reduction of 0.66 (95% [CI] 0.53, 0.76) in the number of fall calls and 0.63 (95% [CI] 0.46, 0.75) in the number of fall-related calls resulting in transports to the hospital were observed.Conclusions: Community-FIT may offer a powerful mechanism for community paramedics to reduce fall-related 9-1-1 calls and transports to hospitals that can be implemented in emergency medical agencies across the country.

Guidelines for Artificial Intelligence in Medicine: Literature Review and Content Analysis of Frameworks.

BACKGROUND: Artificial intelligence (AI) is rapidly expanding in medicine despite a lack of consensus on its application and evaluation. OBJECTIVE: We sought to identify current frameworks guiding the application and evaluation of AI for predictive analytics in medicine and to describe the content of these frameworks. We also assessed what stages along the AI translational spectrum (ie, AI development, reporting, evaluation, implementation, and surveillance) the content of each framework has been discussed. METHODS: We performed a literature review of frameworks regarding the oversight of AI in medicine. The search included key topics such as "artificial intelligence," "machine learning," "guidance as topic," and "translational science," and spanned the time period 2014-2022. Documents were included if they provided generalizable guidance regarding the use or evaluation of AI in medicine. Included frameworks are summarized descriptively and were subjected to content analysis. A novel evaluation matrix was developed and applied to appraise the frameworks' coverage of content areas across translational stages. RESULTS: Fourteen frameworks are featured in the review, including six frameworks that provide descriptive guidance and eight that provide reporting checklists for medical applications of AI. Content analysis revealed five considerations related to the oversight of AI in medicine across frameworks: transparency, reproducibility, ethics, effectiveness, and engagement. All frameworks include discussions regarding transparency, reproducibility, ethics, and effectiveness, while only half of the frameworks discuss engagement. The evaluation matrix revealed that frameworks were most likely to report AI considerations for the translational stage of development and were least likely to report considerations for the translational stage of surveillance. CONCLUSIONS: Existing frameworks for the application and evaluation of AI in medicine notably offer less input on the role of engagement in oversight and regarding the translational stage of surveillance. Identifying and optimizing strategies for engagement are essential to ensure that AI can meaningfully benefit patients and other end users.

Patient-centered quality measurement for opioid use disorder: Development of a taxonomy to address gaps in research and practice.

Background: Evidence-based treatment is provided infrequently and inconsistently to patients with opioid use disorder (OUD). Treatment guidelines call for high-quality, patient-centered care that meets individual preferences and needs, but it is unclear whether current quality measures address individualized aspects of care and whether measures of patient-centered OUD care are supported by evidence. Methods: We conducted an environmental scan of OUD care quality to (1) evaluate patient-centeredness in current OUD quality measures endorsed by national agencies and in national OUD treatment guidelines; and (2) review literature evidence for patient-centered care in OUD diagnosis and management, including gaps in current guidelines, performance data, and quality measures. We then synthesized these findings to develop a new quality measurement taxonomy that incorporates patient-centered aspects of care and identifies priority areas for future research and quality measure development. Results: Across 31 endorsed OUD quality measures, only two measures of patient experience incorporated patient preferences and needs, while national guidelines emphasized providing patient-centered care. Among 689 articles reviewed, evidence varied for practices of patient-centered care. Many practices were supported by guidelines and substantial evidence, while others lacked evidence despite guideline support. Our synthesis of findings resulted in EQuIITable Care, a taxonomy comprised of six classifications: (1) patient Experience and engagement, (2) Quality of life; (3) Identification of patient risks; (4) Interventions to mitigate patient risks; (5) Treatment; and (6) Care coordination and navigation. Conclusions: Current quality measurement for OUD lacks patient-centeredness. EQuIITable Care for OUD provides a roadmap to develop measures of patient-centered care for OUD.

Prioritizing the worries of AML patients: Quantifying patient experience using best-worst scaling.

CONTEXT: Although patients with acute myeloid leukemia (AML) experience significant toxicities and poor outcomes, few studies have quantified patients' experience. METHODS: A community-centered approach was used to develop an AML-specific best-worst scaling (BWS) instrument involving 13 items in four domains (psychological, physical, decision-making, and treatment delivery) to quantify patient worry. A survey of patients and caregivers was conducted using the instrument. Data were analyzed using conditional logistic regression. RESULTS: The survey was completed by 832 patients and 237 caregivers. Patients were predominantly white (88%), married/partnered (72%), and in remission (95%). The median age was 55 years (range: 19-87). Median time since diagnosis was 8 years (range: 1-40). Patients worried most about "the possibility of dying from AML" (BWS score = 15.5, confidence interval [CI] [14.2-16.7]) and "long-term side effects of treatments" (14.0, CI [12.9-15.2]). Patients found these items more than twice as worrisome as all items within the domains of care delivery and decision-making. Patients were least worried about "communicating openly with doctors" (2.50, CI [1.97-3.04]) and "having access to the best medical care" (3.90, CI [3.28-4.61]). Caregiver reports were highly correlated to patients' (Spearman's ρ = 0.89) though noted significantly more worry about the possibility of dying and spending time in the hospital. CONCLUSION: This large convenience sample demonstrates that AML patients have two principal worries: dying from their disease and suffering long-term side effects from treatment. To better foster patient-centered care, therapeutic decision-making and drug development should reflect the importance of both potential outcomes. Further work should explore interventions to address these worries.

Patient experience and quality of life during neoadjuvant therapy for pancreatic cancer: a systematic review and study protocol.

PURPOSE: Neoadjuvant therapy (NT) is increasingly being offered to patients with pancreatic ductal adenocarcinoma (PDAC) prior to surgical resection. However, the experience and quality of life (QOL) of patients undergoing NT are poorly understood. METHODS: A systematic review of the Cinahl, Embase, Medline, Pubmed, Scopus, and Web of Science databases was conducted to evaluate the available literature pertaining to the experience and QOL of patient's undergoing NT for PDAC. RESULTS: Among 6041 articles screened, only six met criteria for full-text review including three prospective clinical trials of NT with QOL secondary endpoints. Overall, global QOL during or following NT did not significantly change from baseline. Pain scores seemed to improve during NT while the impact of NT on physical functioning varied across studies. No studies were identified evaluating other aspects of the patient experience. CONCLUSION: Although NT appears to have a minor impact on the QOL of patients with PDAC, this systematic review identified significant evidence gaps in the literature. A protocol of a prospective observational cohort study utilizing a digital smartphone app that aims to evaluate the patient experience and longitudinal QOL of patients with PDAC undergoing NT is presented.

Do people have differing motivations for participating in a stated-preference study? Results from a latent-class analysis.

BACKGROUND: Researchers and policy makers have long suspected that people have differing, and potentially nefarious, motivations for participating in stated-preference studies such as discrete-choice experiments (DCE). While anecdotes and theories exist on why people participate in surveys, there is a paucity of evidence exploring variation in preferences for participating in stated-preference studies. METHODS: We used a DCE to estimate preferences for participating in preference research among an online survey panel sample. Preferences for the characteristics of a study to be conducted at a local hospital were assessed across five attributes (validity, relevance, bias, burden, time and payment) and described across three levels using a starring system. A D-efficient experimental design was used to construct three blocks of 12 choice tasks with two profiles each. Respondents were also asked about factors that motivated their choices. Mixed logistic regression was used to analyze the aggregate sample and latent class analysis identified segments of respondents. RESULTS: 629 respondents completed the experiment. In aggregate "study validity" was most important. Latent class results identified two segments based on underlying motivations: a quality-focused segment (76%) who focused most on validity, relevance, and bias and a convenience-focused segment (24%) who focused most on reimbursement and time. Quality-focused respondents spent more time completing the survey (p < 0.001) and were more likely to identify data quality (p < 0.01) and societal well-being (p < 0.01) as motivations to participate. CONCLUSIONS: This information can be used to better understand variability in motivations to participate in stated-preference surveys and the impact of motivations on response quality.

Projected 20- and 30-Year Outcomes for Pediatric Liver Transplant Recipients in the United States.

BACKGROUND: Observed long-term outcomes no longer reflect the survival trajectory facing pediatric liver transplant (LT) recipients today. We aimed to use national registry data and parametric models to project 20- and 30-year post-transplant outcomes for recently transplanted pediatric LT recipients. METHODS: We conducted a retrospective cohort study of 13,442 first-time pediatric (age <18) LT recipients using 1987 to 2018 Scientific Registry of Transplant Recipients data. We validated the proposed method (ie, to project long-term patient and graft survival using parametric survival models and short-term data) in 2 historic cohorts (1987-1996 and 1997-2006) and estimated long-term projections among patients transplanted between 2007 and 2018. Projections were stratified by raft type, recipient age, and indication for transplant. RESULTS: Parsimonious parametric models with Weibull distribution can be applied to post-transplant data and used to project long-term outcomes for pediatric LT recipients beyond observed data. Projected 20-year patient survival for pediatric LT recipients transplanted in 2007 to 2018 was 84.0% (95% confidence interval 81.5-85.8), compared to observed 20-year survival of 72.8% and 63.6% among those transplanted in 1997 to 2006 and 1987 to 1996, respectively. Projected 30-year survival for pediatric LT recipients in 2007 to 2018 was 80.1% (75.2-82.7), compared to projected 30-year survival of 68.6% (66.1-70.9) in the 1997 to 2006 cohort and observed 30-year survival of 57.5% in the 1987 to 1996 cohort. Twenty- and 30-year patient and graft survival varied slightly by recipient age, graft type, and indication for transplant. CONCLUSIONS: Projected long-term outcomes for recently transplanted pediatric LT recipients are excellent, reflective of substantial improvements in medical care, and informative for physician-patient education and decision making in the current era.

Syringe Service Program Utilization, Barriers, and Preferences for Design in Rural Appalachia: Differences between Men and Women Who Inject Drugs.

Background People who inject drugs (PWID) in rural areas of the United States have had limited access to syringe service programs (SSP). Rural SSP have recently surged, but accompanying research is lacking about PWID utilization, barriers, and preferences for SSP design and how those preferences vary by gender. Methods: Interviewer-administered surveys elicited information about utilization, barriers, and preferences for SSP design from 234 PWID recruited using respondent-driven sampling in Appalachian, Kentucky. Gender differences among reported barriers to utilizing SSP and preferences for program design were explored using Mantel-Haenszel chi-square tests. Results: Overall, 49% of PWID had ever utilized an SSP. The most common reasons for not utilizing an SSP were lack of awareness (23%), fear of being seen or disclosing drug use (19%), and lack of need (19%). The most preferred SSP design was located within a health department (74%) and operating during afternoon hours (66%). Men were more likely than women to prefer SSP in health departments (80% vs. 65%, p = 0.01), while more women than men preferred staffing by health department personnel (62% vs. 46%, p = 0.02). Women were less likely to favor evening hours (55% vs. 70%, p = 0.02). Fewer women wanted SSP nurses (78% vs. 90%, p = 0.01), social workers (11% vs. 24%, p = 0.01), or people who use drugs (20% vs 34%, p = 0.02) to staff SSP. Conclusions: Despite recent scale-up, SSP in Appalachia remain under-utilized. PWID were open to a range of options for SSP design and staffing, though there were variations by gender. Implementation research that identifies best strategies for tailored SSP scale-up in rural settings should be considered.

Methods for measuring patient preferences: an update and future directions.

PURPOSE OF REVIEW: The current review highlights the growing number of available methods used to measure patient preferences and discusses how this impacts preference research in rheumatology. Spurred by the growing role of preferences in regulatory decisions and drug development, researchers have begun applying preference methods to study questions beyond the clinical context. We explore these trends, provide case studies highlighting changes in measuring patient preferences, compare strengths and weaknesses of common stated-preference methods, and discuss considerations for the future use of these methods. RECENT FINDINGS: Early literature on patient preferences often mimicked clinical practice, asking whether treatment A is better or worse than treatment B for a patient. Early applications of patient preference methods in rheumatology aimed to value different attributes of treatments, but remained focused on informing clinical questions. Spurred by interest in preferences by regulatory agencies and patient-centeredness throughout the product lifecycle, there are now a wide array of methods available to measure preference. SUMMARY: Although these different preference methods have strengths and weaknesses, they serve to highlight the broad number of questions that could help rheumatology beyond the clinical context. Researchers in rheumatology now have the opportunity to better serve diverse stakeholders by considering how these methods could aid in clinical trial design, regulatory policy, and other elements of the medical product life cycle.

Expansion of the Liver Donor Supply Through Greater Use of Split-Liver Transplantation: Identifying Optimal Recipients.

The increased use of split-liver transplantation (SLT) represents a strategy to increase the supply of organs. Although outcomes after SLT and whole liver transplantation (WLT) are similar on average among pediatric recipients, we hypothesized that the relationship between graft type and outcomes may vary depending on patient, donor, and surgical characteristics. We evaluated graft survival among pediatric (<18 years) deceased donor, liver-only transplant recipients from March 2002 until December 2015 using data from the Scientific Registry of Transplant Recipients. Graft survival was assessed in a Cox proportional hazards model, with and without effect modification between graft type and donor, recipient, and surgical characteristics, to identify conditions where the risk of graft loss for SLT and WLT were similar. In a traditional multivariable model, characteristics associated with graft loss included donor age >50 years, recipient weight <10 kg, acute hepatic necrosis, autoimmune diseases, tumor, public insurance, and cold ischemia time (CIT) >8 hours. In an analysis that explored whether these characteristics modified the relationship between graft type and graft loss, many characteristics associated with loss actually had similar outcomes regardless of graft type, including weight <10 kg, acute hepatic necrosis, autoimmune diseases, and tumor. In contrast, several subgroups had worse outcomes when SLT was used, including recipient weight 10-35 kg, non-biliary atresia cholestasis, and metabolic disease. Allocation score, share type, or CIT did not modify risk of graft type and graft failure. Although one might anticipate that individuals with higher rates of graft loss would be worse candidates for SLT, data suggest that these patients actually have similar rates of graft loss. These findings can guide surgical decision making and may support policy changes that promote the increased use of SLT for specific pediatric recipients.