Racial and Ethnic Disparities in Health-Related Outcomes in Crohn's Disease: Results From the National Health and Wellness Survey.

Background: Crohn's disease (CD) is a chronic inflammatory condition affecting the entire gastrointestinal tract that is associated with significant humanistic, clinical, and economic burdens. Few studies have assessed the association between CD severity and patient-reported outcomes (PROs), healthcare resource utilization (HCRU), and medical costs; even fewer have examined differences in disease outcomes among patients of various racial/ethnic groups. Methods: In this cross-sectional study, sociodemographic data, PROs, and economic outcomes for participants with self-reported CD were collected from the National Health and Wellness Survey (2018-2020). Multivariable analyses were used to assess the association of CD severity and race/ethnicity with health-related quality of life (HRQoL), work productivity and activity impairment (WPAI), HCRU, and medical costs. Results: Analyses included 1077 participants with CD (818 non-Hispanic White, 109 non-Hispanic Black, and 150 Hispanic). Participants with self-reported moderate/severe CD reported significantly worse HRQoL and WPAI, greater HCRU, and higher medical costs than those with self-reported mild CD. Non-Hispanic Black participants reported better HRQoL and fewer healthcare provider visits than non-Hispanic White participants. There were no significant differences in PROs between non-Hispanic White and Hispanic groups. Interactions between race/ethnicity and CD severity emerged for some, but not all groups: Specifically, non-Hispanic Black participants with moderate/severe CD reported greater absenteeism and more gastroenterologist visits than non-Hispanic Black participants with mild CD. Conclusions: Participants with moderate/severe CD reported worse PROs, greater HCRU, and higher medical costs than those with mild CD. Additionally, racial/ethnic differences were found across several HCRU and economic outcomes. Further research is needed to better understand factors contributing to burden among patients with varying CD severity across racial/ethnic groups.

Burden of Wilson Disease among patients and care partners in the United States: results from a cross-sectional survey.

OBJECTIVE: This study assessed the burden of Wilson Disease (WD) among patients and care partners (WD-CPs) in the US and compared it to a US general population of adults (GPs) and care partners (GP-CPs). METHODS: This cross-sectional, self-reported survey included patients with WD and WD-CPs aged ≥18 years recruited through the Wilson Disease Association (WDA), while data for GPs and GP-CPs were obtained from the 2022 National Health and Wellness Survey. GPs and GP-CPs were propensity score matched (3:1) with WD patients and WD-CPs for demographics and health characteristics. Bivariate analysis evaluated differences in comorbidity burden and health-related outcomes of the WD cohorts compared to matched GP cohorts. RESULTS: Thirty-seven patients with WD and 53 WD-CPs completed the survey. Most patients reported some treatment burden (73.3%), experienced sleep problems (60%), and visited a healthcare provider (HCP) in the past 6 months (91.9%). Compared with matched GPs, patients with WD had a significantly higher mortality risk (p < .001) and reported greater rates of chronic liver disease, cirrhosis (both, p < .001), migraines (p = .032), non-alcoholic steatohepatitis (p = .004), sleep problems (p = .009) and HCP visits (p = .002). Most WD-CPs (75.5%) reported high burden of caring (mean ZBI-12 score, 26.5) and more negative impact on esteem than GP-CPs. CONCLUSION: This study highlights the burden of WD experienced by patients and WD-CPs, with patients experiencing high treatment burden, comorbidity burden and healthcare resource utilization, and WD-CPs experiencing high impact of caring, including impact on employment and self-esteem.

Wilson Disease (WD) is a rare genetic disorder that results from copper building up in the liver and the central nervous system. The management of WD has been consistent for the past 50 years. We surveyed patients with WD and family members of patients with WD ("care partners," hereby referred to as WD-CPs) residing in the US, to understand the burden of WD. We also used data from the 2022 National Health and Wellness Survey to compare patients with and WD-CPs to a sample of adults and care partners of adults in the general population (hereby referred to as GP and GP-CPs). The study found that the majority of the patients with WD reported some treatment burden (73.3%), experienced sleep problems (60.0%), and visited a healthcare provider (HCP) in the past 6 months (91.9%). Compared to GPs, patients with WD had a significantly higher risk of dying in the next 10 years and reported greater rates of other health conditions (chronic liver disease, cirrhosis, migraines, and non-alcoholic steatohepatitis), sleep problems, and visits to healthcare providers in the last 6 months. The majority of WD-CPs (75.5%) reported high burden of caring and more negative impact on their self-esteem than GP-CPs. Overall, this study highlights the burden of WD and suggests the need for more effective treatments that can reduce this burden.

The clinical, economic, and patient-centric burden of insomnia symptom severity in adults with major depressive disorder in the United States.

INTRODUCTION: Insomnia is prevalent in adults with major depressive disorder (MDD) and is a key diagnostic criterion of MDD; however, little is understood about the burden of insomnia symptom severity in MDD. We evaluated the relationship between insomnia symptom severity and the clinical, economic, and patient-centric burden among community-dwelling individuals with MDD. METHODS: Respondents with diagnosed depression who reported insomnia symptoms in the past 12 months (N = 4402) were identified from the 2019 United States National Health and Wellness Survey. Multivariable analyses assessed the association of Insomnia Severity Index (ISI) with health-related outcomes while controlling for sociodemographic and health characteristics. Further analyses also controlled for depression severity (9-item Patient Health Questionnaire). RESULTS: Mean ISI score was 14.3 ± 5.6. Higher ISI was associated with greater depression severity (r = .51, p < .001). After adjustments, a one-standard deviation (5.6-point) increase in ISI score was significantly associated with higher depression (rate ratio [RR] = 1.36), anxiety (RR = 1.33) and daytime sleepiness (RR = 1.16) levels, more healthcare provider (RR = 1.13) and emergency room visits (RR = 1.31), hospitalizations (RR = 1.21), work productivity and activity impairment (RRs = 1.27 and 1.23, respectively), and poorer mental and physical health-related quality of life (ß = -3.853 and -1.999, respectively) (p < .001). These findings remained statistically significant when controlling for concurrent depression severity. CONCLUSION: In adults with MDD, greater insomnia symptom severity is associated with worse health-related outcomes, which suggests the importance of addressing insomnia symptoms as a clinical target for treating MDD.

The real-world burden of adults with major depressive disorder with moderate or severe insomnia symptoms in the United States.

BACKGROUND: Although insomnia is a common core symptom of major depressive disorder (MDD), the burden of moderate-to-severe insomnia symptoms in patients with MDD is not well-understood. This study quantified the clinical, patient-centric, and economic burden of adults with MDD with moderate-to-severe insomnia symptoms (MDDIS) compared to adults with MDD with no-to-mild insomnia symptoms (other-MDD) and adults without MDD. METHODS: Data from 2019 US National Health and Wellness Survey identified adults self-reporting physician-diagnosed depression, stratified by insomnia status (MDDIS: Insomnia Severity Index [ISI] score ≥15; other-MDD: ISI score <15), and adults not reporting depression (non-MDD). Other-MDD and non-MDD were matched 2:1 to MDDIS on age/sex/race. Matched bivariate analyses examined differences in health-related outcomes by depression-insomnia status. RESULTS: Of 74,994 survey respondents, 2045 (2.7%) were classified as MDDIS, 8220 (11.0%) as other-MDD, and 59,859 (79.8%) as non-MDD. MDDIS respondents (vs other-MDD and non-MDD) reported greater depression severity, anxiety severity, daytime sleepiness, activity impairment, direct costs, and costs due to work productivity impairments, and lower mental and physical functioning (all P < 0.05). LIMITATIONS: Depression diagnosis was not based on clinical/diagnostic interview; causal relationships cannot be determined due to the cross-sectional design. CONCLUSIONS: Among US adults with MDD, presence of moderate-to-severe insomnia symptoms is associated with additional burden and notable impairments across several health outcomes versus those with MDD but no-to-minimal insomnia symptoms and general population without MDD. This study highlights the burden of MDDIS and the need for better identification and management of moderate-to-severe insomnia symptoms in adults with MDD.

Development of a Measure to Assess Attitudes Towards Nasal versus Autoinjector Glucagon Delivery Devices for Treatment of Severe Hypoglycemia.

Background: For individuals managing diabetes, the administration of glucagon for severe hypoglycemia can be lifesaving, yet, until recently, there were no easy-to-use devices for these stressful emergencies. New products have emerged to meet this need, including nasal glucagon (NG) and auto-injector glucagon (AI). This study evaluated the psychometric properties of a new measure, the Glucagon Device Attitudes Questionnaire (GDAQ), in assessing attitudes toward NG and AI from the perspectives of persons with diabetes on insulin (PWDs), caregivers, and acquaintances. Methods: Developed based on qualitative research, the GDAQ consists of 38 rating items for each device and 16 direct-elicitation of attitudes of device relative to each other. It was administered to participants via a cross-sectional online survey. Twenty-six rating items were included in principal component analysis and confirmatory factor analysis. Items comprising each factor were averaged to form scales. Additionally, 12 direct elicitation items were averaged to form an overall "Attitudes" scale. Reliability and validity analyses were conducted. Descriptive statistics were provided for the rating items not included in the factor analysis. Results: A total of 405 PWDs, 313 caregivers, and 305 acquaintances participated. Three factors were identified: "Prepared and Protected" (7 items), "Hesitation" (12 items), and "Device Perceptions by Others" (7 items); factor loadings ranged from 0.13 to 0.92, 0.50 to 0.89, and 0.16 to 0.92, respectively. Cronbach's alpha for the four scales ranged from 0.76 to 0.96. Correlations of the scales with their global item ranged from 0.30 to 0.90. The items outside of the factor analysis showed good distribution in responses and differentiation between the two devices. Discussion: This study supports the validity and reliability of the GDAQ, which successfully conceptualizes attitudes towards devices for administering glucagon among different respondent groups. Use of the GDAQ can help guide the development and testing of new glucagon drug/device combinations.

Examining the impact of excessive daytime sleepiness on utility scores in patients with obstructive sleep apnoea and/or narcolepsy in five European countries.

BACKGROUND: Excessive daytime sleepiness (EDS) is a cardinal symptom of narcolepsy and affects many patients with obstructive sleep apnoea (OSA). EDS is associated with reduced quality of life, increased accident risk, and poor workplace performance. Given the impact of EDS, the ability to predict health-related utility from sleepiness is valuable for examining the cost effectiveness of novel treatments. The aim of this study was to examine the association between EDS and EQ-5D in patients with OSA and/or narcolepsy by modelling EQ-5D utility scores from Epworth Sleepiness Scale (ESS) scores. METHODS: Data were obtained from the Europe 2016/2017 National Health and Wellness Survey, an online, general population survey, designed to represent the age and gender composition of each country's adult population. Analyses included 2,348 patients self-reporting symptomatic and diagnosed OSA (n = 2,277), narcolepsy (n = 48), or both (n = 23). Multivariable models were used to examine ESS as a predictor of EQ-5D utility while adjusting for covariates of interest. Results were validated following the National Institute for Health and Care Excellence Decision Support Unit guidelines for predictive modelling. RESULTS: Utility decreased as EDS severity increased (no EDS: 0.711 ± 0.251, mild: 0.685 ± 0.261, moderate: 0.643 ± 0.268, severe: 0.559 ± 0.323). Whereas participants with only OSA or only narcolepsy did not differ in utility, those with both conditions had lower scores (0.685 ± 0.266 and 0.627 ± 0.325 vs. 0.439 ± 0.340, respectively). Piecewise linear regression identified a single breakpoint at ESS score of 11.29. In the final model, for each point increase in ESS score, the corresponding decrease in EQ-5D utility was larger among patients with ESS scores ≥ 12 compared to patients with ESS scores ≤ 11 (model slopes: -0.0131 vs. -0.0026, respectively). Findings from the validation sample confirmed these results. CONCLUSIONS: This study demonstrates the impact of sleepiness on quality of life (QoL) and its negative impact irrespective of sleep condition (OSA or narcolepsy). The breakpoint identified is relatively consistent with the established ESS cutoff score ≥ 11, which demarcates pathological sleepiness. Furthermore, as EDS severity worsens (increases) on the ESS, the impact on QoL is greater.

Benefit-risk trade-offs in treatment choice in advanced HER2 negative breast cancer: patient and oncologist perspectives.

Aim: To evaluate which treatment attributes US patients and oncologists prioritize in HER2 negative advanced breast cancer (ABC). Methods: Preferences were assessed via a discrete choice experiment. Also, treatment goal statements were rated on an agreement scale. Results: Patients (n = 169) most valued improving overall survival (OS), followed by improving nausea and neuropathy. Oncologists (n = 117) most valued improving OS, followed by neuropathy and progression-free survival. Regarding treatment goals, oncologists (67%) perceived that patients are more focused on efficacy than quality of life; fewer patients (29%) agreed with this statement; 81% of oncologists and 51% of patients agreed that patients prefer oral treatment. Conclusion: Patients and oncologists were willing to accept increases in toxicities in exchange for efficacy improvements in HER2 negative ABC.

The goal of this study was to understand the preferences of patients and physicians for the benefits and risks associated with the treatment of advanced breast cancer that expresses normal amounts of the HER2 protein (termed as HER2 negative). Respondents completed an exercise where they compared two treatment options at a time that varied in their level of effectiveness and their potential for certain side effects and then selected the treatment option they most preferred. From this exercise, the treatment features that matter the most to patients and physicians were discovered. The most important treatment features for patients were lengthening life expectancy, decreasing the chance of experiencing nausea affecting appetite, and decreasing the chance of experiencing nerve damage involving numbness and/or pain, possibly severe, in hands and feet which may limit daily activities. The most important treatment features for physicians were lengthening life expectancy, decreasing the change of experiencing nerve damage involving numbness and/or pain, possibly severe, in hands and feet which may limit daily activities, and lengthening the time that cancer remains stable and does not worsen. Patients and physicians also rated how much they agreed with statements about their goals for treatment. While 67% of oncologists believed that their patients are more focused on killing the cancer than their quality of life, only 27% of patients were more focused on killing the cancer than their quality of life.

Patient and Caregiver Preferences for First-Line Treatments of Metastatic Non-Small Cell Lung Cancer: A Discrete Choice Experiment.

PURPOSE: The approval of immune checkpoint inhibitors for metastatic non-small-cell lung carcinomas (mNSCLC) treatment has presented more care options. Therefore, it is important to identify the benefit-risk trade-offs patients and caregivers are willing to make among potential treatment options. This study quantified the preferences of patients and caregivers for attributes of mNSCLC treatment. METHODS: Patients with mNSCLC and caregivers completed an online survey assessing preferences using a discrete choice experiment. Respondents chose between hypothetical treatment profiles, with varying levels for 7 attributes associated with first-line treatment, including overall survival (OS), progression-free survival, select adverse events (AEs), and regimen (caregivers). Hierarchical Bayesian modeling was used to estimate attribute-level preference weights. RESULTS: Patients (n = 308) and caregivers (n = 166) most valued increasing OS from 11 to 30 months, followed by decreasing the risk of a serious AE (grade 3/4) that may lead to hospitalization from 70% to 18%. These attributes were over twice as important to both sets of respondents as the other attributes measured. Patients and caregivers would accept increases in the risks of a serious AE (grade 3/4) from 18% to 70% and all grades nausea from 10% to 69% if OS increased by 16.8 and 4.0 months, respectively. The least valued attributes were all grades of pneumonitis (patients) and all grades of skin rash (caregivers). CONCLUSION: Patients and caregivers are willing to make trade-offs between efficacy and toxicity and may require up to 1.5 years of increased OS to accept a higher risk of AEs. These results can provide guidance to oncologists when engaging in shared-decision making discussions.

Diagnosis, consultation, treatment, and impact of migraine in the US: Results of the OVERCOME (US) study.

OBJECTIVE: The ObserVational survey of the Epidemiology, tReatment and Care of MigrainE (OVERCOME; United States) study is a multicohort, longitudinal web survey that assesses symptomatology, consulting, diagnosis, treatment, and impact of migraine in the United States. BACKGROUND: Regularly updating population-based views of migraine in the United States provides a method for assessing the quality of ongoing migraine care and identifying unmet needs. METHODS: The OVERCOME (US) 2018 migraine cohort involved: (I) creating a demographically representative sample of US adults using quota sampling (n = 97,478), (II) identifying people with active migraine in the past year via a validated migraine diagnostic questionnaire and/or self-reported medical diagnosis of migraine (n = 24,272), and (III) assessing consultation, diagnosis, and treatment of migraine (n = 21,143). The current manuscript evaluated whether those with low frequency episodic migraine (LFEM; 0-3 monthly headache days) differed from other categories on outcomes of interest. RESULTS: Among the migraine cohort (n = 21,143), 19,888 (94.1%) met our International Classification of Headache Disorders, 3rd edition-based case definition of migraine and 12,905 (61.0%) self-reported a medical diagnosis of migraine. Respondents' mean (SD) age was 42.2 (15.0) years; 15,697 (74.2%) were women. Having at least moderate disability was common (n = 8965; 42.4%) and around half (n = 10,783; 51.0%) had consulted a medical professional for migraine care in the past year. Only 4792 (22.7%) of respondents were currently using a triptan. Overall, 8539 (40.4%) were eligible for migraine preventive medication and 3555 (16.8%) were currently using migraine preventive medication. Those with LFEM differed from moderate and high frequency episodic migraine and chronic migraine on nearly all measures of consulting, diagnosis, and treatment. CONCLUSION: The OVERCOME (US) 2018 cohort revealed slow but steady progress in diagnosis and preventive treatment of migraine. However, despite significant impact among the population, many with migraine have unmet needs related to consulting for migraine, migraine diagnosis, and getting potentially beneficial migraine treatment. Moreover, it demonstrated the heterogeneity and varying unmet needs within episodic migraine.

Patients' and oncologists' preferences for second-line maintenance PARP inhibitor therapy in epithelial ovarian cancer.

Aim: To understand the preferences of US patients and oncologists for PARP inhibitors as second-line maintenance (2LM) for epithelial ovarian cancer. Methods: A discrete choice experiment was conducted to assess the preferences of treatment attributes. Results: The most valued attributes were risk of grade 3/4 adverse events (AEs; patients, n = 204) and progression-free survival (PFS; oncologists, n = 151). To accept a 37% increased risk of grade 3/4 AEs, PFS would need to increase by 27.9 months (patients) and 6.3 months (oncologists). The least valued attributes were dosing form/frequency (patients) and grade 3/4 anemia risk (oncologists). Conclusion: Patients' and oncologists' willingness to make benefit-risk trade-offs in the 2LM setting suggests that the PFS gains observed in selected studies of poly (ADP-ribose) polymerase inhibitors in BRCA-mutated disease are worth the toxicity risk.

Plain language summary Maintenance therapy is a treatment option intended to keep ovarian cancer from coming back or getting worse for as long as possible after responding to chemotherapy. PARP inhibitors are a new type of maintenance therapy for ovarian cancer. This study aimed to understand the patients' and physicians' preferences for the benefits and risks associated with different PARP inhibitors used as maintenance therapy for ovarian cancer. Participants were asked to compare various treatment options based on their different safety profiles, effectiveness and form of medication (e.g., three capsules by mouth once a day versus two tablets by mouth twice a day), and then choose the treatment they most preferred. Through this exercise, the treatment features that mattered most to patients and physicians were identified. The most important treatment feature for patients was decreasing the chance of experiencing a serious side effect that requires medical intervention or hospitalization. In contrast, physicians valued lengthening the time that a cancer remains stable and does not worsen. To accept a 37% higher chance of experiencing a side effect that requires medical intervention or hospitalization, patients expect their cancer to remain stable and not worsen for an additional 28 months. This was a large difference from the 6 months that the physicians would consider as acceptable. The least important treatment features for patients are the amount of pills required per dose, the form of the given medication (e.g., tablet vs capsule) and the schedule of taking the treatment. On the other hand, physicians were least concerned about lowering the risk of experiencing low blood counts that, requiring medical intervention.

Patient and Neurologist Preferences in the United States for Relapsing-Remitting Multiple Sclerosis Treatments: Findings from a Discrete Choice Experiment.

BACKGROUND AND OBJECTIVE: Relapsing-remitting multiple sclerosis (RRMS) is a chronic inflammatory disease associated with central nervous system dysfunction and accelerated brain volume loss (BVL). There exists a paucity of research examining the importance of BVL to patients and neurologists and exploring whether such preferences may differ between these two groups. This study sought to evaluate the preferences of patients and neurologists for RRMS treatments by considering benefits and risks associated with novel and common disease-modifying therapies (DMTs). PATIENTS AND METHODS: US patients diagnosed with non-highly active RRMS and US-based neurologists completed an online cross-sectional survey. A discrete choice experiment was used to assess patient and neurologist treatment preferences, with neurologists considering preferences for patients with non-highly active RRMS. Respondents chose between two treatment profiles with seven attributes identified in qualitative research: 2-year disability progression; 1-year relapse rate; rate of BVL; and risks of gastrointestinal symptoms, flu-like symptoms, infection, and life-threatening events. Attribute-level weighted preferences were estimated using a hierarchical Bayesian model. RESULTS: Analyses included 150 patients with non-highly active RRMS (mean age: 54 years) and 150 neurologists (65% in private practice). Among patients, the most important treatment attribute was reducing the rate of BVL, followed by reducing the risk of infection and risk of flu-like symptoms. In contrast, the most important treatment attribute among neurologists was reducing the risk of a life-threatening event, followed by slowing the rate of 2-year disability progression and risk of infection. CONCLUSION: The findings highlight differences in treatment preferences between US patients and neurologists for non-highly active RRMS. The importance placed by patients on slowing the rate of BVL makes this a key topic that should be covered in the shared decision-making process.

Patient and neurologist preferences in the UK for relapsing-remitting multiple sclerosis treatments: findings from a discrete choice experiment.

OBJECTIVE: To evaluate and compare patient and neurologist preferences for relapsing-remitting multiple sclerosis (RRMS) treatments with respect to benefits and risks associated with common and novel disease-modifying therapies, including brain volume loss (BVL). METHODS: Patients with non-highly-active RRMS and neurologists in the United Kingdom completed an online cross-sectional survey. Patients completed one discrete choice experiment (DCE) exercise and providers completed two, one focusing on treatment for non-highly-active RRMS and another focused on highly active RRMS. Respondents chose between two treatment profiles that varied on seven attributes identified in qualitative research: 2 year disability progression; 1 year relapse rate; rate of BVL; and risks of gastrointestinal symptoms, flu-like symptoms, infection and life-threatening event. Bayesian modeling was used to estimate attribute-level weighted preferences. RESULTS: Patients (n = 144) prioritized slowing the rate of BVL, followed by reducing risk of infection, rate of 2 year disability progression and 1 year relapse rate. For non-highly-active patients, neurologists (n = 101) prioritized slowing the rate of BVL, followed by reducing 2 year disability progression, risk of infection and 1 year relapse rate. For highly active patients, neurologists prioritized lowering the 1 year relapse rate, followed by slowing the rate of BVL and 2 year disability progression. In all three DCEs, rate of BVL was approximately twice as important as reducing the risks of flu-like symptoms, gastrointestinal symptoms and life-threatening event. CONCLUSIONS: This study highlights similarities in treatment preferences for non-highly-active RRMS among patients and neurologists and differences in neurologists' preferences for treating non-highly-active vs. highly active RRMS. This research identifies BVL as a treatment outcome that should be discussed when physicians engage in shared decision-making with patients.

Socioeconomic and humanistic burden of illness of excessive daytime sleepiness severity associated with obstructive sleep apnoea in the European Union 5.

OBJECTIVE/BACKGROUND: Evaluate the impact of excessive daytime sleepiness (EDS) severity on burden of illness among adults with obstructive sleep apnoea (OSA) in European Union 5 (EU5) countries (France, Germany, Italy, Spain, United Kingdom). PATIENTS/METHODS: This retrospective observational study used data from the 2017 EU5 National Health and Wellness Survey, a self-administered, internet-based, non-screening survey. Respondents who self-reported both having experienced OSA in the last 12 months and having had their OSA diagnosed by a physician were considered to have OSA. Respondents completed the Epworth Sleepiness Scale (ESS) and were consequently categorised into 4 groups: OSA-with-EDS (ESS >10) subdivided by EDS severity (mild [ESS = 11-12], moderate [ESS = 13-15], severe [ESS = 16-24]), and OSA-without-EDS (ESS ≤10). Bivariate and multivariable analyses examined group differences in health-related quality of life (HRQoL), work productivity and activity impairment, and health care utilisation. RESULTS: The analysis included 2008 respondents with OSA: n = 661 (32.9%) with EDS (29.5% mild, 34.5% moderate, 36.0% severe) and n = 1347 without EDS. Compared with the OSA-without-EDS group, the OSA-with-EDS subgroups generally had higher rates of obesity, depression, and other reported comorbidities. Greater severity of EDS was associated with worse self-reported HRQoL (all domains, P < 0.001) and work productivity and activity impairment (absenteeism, P = 0.031; presenteeism, overall work impairment, and non-work activity impairment, P < 0.001), as well as increased numbers of health care provider visits (P < 0.001). CONCLUSIONS: Compared to patients with OSA but without EDS, those with EDS had substantially higher socioeconomic and humanistic burden of disease, which was more profound among those reporting greater EDS.

Adult Patient Preferences for Long-Acting ADHD Treatments: A Discrete Choice Experiment.

BACKGROUND AND OBJECTIVE: Treatment for attention deficit hyperactivity disorder (ADHD) requires a multifaceted approach including psychosocial interventions and pharmacological treatment. This study evaluates preferences for specific attributes associated with different long-acting stimulant treatment among US adults with ADHD. METHODS: Patients completed an online, cross-sectional survey, incorporating a discrete choice experiment to assess preferences for attributes. RESULTS: Analyses included 200 adults with ADHD (mean age 33.0 years; 60% self-reporting moderate severity); the mean (SD) Adult ADHD Self-Report Scale-v1.1 score was 45.9 (12.4). Overall, patients valued speed of onset most and risk of rebound least. Three population groups with distinct preferences were identified: side effect-driven (n=69, 35%), quick onset-driven (n=47, 24%) and quick onset and long duration-driven (n=84, 42%). CONCLUSION: This study shows differences in how adults with ADHD value and assess benefit-risk trade-offs when considering the desired attributes of stimulant treatments, highlighting the importance of patient-physician shared decision-making to optimize the desired benefits of individualized treatment.

Demographic and clinical characteristics of prevention-eligible patients with migraine in the US: a linked national survey and administrative claims database study.

OBJECTIVE: To characterize burden of migraine in prevention-eligible patients compared with prevention non-eligible patients in the United States (US). Receipt of preventive therapy was also examined among prevention-eligible patients. METHODS: This retrospective study utilized data from the 2017 US National Health and Wellness Survey linked with medical and pharmacy claims. Patients aged ≥18 years who self-reported experiencing migraine and had confirmed evidence of migraine (≥1 medical or pharmacy claim) were included. Prevention eligibility was based on number of headache days in the past 30 days (prevention-eligible: ≥4 and prevention non-eligible: <4). Descriptive statistics summarized study variables; bivariate and multivariable analyses were conducted to examine the association of prevention-eligibility status with outcomes. RESULTS: Analyses included 450 patients, 291 (65%) prevention-eligible, and of these 56 (19%) received preventive therapy. Overall, patients were 42.98 ± 14.51 years old; 84% were female. Prevention-eligible patients reported significantly more migraine headache days in the past 6 months (29.27 ± 37.96 vs. 8.61 ± 7.88), had lower mental component summary scores (35.80 ± 2.73 vs. 37.90 ± 2.96), and more presenteeism (47.30 ± 2.98% vs. 37.90 ± 2.60%), overall work impairment (46.30 ± 2.87% vs. 37.90 ± 2.55%) and activity days missed due to migraine (8.16 ± 3.05 vs. 3.82 ± 1.58) than prevention non-eligible patients (all p<.001). Prevention-eligible patients receiving preventive therapy reported more migraine headache days during the past month (9.21 ± 7.99 vs. 6.06 ± 7.10; p=.002) and activity days lost due to migraine (18.39 ± 28.08 vs. 10.69 ± 21.43, p=.015) than those not receiving preventive therapy. CONCLUSIONS: Prevention-eligible patients experience greater burden due to migraine, including more headache days, worse health-related quality-of-life, and greater work and activity impairment than prevention non-eligible patients.

Determinants and impact of physical impairment in patient-reported outcomes among older patients with type 2 diabetes mellitus in Japan.

OBJECTIVE: To investigate the predictive factors associated with physical impairment among older patients with type 2 diabetes mellitus (T2DM) in Japan and to examine the potential impact of physical impairment on patient-reported health outcomes in this population. METHODS: A cross-sectional analysis was conducted using patient-reported data from the 2012-2014 Japan National Health and Wellness Survey. Physical impairment was measured using the Physical Component Summary (PCS) score of the Short-Form 36-Item Health Survey (SF-36) three-component model (using Japanese norms). Older T2DM patients (≥65 years old; n = 1511) were dichotomized into physically impaired (PCS ≤ 25th percentile; n = 378) and non-physically impaired (PCS > 25th percentile; n = 1133). Work productivity (absenteeism, presenteeism and overall work impairment), activity impairment and healthcare resource utilization were compared between these groups. RESULTS: Age, female sex, low and high body mass index (BMI), diabetes-related complications, cardiovascular events, unawareness of having hypoglycemic events in the past 3 months, and lack of regular exercise were significant factors associated with physical impairment in multivariable analysis. The physically impaired group reported significantly more regular outpatient visits (13.48 vs. 10.16, respectively, p < .001), 1% or greater absenteeism (16.7% vs. 4.1%, p = .005), greater presenteeism (27.8% vs. 12.2%, p = .001), overall work impairment (30.0% vs. 13.0%, p = .001) and overall activity impairment (39.5% vs. 17.2%, p < .001) than the non-physically-impaired group after adjusting for covariates. CONCLUSIONS: This study identified age, BMI, diabetes-related comorbidities, history of cardiovascular events and lack of exercise as key predictors associated with physical impairment in older patients with T2DM in Japan, which predicted low work productivity as well as activity impairment. This study provides support that physical impairment in patients with T2DM may lead to low work productivity and activity impairment.Supplemental data for this article is available online at https://doi.org/10.1080/03007995.2020.1846170.

Real-world treatment patterns and outcomes in non-transplant newly diagnosed multiple Myeloma in France, Germany, Italy, and the United Kingdom.

OBJECTIVES: The treatment paradigm in newly diagnosed multiple myeloma (NDMM) is evolving toward individualized, risk-directed, and longer duration of therapy (DOT). The objective of this study was to describe treatment patterns and outcomes in non-transplant NDMM in four European countries. METHODS: This retrospective chart review included adults with NDMM diagnosed between January 1, 2012, and December 31, 2013 (early cohort), or April 1, 2016, and March 31, 2017 (recent cohort). RESULTS: Among 836 patients, molecular testing was performed in 21% and 35% patients of early vs recent cohorts; proteasome inhibitor (PI)/alkylator combinations were the principal first-line (1 L) therapy (39% vs 43%). Use of immunomodulatory drug (IMID)/alkylator combinations declined from early to recent cohort (26% vs 13%) but IMID (7% vs 16%) use increased. Few patients (5%) received 1 L maintenance therapy. Two-thirds of patients were treated with a fixed duration intent, with a median 7-month 1 L DOT and progression-free survival (PFS) of 32.8 months in the early cohort. Both 1 L DOT and PFS were longer with oral compared to injectable regimens. CONCLUSIONS: Although frontline treatment patterns changed significantly, 1 L DOT is short. The uptake of molecular testing and 1 L maintenance is low. These results highlight areas of unmet need in NDMM.

Perceptions About Glucagon Delivery Devices for Severe Hypoglycemia: Qualitative Research With Patients, Caregivers, and Acquaintances.

PURPOSE: People with diabetes taking insulin are at risk of severe hypoglycemia, an unpredictable, life-threatening event that requires assistance from others. Outside the clinical setting, glucagon is indicated for the treatment of hypoglycemia. However, there is significant unmet medical need to improve successful administration of glucagon by caregivers and acquaintances. This study assesses perceptions about glucagon delivery and potential effects of 2 glucagon delivery devices for severe hypoglycemia. METHODS: Qualitative interviews were conducted with people with diabetes (patients), caregivers, and acquaintances from a general population panel composed of individuals across the United States who have agreed to be recruited for research studies. Participants were recruited via email with a link to an online screener to determine eligibility. Experienced qualitative researchers conducted telephone interviews after a semistructured discussion guide, developed by the authors, that focused on aspirational device features and perceptions about the 2 devices: nasal glucagon and autoinjector glucagon; visuals of the instructions for use were displayed. Verbatim transcripts were developed, and a qualitative analysis software program, MaxQDA, was used to code responses and themes that emerged from the data. FINDINGS: A total of 45 (15 patients, 15 caregivers, and 15 acquaintances) interviews were conducted (mean ages, 55, 40, and 51, respectively). The most frequently spontaneously identified aspirational features for a new glucagon device were ease of use (29 [64%]), including being uncomplicated, premixed/ready to use, and ability to use quickly; small/easy to carry (9 [20%]); needle-free/no long needles (8 [18%]); and easy instructions (4 [9%]). In general, participants indicated that having a glucagon delivery device on hand would make them feel prepared, protected, and confident that others could assist in the event of severe hypoglycemia. More participants across all subgroups preferred nasal glucagon versus autoinjector glucagon (33 [73%] vs 12 [27%]). Favorable comments about nasal glucagon included that it appeared to be easy to carry, easy to use, and lacked a needle, that one does not need to remove clothing to use it, and that others likely would be more comfortable using it. Favorable comments about autoinjector glucagon included that it was familiar as a rescue device and that patients felt confident that the full dose would be delivered with it. There may be more hesitation using autoinjector glucagon versus nasal glucagon because of anxiety about needles and locating an injection site. Participants indicated that they would feel more comfortable socially using nasal glucagon because it was viewed as more discreet and less embarrassing than using autoinjector glucagon; it was also considered less traumatic for use in children. IMPLICATIONS: This research suggests that patients with diabetes, caregivers, and acquaintances prefer a device that is simple, compact, and ready to use. Nasal glucagon was generally preferred over autoinjector glucagon primarily because it lacks a needle and it appears to be less complicated.