Patient Preferences for Adjuvant Treatment in Muscle-Invasive Urothelial Carcinoma: A Multi-Country Discrete Choice Experiment.

Purpose: The evolving treatment landscape in muscle-invasive urothelial carcinoma creates challenges for clinicians and patients in selecting the most appropriate therapy. Here, we aimed to understand adjuvant treatment preferences among patients with muscle-invasive urothelial carcinoma who underwent radical resection, including tradeoffs between efficacy outcomes and toxicity risks. Patients and Methods: An observational, cross-sectional study utilizing a discrete choice experiment was conducted across the United States, United Kingdom, Canada, France, and Germany via a web-based survey. Patients ≥18 years of age who self-reported as having been diagnosed with muscle-invasive urothelial carcinoma were included. Patients indicated their preferences between hypothetical treatment profiles varying in eight attributes relating to efficacy, regimen, and side effects. Preference weights were estimated using hierarchical Bayesian logistic regression; relative attribute importance estimates were calculated. Results: Overall, 207 patients were included (age ≥56 years, 65.7%; male, 54.1%). Patients chose adjuvant treatment 91.2% of the time vs no treatment. Prolonging overall survival from 25 to 78 months was most important, followed by reducing serious side effect risks. Increasing disease-free survival from 12 to 24 months was more important than decreasing risks of fatigue from 54% to 15% and nausea from 53% to 7%. Treatment with the shortest dosing regimen was more important for patients who received neoadjuvant chemotherapy vs patients who did not receive neoadjuvant chemotherapy; prolonging overall survival was more important than reducing the risk of a serious side effect in non-US patients; the opposite was found in the United States. Conclusion: Patients with muscle-invasive urothelial carcinoma who underwent radical resection preferred adjuvant treatment over no treatment regardless of side effects. Patients prioritized overall survival improvements followed by a reduced side effect profile.

A global neuronopathic gaucher disease registry (GARDIAN): a patient-led initiative.

BACKGROUND: Gaucher disease (GD) is a rare autosomal recessive lysosomal storage disorder. GD types 2 and 3 are known as neuronopathic Gaucher disease (nGD) because they have brain involvement that progresses over time. Implementing a systematic approach to the collection of real-world clinical and patient-relevant outcomes data in nGD presents an opportunity to fill critical knowledge gaps and ultimately help healthcare providers in the management of this patient population. This paper summarizes the development of a patient-initiated Gaucher Registry for Development Innovation and Analysis of Neuronopathic Disease (GARDIAN). METHODS: The International Gaucher Alliance led the GARDIAN planning, including governance, scope, stakeholder involvement, platform, and reporting. Registry element input was determined in a series of meetings with clinical experts, patients, and caregivers, who identified key clinical variables and the draft content of nGD patient-reported outcomes (PRO) and observer-reported outcomes (ObsRO) focusing on symptoms, patient physical and emotional functioning. These were then tested in cognitive interviews with patients with nGD (> 12 years of age) and caregivers. RESULTS: Core registry data elements (n = 138) were identified by seven global clinical experts from Egypt, Germany, Israel, Japan, United Kingdom (UK), and United State (US) and reviewed via online Delphi method by 14 additional clinicians with experience of nGD from six countries and three pharmaceutical representatives. The elements were consistent with those identified via interviews with 10 patients/caregivers with nGD from Japan, Sweden, UK, and US. Key domains identified were demographics, diagnostic information, health status, clinical symptomatology, laboratory testing, treatment, healthcare resource utilization, aids/home improvements, and patient/caregiver burden and quality of life, specifically physical functioning, self-care, daily and social activities, emotional impacts, support services, and caregiver-specific impacts. Nine caregivers and six patients from the US, UK, China, Mexico, Egypt, and Japan participated in the cognitive interviews that informed revisions to ensure that all items are understandable and interpreted as intended. CONCLUSIONS: The comprehensive set of clinical and patient relevant outcomes data, developed collaboratively among all stakeholders, to be reported using GARDIAN will bridge the many gaps in the understanding of nGD and align with regulatory frameworks on real-world data needs.

Outcomes and Factors Associated with Insufficient Effectiveness of Acute Treatments of Migraine in Japan: Results of the ObserVational survey of the Epidemiology, tReatment, and Care Of MigrainE (OVERCOME [Japan]) Study.

BACKGROUND: Knowledge of patient outcomes and treatment effectiveness associated with acute migraine treatments in Japan is lacking. OBJECTIVE: To describe patient-reported outcomes (PROs) and treatment effectiveness in three acute treatment groups from OVERCOME (Japan): over-the-counter (OTC) only, prescription nonsteroidal anti-inflammatory drugs/acetaminophen (Rx-NSAIDs/ACE) only, and triptans. METHODS: OVERCOME (Japan) was an observational, cross-sectional, population-based web survey of people with migraine (July-September 2020). PROs, including the Migraine-Specific Quality of Life Questionnaire (MSQ), Migraine Interictal Burden Scale (MIBS-4), Migraine Disability Assessment (MIDAS), and Work Productivity and Activity Impairment Questionnaire: Migraine (WPAI-M), were compared pairwise between treatment groups. Logistic regression was used to examine treatment effectiveness. RESULTS: The analysis included 9075 survey respondents (OTC only: n = 5791; Rx-NSAIDs/ACE only: n = 751; triptans: n = 2533). Triptan users reported the lowest MSQ scores, most severe disability (MIDAS: 20.7% versus 6.3% and 11.6%) and severe interictal burden (MIBS-4: 50.1% versus 21.2% and 19.8%), and greatest work impairment (WPAI-M: 50.4% versus 32.2% and 30.8%) compared with the OTC and Rx-NSAIDs/ACE groups, respectively. Treatment effectiveness was very poor-to-poor for 60.9%, 43.1%, and 47.6% of the triptan, OTC, and Rx-NSAIDs/ACE groups, respectively. Severe interictal burden was significantly associated with insufficient treatment effectiveness (odds ratios, severe versus no burden: 0.47 [95% confidence interval: 0.40-0.54], 0.56 [0.35-0.89], and 0.41 [0.32-0.52], for the OTC, Rx-NSAIDs/ACE, and triptan groups, respectively). CONCLUSION: People with high migraine burden used triptans for acute treatment, but many reported poor treatment effectiveness. Education may be required to promote better treatments, including earlier introduction of migraine-specific acute and preventive medications.

Prevalence of alopecia areata in Japan: Estimates from a nationally representative sample.

Data on the prevalence of alopecia areata (AA) in Japan is limited and the epidemiology of the disease there is not well understood; therefore, it is critical to examine the prevalence and severity of AA in Japan to inform the need for future treatments and research. A cross-sectional, web-based survey was conducted in Japan from January through March 2021. A total of 45 006 participants were identified through general population survey panels and asked about their experience with AA and hair loss. The Alopecia Assessment Tool and the Scalp Hair Assessment PROTM were adopted to screen for history of AA and assess disease severity, respectively. Eligible participants submitted photos of their scalp, which were reviewed by three board-certified dermatologists to evaluate the presence and severity of AA. Prevalence and severity estimates were calculated using participants' self-reported data and verified through the dermatologists' assessments. The participant-reported point prevalence of AA was 2.18%. The adjusted point prevalence following physician adjudication using participant-submitted photos was 1.45%. Topical corticosteroids were the most commonly used treatments, with 34.6% of participants diagnosed with AA reported having ever used them. Participants also reported negative impacts on their mood (70.2%), self-esteem (55.8%), and social interactions (48.9%). Despite the social and emotional impact of hair loss, more than one third of those reporting a physician diagnosis of AA were not currently seeking treatment. The current study identified an estimated prevalence of AA in Japan between 1.45% and 2.18% based on the survey results and physician-adjudication of those findings. Considering the impactful psychological burden of AA, the survey results showing that 38.90% of surveyed patients do not currently seek treatment may indicate an unmet need for remedies.

Potential Unmet Needs in Acute Treatment of Migraine in Japan: Results of the OVERCOME (Japan) Study.

INTRODUCTION: Using data from the ObserVational survey of the Epidemiology, tReatment, and Care Of MigrainE study in Japan (OVERCOME [Japan]), we describe the current status of the acute treatment of migraine in Japan. METHODS: OVERCOME (Japan) was a cross-sectional, observational, population-based web survey of people with migraine in Japan (met modified International Classification of Headache Disorders criteria or had a physician diagnosis of migraine) conducted between July and September 2020. Respondents reported current acute medication use and effectiveness (assessed using the Migraine Treatment Optimization Questionnaire [mTOQ-4]). Cardiovascular history and risk factors of the respondents were also recorded. Potential unmet acute treatment needs were defined as insufficient effect of current acute treatments (mTOQ-4 score ≤ 5), a history of oral triptan use (and not currently taking any triptan), potential contraindications to triptans due to cardiovascular comorbidities, and/or cardiovascular risk factors. RESULTS: In total, 17,071 people with migraine in Japan completed the survey; 14,869 (87.1%) of these were currently using acute treatments. Poor effectiveness of current acute treatment was reported by 7170 respondents (42.0%), 900 respondents (5.3%) were former triptan users, 1759 (10.3%) had contraindications to triptans, and 9026 (52.9%) reported at least one cardiovascular risk factor. Overall, 12,649 (74.1%) of OVERCOME (Japan) respondents were categorized into one or more of these groups and were considered to have potential unmet acute treatment needs. CONCLUSION: Almost three-quarters of people with migraine in Japan may have potential unmet needs for acute treatment of migraine. There are substantial opportunities for improving care for people with migraine in Japan, including prescription of novel acute medications.

A global perspective in second-line treatment patterns for patients with advanced esophageal squamous cell carcinoma.

BACKGROUND: Esophageal cancer is a highly prevalent cancer associated with low survival, especially among those with advanced disease. Second-line (2L) treatment patterns and related clinical outcomes of patients with advanced esophageal squamous cell carcinoma (advESCC) treated in routine clinical care were examined globally and regionally. METHODS: A retrospective, noninterventional study collected physician-provided chart data of patients aged ≥20 years receiving either 2L active systemic therapy or BSC following first-line active therapy for advESCC from 11 countries in Asian and Western regions (September-October 2018). Bivariate analyses examined treatment and outcomes by region. RESULTS: AdvESCC patients (Asia = 192; West = 195) were examined, of which 58.1% (Asia n = 101; West n = 124) received active systemic therapy. While regional differences in tumor classification and staging at diagnosis were observed with less advanced tumors in Asia, no regional differences for these characteristics at 2L initiation were reported. Both taxane- and nontaxane-based therapies were used as 2L therapy among Asian and Western patients, although more western than Asian patients received immuno- or targeted therapies (17.0% vs. 3.0%; p = 0.001). Alopecia (10.7%), neutropenia (9.3%), and fatigue (9.3%) were the most-commonly reported adverse events (AEs) in both regions. Significantly higher 2L AE-related emergency room visits (Asia = 22.5% vs. West = 8.0%; p < 0.001) and hospitalizations (Asia = 25.9 ± 31.2 vs. West = 4.7 ± 7.0, p < 0.001) were observed in Asian than in Western patients. No regional differences were reported for response to 2L treatment or the percent of patients who received third-line treatment/died. CONCLUSIONS: While regional variations were observed throughout the course of a patient's advESCC journey, disease response and treatment outcomes were similar.

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.

Burden of Migraine in Japan: Results of the ObserVational Survey of the Epidemiology, tReatment, and Care Of MigrainE (OVERCOME [Japan]) Study.

INTRODUCTION: The ObserVational survey of the Epidemiology, tReatment, and Care Of MigrainE study in Japan (OVERCOME [Japan]) assessed the impact and burden of migraine in Japan. METHODS: OVERCOME (Japan) was a cross-sectional, observational, population-based web survey of Japanese people with migraine conducted between July and September 2020. The burden and impact of migraine were assessed using the Migraine Disability Assessment (MIDAS), Migraine-Specific Quality-of-Life Questionnaire (MSQ), Migraine Interictal Burden Scale (MIBS-4), and Work Productivity and Activity Impairment-Migraine scale. Results were stratified by average number of monthly headache days (0-3, 4-7, 8-14, ≥ 15). RESULTS: In total, 17,071 Japanese people with migraine completed the survey. Of these, 14,033 (82.2%) met International Classification of Headache Disorders, 3rd edition criteria for migraine and 9667 (56.6%) reported a physician diagnosis of migraine. Overall, 20.7% of respondents experienced moderate-to-severe disability (MIDAS). Moderate-to-severe interictal burden (MIBS-4) was experienced by 41.5% of respondents. MSQ scores in all domains were lowest in respondents with the most frequent headaches (≥ 15 monthly headache days) and highest in those with the lowest frequency headaches (≤ 3 monthly headache days), indicating poorer quality of life in those with more frequent headaches. Work time missed due to migraine (absenteeism) increased with increasing headache frequency, from 3.8 to 6.2%; presenteeism affected 29.8-49.9% of work time. Although migraine burden was greatest in people with the most frequent headaches, those with the lowest headache frequency still experienced substantial disability, interictal burden, and impacts on productivity and quality of life. There was also substantial unmet need for migraine care: 36.5% of respondents had ever hesitated to seek medical care for their headaches, and 89.8% had never used preventive medication. CONCLUSION: In Japan, the burden of migraine and barriers to migraine care are substantial. Improving patient awareness and healthcare provider vigilance may help improve patient outcomes.

Burden of chronic low back pain: Association with pain severity and prescription medication use in five large European countries.

OBJECTIVE: This study assessed associations between severity of, and prescription medication use for, chronic low back pain (CLBP) and health-related quality of life, health status, work productivity, and healthcare resource utilization. METHODS: This cross-sectional study utilized SF-12, EQ-5D-5L, and work productivity and activity impairment (WPAI) questionnaires, and visits to healthcare providers among adults with self-reported CLBP participating in the National Health and Wellness Survey in Germany, France, UK, Italy, and Spain. Respondents were stratified into four groups according to pain severity (mild or moderate/severe) and prescription medication use (Rx-treated or Rx-untreated). Differences between groups were estimated using generalized linear models controlling for sociodemographics and health characteristics. RESULTS: Of 2086 respondents with CLBP, 683 had mild pain (276 Rx-untreated, 407 Rx-treated) and 1403 had moderate/severe pain (781 Rx-untreated, 622 Rx-treated). Respondents with moderate/severe pain had significantly worse health-related quality of life (SF-12v2 physical component summary), health status (EQ-5D-5L), and both absenteeism and presenteeism compared with those with mild pain, including Rx-untreated (moderate/severe pain Rx-untreated vs. mild pain Rx-untreated, p ≤ 0.05) and Rx-treated (moderate/severe pain Rx-treated vs. mild pain Rx-treated, p ≤ 0.05) groups. Significantly more visits to healthcare providers in the last 6 months were reported for moderate/severe pain compared with mild pain for Rx-treated (least squares mean 13.01 vs. 10.93, p = 0.012) but not Rx-untreated (8.72 vs. 7.61, p = 0.072) groups. Health-related quality of life (SF-12v2 physical component summary) and health status (EQ-5D-5L), as well as absenteeism and presenteeism, were significantly worse, and healthcare utilization was significantly higher, in the moderate/severe pain Rx-treated group compared with all other groups (all p ≤ 0.05). CONCLUSION: Greater severity of CLBP was associated with worse health-related quality of life, health status, and absenteeism and presenteeism, irrespective of prescription medication use. Greater severity of CLBP was associated with increased healthcare utilization in prescription medication users.

Comprehensive population-based survey of migraine in Japan: results of the ObserVational Survey of the Epidemiology, tReatment, and Care Of MigrainE (OVERCOME [Japan]) study.

OBJECTIVE: The ObserVational survey of the Epidemiology, tReatment, and Care Of MigrainE study in Japan (OVERCOME [Japan]) aimed to provide an up-to-date assessment of migraine epidemiology in Japan. METHODS: OVERCOME (Japan) was a cross-sectional, population-based web survey of Japanese adults recruited from consumer panels. People with active migraine (met modified International Classification of Headache Disorders, 3rd edition [ICHD-3] criteria or had a self-reported physician diagnosis of migraine) answered questions about headache features, physician consultation patterns, and migraine medication use. The burden and impact of migraine were assessed using Migraine Disability Assessment (MIDAS) and Work Productivity and Activity Impairment scales. RESULTS: In total, 231,747 respondents accessed the screener, provided consent, and were eligible for the survey. The migraine group included 17,071 respondents (mean ± SD age 40.7 ± 13.0 years; 66.5% female). ICHD-3 migraine criteria were met by 14,033 (82.2%) respondents; 9667 (56.6%) self-reported a physician diagnosis of migraine. The mean number of monthly headache days was 4.5 ± 5.7 and pain severity (0-10 scale) was 5.1 ± 2.2. In the migraine group, 20.7% experienced moderate to severe migraine-related disability (MIDAS score ≥ 11). Work productivity loss was 36.2% of work time missed, including 34.3% presenteeism. Only 57.4% of respondents had ever sought medical care for migraine/severe headache. Most respondents (75.2%) were currently using over-the-counter medications for migraine; 36.7% were using prescription nonsteroidal anti-inflammatory drugs, and only 14.8% were using triptans. Very few (9.2%) used preventive medications. CONCLUSIONS: Unmet needs for migraine health care among people with migraine in Japan include low rates of seeking care and suboptimal 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.

Adherence to national guidelines for colorectal cancer screening in Israel: Comprehensive multi-year assessment based on electronic medical records.

OBJECTIVES: To assess time trends in colorectal cancer screening uptake, time-to-colonoscopy completion following a positive fecal occult blood test and associated patient factors, and the extent and predictors of longitudinal screening adherence in Israel. SETTING: Nation-wide population-based study using data collected from four health maintenance organizations for the Quality Indicators in Community Healthcare Program. METHODS: Screening uptake for the eligible population (aged 50-74) was recorded 2003-2018 using aggregate data. For a subcohort (2008-2012, N = 1,342,617), time-to-colonoscopy following a positive fecal occult blood test and longitudinal adherence to screening guidelines were measured using individual-level data, and associated factors assessed in multivariate models. RESULTS: The annual proportion screened rose for both sexes from 11 to 65%, increasing five-fold for age group 60-74 and >six-fold for 50-59 year olds, respectively. From 2008 to 2012, 67,314 adults had a positive fecal occult blood test, of whom 71% eventually performed a colonoscopy after a median interval of 122 (95% confidence interval 110.2-113.7) days. Factors associated with time-to-colonoscopy included age, socioeconomic status, and comorbidities. Only 25.5% of the population demonstrated full longitudinal screening adherence, mainly attributable to colonoscopy in the past 10 years rather than annual fecal occult blood test performance (83% versus 17%, respectively). Smoking, diabetes, lower socioeconomic status, cardiovascular disease, and hypertension were associated with decreased adherence. Performance of other cancer screening tests and frequent primary care visits were strongly associated with adherence. CONCLUSIONS: Despite substantial improvement in colorectal cancer screening uptake on a population level, individual-level data uncovered gaps in colonoscopy completion after a positive fecal occult blood test and in longitudinal adherence to screening, which should be addressed using focused interventions.

Health Inequalities in the Use of Telehealth in the United States in the Lens of COVID-19.

The use of remote health care services, or telehealth, is a promising solution for providing health care to those unable to access care in person easily and thus helping to reduce health inequalities. The COVID-19 pandemic and resulting stay-at-home orders in the United States have created an optimal situation for the use of telehealth services for non-life-threatening health care use. A retrospective cohort study was performed using Kantar's Claritis™ database, which links insurance claims encounters (Komodo Health) with patient-reported data (Kantar Health, National Health & Wellness Survey). Logistic regression models (odds ratios [OR], 95% confidence intervals [CI]) examined predictors of telehealth versus in-person encounters. Adults ages ≥18 years eligible for payer-complete health care encounters in both March 2019 and March 2020 were identified (n = 35,376). Telehealth use increased from 0.2% in 2019 to 1.9% in 2020. In adjusted models of respondents with ≥1 health care encounter (n = 11,614), age, marital status, geographic residence (region; urban/rural), and presence of anxiety or depression were significant predictors of telehealth compared with in-person use in March 2020. For example, adults 45-46 years versus 18-44 years were less likely to use telehealth (OR 0.684, 95% CI: 0.561-0.834), and respondents living in urban versus rural areas were more likely to use telehealth (OR 1.543, 95% CI: 1.153-2.067). Substantial increases in telehealth use were observed during the onset of the COVID-19 pandemic in the United States; however, disparities existed. These inequalities represent the baseline landscape that population health management must monitor and address during this pandemic.

Factors associated with the discontinuation of dipeptidyl peptidase-4 inhibitors (DPP-4is) after initiation of insulin.

Objective: Type 2 diabetes (T2D) is a prevalent health problem. Oral agents, with the exception of metformin, are often discontinued with the initiation of insulin. The objective was to understand the proportion of patients discontinuing dipeptidyl peptidase-4 inhibitors (DPP-4is) and the reasons for the decision to discontinue.Methods: A retrospective study using a health claims database investigated discontinuation of DPP-4i in adult patients on a dual therapy of metformin and DPP-4i who initiated insulin (n = 3391). An online survey administered to 406 physicians in the US examined reasons for discontinuation. Physicians surveyed included endocrinologists (34.5%), general practitioners (32.5%), internal medicine specialists (30.5%), and diabetologists (2.5%), treating a monthly average of 154 patients.Results: Among patients treated with metformin and DPP-4is who were newly prescribed insulin, 33.3 and 57.3% discontinued DPP-4i therapy within 3 and 12 months, respectively. Patients who discontinued DPP-4i therapy had higher out-of-pocket costs and a greater proportion of renal and liver disease. Top 3 responses for discontinuation included adverse events/tolerability issues (58.9%), lack of efficacy/treatment goals not being met (55.4%) and additional cost of DPP-4i with insulin (48.5%). Top 3 responses for continuing DPP-4i included meeting treatment goals (70.7%), using a lower dose of insulin (65.3%) and good tolerability (48.0%). Physician characteristics, such as physician specialty, age, gender and location impacted to some extent the reasons for treatment decisions.Conclusions: A large proportion of patients discontinue DPP-4is in the real world when initiating insulin. The impact of physician characteristics in treatment decisions highlights the need for enhanced physician training and support as new clinical data emerges and therapy options are available.

The burden of suicidal ideation across Europe: a cross-sectional survey in five countries.

BACKGROUND: Suicidal ideation (SI) is an associated risk of depression, affecting 30-40% of the depressed population. However, there is a paucity of studies investigating the impact of SI in Europe. This retrospective observational study examined the burden of SI among adults with major depressive disorder (MDD) in the 2017 National Health and Wellness Survey in five European countries: France, Germany, Italy, Spain, and the UK. METHODS: Bivariate analyses evaluated group differences between respondents with MDD with and without SI according to demographic characteristics, self-reported health-related quality of life, work productivity and activity impairment (WPAI), and healthcare resource utilization (HRU). Generalized linear models examined group differences country-wise, after controlling for relevant confounders. RESULTS: Among 52,060 respondents, 3,308 individuals were diagnosed with MDD, comprising SI (n=905) and non-SI (nSI) (n=2403) patients. Adjusted differences (ADs), compared to the general population, were observed using the Medical Outcomes Study Short Form Survey (SF-12v2) mental component summary scores (AD: SI=-20.02, nSI=-10.77), physical component summary scores (AD: SI=-4.49, nSI=-2.50), and EuroQoL-5 Dimensions (AD: SI=-0.34, nSI=-0.15) (for all, p<0.001). Significantly greater WPAI and higher HRU were associated with SI compared to nSI. CONCLUSION: The results illustrate the unique impact of SI within the MDD population and the need to reduce the burden.

The humanistic and economic burden of treatment-resistant depression in Europe: a cross-sectional study.

BACKGROUND: A patient is considered to suffer from treatment resistant depression (TRD) when consecutive treatment with two products of different pharmacological classes, used for a sufficient length of time at an adequate dose, fail to induce a clinically meaningful effect (inadequate response). The primary aim of the current study was to examine the humanistic and economic burden of TRD in five European countries, France, Germany, Italy, Spain and the United Kingdom, by comparing with non-treatment resistant depression (nTRD) and general population respondents. METHODS: The sample for this retrospective observational study was taken from the 2017 National Health and Wellness Survey conducted in five European countries. Demographic and patient characteristics were examined for TRD patients compared to respondents with nTRD and to the general population using chi-square tests or one-way analysis of variance for categorical or continuous variables, respectively. Generalized linear models were performed to examine group differences after adjusting these estimates for confounders. RESULTS: A total 52,060 survey respondents were examined, of which 2686 and 622 were considered to have non-treatment resistant and treatment-resistant depression, respectively. Relative to the general population, nTRD and TRD respondents reported significant decrements in health-related quality of life, including lower adjusted mental (- 12.1 vs. -18.1) and physical (- 2.5 vs. -5.4) component scores of the SF-12v2 and increased adjusted relative risk for work (2.2 vs. 2.7) and activity (1.9 vs. 2.5) impairment (all p < 0.001). Additionally, healthcare resource utilization was significantly higher for TRD patients more so than nTRD, compared to the general population, especially for healthcare professional visits (odds ratio nTRD = 5.4; TRD = 15.9, p < 0.001). CONCLUSIONS: In conclusion, TRD patients had significantly lower quality of life, greater work productivity and activity impairment, and increased healthcare resource utilization as compared with nTRD and general population. The study findings suggest an unmet need exists among TRD patients in Europe.

The added burden of depression in patients with osteoarthritis in Japan.

Objectives: In Japan, osteoarthritis (OA) is a leading source of pain and disability; depressive disorders may limit patients' ability to cope with OA. This study examined the incremental effect of depression on the relationship between OA and health-related outcomes. Methods: Data from the 2014 Japan National Health and Wellness Survey (N=30,000) were collected on demographics, OA characteristics, and health characteristics of patients with OA. Depression symptoms were measured, and outcomes included health-related quality of life (HRQoL), work productivity and activity impairment, and health care resource utilization. Generalized linear regression models controlling for confounders were used to predict health-related outcomes. Results: Of 565 respondents with OA, 63 (11%) had symptoms of moderate or severe depression. In adjusted models, HRQoL remained lower among respondents with than without depression (p<0.001). Higher levels of presenteeism (mean±SE: 50%±9% vs 23%±2%) and activity impairment (mean±SE: 57%±7% vs 30%±1%) were observed for patients with than without depression (p<0.001); however, there were no differences for absenteeism (p=0.534). Patients with depression (vs no depression) reported more health care provider visits, emergency room visits, and hospitalizations (for all, p<0.001). Conclusion: Depression heightens the health-related burden of OA. Greater attention to depression among patients with OA is warranted.

Incident gout and chronic Kidney Disease: healthcare utilization and survival.

BACKGROUND: Uncontrolled gout can cause significant joint and organ damage and has been associated with impairments in quality of life and high economic cost. Gout has also been associated with other comorbid diseases, such as chronic kidney disease. The current study explored if healthcare resource utilization (HRU) and survival differs between patients with incident gout in the presence or absence of chronic kidney disease (CKD). METHODS: Clalit Health Services (CHS) data were used to conduct a retrospective population-based cohort study of incident gout between 1/1/2006-31/12/2009. Incident cases of gout were identified and stratified by CKD status and by age group (< 55 and 55+ years). CKD status was defined as a pre-existing diagnosis of chronic kidney disease, chronic renal failure, kidney transplantation, or dialysis at index date. Demographic and clinical characteristics, as well as healthcare resource use, were reported. RESULTS: A total of 12,940 incident adult gout patients, with (n = 8286) and without (n = 4654) CKD, were followed for 55,206 person-years. Higher rates of HRU were observed for gout patients with CKD than without. Total annual hospital admissions for patients with gout and CKD were at least 3 times higher for adults < 55 (mean = 0.51 vs 0.13) and approximately 1.5 times higher for adults 55+ (mean = 0.46 vs 0.29) without CKD. Healthcare utilization rates from year 1 to year 5 remained similar for gout patients < 55 years irrespective of CKD status, however varied according to healthcare utilization by CKD status for gout patients 55+ years. The 5-year all-cause mortality was higher among those with CKD compared to those without CKD for both age groups (HR< 55 years = 1.65; 95% CI 1.01-2.71; HR55+ years = 1.50; 95% CI 1.37-1.65). CONCLUSIONS: The current study suggests important differences exist in patient characteristics and outcomes among patients with gout and CKD. Healthcare utilization differed between sub-populations, age and comorbidities, over the study period and the 5-year mortality risk was higher for gout patients with CKD, regardless of age. Future work should explore factors associated with these outcomes and barriers to gout control in order to enhance patient management among this high-risk subgroup.

Population-based cohort of 500 patients with Gaucher disease in Israel.

OBJECTIVE: To characterise a population-based cohort of patients with Gaucher disease (GD) in Israel relative to the general population and describe sociodemographic and clinical differences by disease severity (ie, enzyme replacement therapy [ERT] use). DESIGN: A cross-sectional study was conducted. SETTING: Data from the Clalit Health Services electronic health record (EHR) database were used. PARTICIPANTS: The study population included all patients in the Clalit EHR database identified as having GD as of 30 June 2014. RESULTS: A total of 500 patients with GD were identified and assessed. The majority were ≥18 years of age (90.6%), female (54.0%), Jewish (93.6%) and 34.8% had high socioeconomic status, compared with 19.0% in the general Clalit population. Over half of patients with GD with available data (51.0%) were overweight/obese and 63.5% had a Charlson Comorbidity Index ≥1, compared with 46.6% and 30.4%, respectively, in the general Clalit population. The majority of patients with GD had a history of anaemia (69.6%) or thrombocytopaenia (62.0%), 40.4% had a history of bone events and 22.2% had a history of cancer. Overall, 41.2% had received ERT. CONCLUSIONS: Establishing a population-based cohort of patients with GD is essential to understanding disease progression and management. In this study, we highlight the need for physicians to monitor patients with GD regardless of their ERT status.

Disease burden of fractures among patients with osteoporosis in Japan: health-related quality of life, work productivity and activity impairment, healthcare resource utilization, and economic costs.

Osteoporosis remains undertreated in Japan, and bone fractures are the most frequent complications imposing heavy burden on individuals and the community. This paper investigates the clinical and economic burden of fractures among osteoporosis patients in Japan. The Japan National Health and Wellness Survey 2012-2014 database was used for analysis. Respondents aged ≥ 50 years and indicated a physician diagnosis of osteoporosis (N = 1107) were categorized into three subgroups: no prior fracture (N = 693), single fracture (N = 242), and multiple (≥ 2) fractures (N = 172). Health-related quality of life (HRQoL), work productivity and activity impairment, healthcare resource utilization and associated direct and indirect costs were compared across three fracture subgroups adjusting for respondents' sociodemographic and clinical characteristics using generalized linear regression models. The estimated fracture prevalence among respondents with osteoporosis who were ≥ 50 years was 37.4%, of whom 41.5% had multiple fractures. Relative to osteoporosis respondents with no fracture and with single fracture, those with multiple fractures reported significant higher disability in HRQoL, more healthcare resource utilization, and were associated with higher direct costs. Improved treatment of fractures among osteoporosis patients is necessary and may help reduce the clinical and economic burden in this osteoporosis population.