Burden of illness in not adequately controlled chronic hypoparathyroidism: Findings from a 13-country patient and caregiver survey.

OBJECTIVE: To address knowledge gaps regarding burdens associated with not adequately controlled chronic hypoparathyroidism. DESIGN: Global patient and caregiver survey. STUDY POPULATIONS: Patients with chronic hypoparathyroidism not adequately controlled on conventional therapy and their caregivers. MEASUREMENTS: Health-related quality of life (HRQoL) and health status were evaluated using the 36-item Short Form version 2 (SF-36 v2.0) and Five-Level EuroQoL 5 Dimensions (EQ-5D-5L) instruments, respectively. Hypoparathyroidism-associated symptoms were assessed by a disease-specific Hypoparathyroidism Symptom Diary and caregiver burden via the Modified Caregiver Strain Index (MCSI). RESULTS: Data were obtained from 398 patients and 207 caregivers. Patients' self-rated hypoparathyroidism-related symptom severity was none (3%), mild (32%), moderate (53%) or severe (12%). Per the Hypoparathyroidism Symptom Diary, patients reported moderate, severe or very severe symptoms of physical fatigue (73%), muscle cramps (55%), heaviness in limbs (55%) and tingling (51%) over a 7-day recall period. Impacts (rated 'somewhat' or 'very much') were reported by 84% of patients for ability to exercise, 78% for sleep, 75% for ability to work and 63% for family relationships. Inverse relationships were observed between patient self-rated overall symptom severity and HRQoL and health status assessment scores-the greater the symptom severity, the lower the SF-36 and EQ-5D-5L scores. Caregiver burden increased with patient self-rated symptom severity: none, 1.7 MCSI; mild, 5.4 MCSI; moderate, 9.5 MCSI; and severe, 12.5 MCSI. CONCLUSION: Patients with not adequately controlled hypoparathyroidism reported substantial symptoms and impacts. Greater patient symptom severity was associated with decreased patient HRQoL and health status assessments and increased caregiver burden.

Characterizing the experience of agitation in patients with bipolar disorder and schizophrenia.

BACKGROUND: Agitation is a common manifestation of bipolar disorder and schizophrenia which includes symptoms ranging from inner tension and unease to violence and aggression. Much of the existing literature has focused on agitation in the acute setting, with the patient experience poorly defined. Thus, the aim of this study was to characterize agitation and its management from a patient perspective, with the focus on those who reside in the community. METHODS: Surveys were completed across Germany, Spain and the UK by 583 community dwelling patients with schizophrenia or bipolar disorder who experienced episodes of agitation. Patients were recruited via either their physician or through patient support groups. The survey captured information on demographics, disease characteristics, frequency of agitation episodes and different pre-defined severity levels ranging from mild to severe, symptoms experienced during an episode, awareness of agitation and coping strategies employed by the patient. Statistics were descriptive in nature. RESULTS: The most commonly reported symptoms during an episode of agitation were feeling uneasy (n = 373, 64%), restless (n = 368, 63%) or nervous (n = 368, 63%). Patients experienced an average of 22.4 (SD 57.2) mild, 15.4 (SD 61.2) moderate, 6.8 (SD 63.3) moderate-intense and 2.9 (SD 24.4) severe episodes within the last 12 months; on average 2.7 (SD 6.8) required hospital attendance. Half of patients (n = 313) had attended hospital due to agitation. In total, 71% of patients (n = 412) were aware they were becoming agitated either always or sometimes and 61% of patients (n = 347) were aware of agitation triggers either always or sometimes. The majority of patients reported being able to sometimes control their agitation (56%, n = 329) but 16% (n = 94) stated that there is typically nothing they can do. To cope with episodes 55% (n = 125) of schizophrenia patients and 66% (n = 234) of bipolar disorder patients reported taking prescribed medication. CONCLUSION: Community based patients with schizophrenia and bipolar disorder reported frequently experiencing agitation episodes which they defined most commonly as feeling uneasy, restless or nervous. A range of coping strategies were reported but they were not always successful, highlighting an area of unmet need in this population.

A survey of caregiver burden in those providing informal care for patients with schizophrenia or bipolar disorder with agitation: results from a European study.

BACKGROUND: Agitation is a common feature of bipolar disorder and schizophrenia. Previous research indicates that specific symptoms impact caregiver burden in these conditions, but the impact of agitation on caregiver experience is poorly understood. The aim of this study was to characterise caregiver burden in providers of informal care for patients with bipolar disorder and schizophrenia who experience agitation. METHODS: In total, 297 matched patient and caregiver surveys were collected across the UK, Germany and Spain between October 2016 and January 2017. To be eligible, caregivers needed to provide informal care to a patient with a diagnosis of bipolar disorder or schizophrenia with agitation managed in a community setting and participating in the patient survey. The caregiver survey captured information on demographics and their role in managing the patient's agitation. Caregiver burden was assessed using the Involvement Evaluation Questionnaire. Descriptive analysis was conducted. RESULTS: Caregivers provided 38.3 h (SD ± 40.34) a week of support to the patient with 20% providing 50 h or more. Most caregivers reported that they recognised an episode of agitation all of the time (44%, n = 130) or sometimes (40%, n = 119). Verbal de-escalation techniques (talking (80%, n = 239) and soothing (73%, n = 218) were the most commonly reported strategies used by caregivers during an episode of agitation; 14% (n = 43) reported resorting to physically restraining the patient. Caregivers supervised rescue medication administration regularly (41%, n = 69) or occasionally (49%, n = 82). Mean Involvement Evaluation Questionnaire score was 32.2 (± 15.27), equivalent to 28.4 (± 13.56) in Germany, 35.6 (± 16.55) in Spain and 33.3 (± 15.15) in the UK. Involvement Evaluation Questionnaire scores were higher for caregivers who reported hostile (41.7 ± 17.07) lack of control (40.3 ± 16.35) and violent (39.5 ± 16.40) patient behaviours when agitated. Over excitement (31.8 ± 15.05), restless (32.6 ± 14.77) and tense (32.9 ± 15.64) behaviours were associated with a lower Involvement Evaluation Questionnaire score. CONCLUSIONS: Caregivers are active participants in the recognition and management of agitation episodes. The substantial burden reported by these caregivers is impacted by factors including the number of hours of care provided, patient behaviours and country. These may be viable targets for effective interventions to reduce caregiver burden.

Socioeconomic burden of schizophrenia: a targeted literature review of types of costs and associated drivers across 10 countries.

AIMS: Schizophrenia has the highest median societal cost per patient of all mental disorders. This review summarizes the different costs/cost drivers (cost components) associated with schizophrenia in 10 countries, including all cost types and stakeholder perspectives, and highlights aspects of disease associated with greatest costs. MATERIALS AND METHODS: Targeted literature review based on a search of published research from 2006 to 2021 in the United States (US), United Kingdom (UK), France, Germany, Italy, Spain, Canada, Japan, Brazil, and China. RESULTS: Sixty-four published articles (primary studies and literature reviews) were included. Comprehensive data were available on costs in schizophrenia overall, with very limited data for individual countries except the US. Most data is related to direct and not indirect costs, with extremely scarce data for several key cost components (adverse events, suicide, long-term care). Total schizophrenia-related per person per year (PPPY) costs were $2,004-94,229, with considerable variability among countries. Indirect costs were the main cost driver (50-90% of all costs), ranging from $1,852 to $62,431 PPPY. However, indirect costs are not collected systematically or incorporated in health technology assessments. Total schizophrenia-related PPPY direct costs were $4,394-31,798, with inpatient cost as the main cost driver (∼20-99% of direct costs). Intangible costs were not reported. Despite limited evidence, total schizophrenia-related costs were higher in patients with than without negative symptoms, largely due to increased costs of medication and medical visits. LIMITATIONS: As this was not a systematic review, prioritization of studies may have resulted in exclusion of potentially relevant data. All costs were converted to USD but not corrected for inflation or subjected to a gross domestic product deflator. CONCLUSIONS: Direct costs are most commonly reported in schizophrenia. The substantial underreporting of indirect and intangible costs undervalues the true economic burden of schizophrenia from a payer, patient, and societal perspective.

The true costs of diseases such as schizophrenia extend far beyond the obvious direct costs of hospital visits, outpatient appointments and medications to include indirect costs such as loss of productivity among patients and caregivers due to unemployment, early retirement and premature death. This review of literature published between 2006 and 2021 reveals that the indirect costs of schizophrenia actually account for between 50% and 90% of all costs, but are often not taken into account in healthcare planning. In addition, intangible costs, including the pain, suffering, stress, and anxiety experienced by patients and caregivers due to schizophrenia have not been reported in the literature. Costs were also higher for patients with negative symptoms of schizophrenia (where patients appear withdrawn and lacking in emotion, with few social relationships) compared with those with positive symptoms (including delusions or hallucinations). This is largely due to the greater costs for medications and medical visits among patients with negative symptoms. In summary, this review demonstrates that the true cost of schizophrenia, including direct, indirect, and intangible costs, is likely to be substantially higher than the values for the cost of disease currently reported.

Physician Practice Patterns Associated with Diagnostic Evaluation of Patients with Suspected Mild Cognitive Impairment and Alzheimer's Disease.

The diagnostic process for patients presenting with cognitive decline and suspected dementia is complex. Physicians face challenges distinguishing between normal aging, mild cognitive impairment, Alzheimer's disease, and other dementias. Although there is some evidence for improving attitudes towards the importance of prompt diagnosis, there is limited information describing how physicians approach this diagnostic challenge in practice. This was explored in the present study. Across-sectional survey of primary care and specialist physicians, in 5 European countries, Canada, and the United States, was conducted. Participants were asked about their use of cognitive screening tools and diagnostic technologies, as well as the rationales and barriers for use. In total, 1365 physicians participated in the survey, 63% of whom were specialists. Most physicians stated they use objective cognitive tools to aid the early detection of suspected mild cognitive impairment or Alzheimer's disease in patients. The Mini-Mental State Examination was the most common tool used for initial screening; respondents cited speed and ease of use but noted its lack of specificity. Cerebrospinal fluid biomarker and amyloid positron emission tomography tests, respectively, had been used by only 26% and 32% of physicians in the preceding 6 months, although patterns of use varied across countries. The most commonly cited reasons for not ordering such tests were invasiveness (for cerebrospinal fluid biomarker testing) and cost (for amyloid positron emission tomography imaging). Data reported by physicians reveal differences in the approaches to the diagnostics process in Alzheimer's. A higher proportion of primary care physicians in the United States are routinely incorporating cognitive assessment tools into annual visits, but this is due to country differences in clinical practice. The value of screening tools and regular use could be discussed further with physicians; however, lack of specificity associated with cognitive tools and the investment required from patients and the healthcare system are limiting factors.

Physician Perceptions about the Barriers to Prompt Diagnosis of Mild Cognitive Impairment and Alzheimer's Disease.

Prior studies have identified numerous barriers to the prompt diagnosis of patients with suspected Alzheimer's disease (AD). The aim of the study was to evaluate physician's perceptions of the importance of previously identified barriers to diagnosis, but with a specific focus on the presentation of mild cognitive impairment (MCI), which may be indicative of neurodegenerative disorders such as AD. A second aim was to evaluate how the perspective of primary care physicians (PCPs) may differ from that of specialists. A cross-sectional online survey of PCPs and specialists who routinely manage patients with complaints of age-related cognitive impairment was conducted. Participants were asked to identify barriers to prompt diagnosis from prespecified lists of known diagnostic challenges categorized into 4 domains: patient-related, physician-related, setting-related, and those relating to the clinical profile of AD. Physicians report a range of barriers when attempting to diagnose MCI and AD. Major themes included patients seeing cognitive decline as a normal part of aging and not disclosing symptoms, long waiting lists, and a lack of treatment options and definitive biomarker tests. Generally, PCPs and specialists showed broad agreement; however, PCPs were more likely to identify burdens on the healthcare system, such as long waiting lists and inadequate time to evaluate patients. Substantial barriers continue to hinder early diagnosis of MCI and AD. There are numerous areas where improvements might be made but the implementation of potential interventions will likely be associated with financial strain for many healthcare systems.

Remote Monitoring of Patient-Reported Outcomes in Ulcerative Colitis: A Prospective Real-World Pilot Study.

INTRODUCTION: The 6-point version of the Mayo score relies on two patient-reported outcomes (PRO2): stool frequency and rectal bleeding. We assessed the feasibility and acceptability of remote online PRO2 reporting for golimumab-treated ulcerative colitis (UC) patients. PATIENTS AND METHODS: This was a UK-based, multi-centre, prospective, real-world, non-interventional pilot study. Eligible patients completed PRO2 scores at baseline and every 4 weeks over a period of 6 months. Demographics were collected at baseline and a satisfaction questionnaire was completed at study end. Each patient provided data anonymously via an online platform. RESULTS: Fifty-two patients enrolled in the study. Mean (SD) patient age was 40.8 (13.6); 52% were male. Patients provided data on a personal computer (44%), mobile phone (38%) or tablet (18%). Forty-seven (90%) patients completed the baseline questionnaire within the accepted time range. Subsequent scores were reported on time by eligible patients with a success rate of 94%, 92%, 90%, 87%, 90% and 81% at end of months 1-6, respectively. CONCLUSIONS: Remote monitoring of PRO2 in UC was feasible amongst the sample tested. Of those initially willing to provide data in this way, attrition was low. Formal roll-out of this system could be used to support a more frequent assessment of UC symptoms without over-burdening the healthcare system.

Functional performance in patients with COPD: association with treatment regimen, GOLD group, lung function, and symptom burden in a cross-sectional study.

Background: Data suggesting that low physical activity levels are associated with increased mortality and exacerbations in patients with COPD have led to increasing interest in the role of physical activity in COPD. This study evaluated self-reported functional performance, a measure of physical activity impairment, according to current treatment regimen, lung function, symptoms, and Global Initiative for Chronic Obstructive Lung Disease (GOLD) 2017 groups in a large sample of patients with COPD. Methods: This multicenter, cross-sectional, observational study (study identifier: D5970R00003) included patients with COPD (≥40 years) in the USA. A self-completion questionnaire captured demographics and patient-reported outcomes, including the Functional Performance Inventory-Short Form (FPI-SF). Diagnosis and treatment history (including spirometry results) were extracted from medical charts. Multiple linear regression was used to determine the relationship between FPI-SF and FEV1 % predicted, and FPI-SF and COPD assessment test (CAT) score. Results: Overall, 1,775 patients participated (classified as GOLD 2017 group A, 14.8%; B, 46.6%; C, 2.6%; D, 36.0%). Physical activity impairment affected patients across all treatment regimens and GOLD groups (mean FPI-SF total score: 2.1), with the greatest impairment within FPI-SF observed for domains requiring most physical exertion, "physical exercise" and "maintaining the household" (mean FPI-SF scores: 1.7 and 1.8, respectively). Patients receiving loose triple therapy and those in GOLD group D had the highest impairment (mean FPI-SF total scores: both 1.9), and the lowest FEV1 % predicted (55.5% and 54.7%, respectively). FPI-SF total score correlated with FEV1 % predicted and more strongly with CAT score (all P<0.05). Conclusion: The stronger correlation between FPI-SF and CAT scores compared to FPI-SF and FEV1 % predicted suggests that symptoms may have a greater impact on patients' functional performance than lung function. Further longitudinal studies are required to establish a correlation between the effect of treatment on symptoms, lung function, and physical activity.

Chart review across EU5 in MM post-ASCT patients.

AIM: To understand the current treatment patterns, clinical outcomes and healthcare resource utilization-associated costs for multiple myeloma patients, post autologous stem cell transplant (ASCT) across Europe. PATIENTS & METHODS: Medical records were used to abstract data for 337 multiple myeloma patients who had received ASCT. RESULTS: Following ASCT, 7% received maintenance therapy prior to progression. Lenalidomide was the most frequently prescribed maintenance, second- and third-line therapy. Monthly resource use was considerably lower in patients who received maintenance therapy (€638.14 vs €1001.74). Median time to progression was longer for patients who had received maintenance therapy. CONCLUSION: The study highlights the diversity in current treatment patterns post-ASCT. Results suggest patients who receive maintenance therapy have a prolonged remission period, and as a result their associated healthcare resource utilization is spread across the treatment pathway.