Minimizing the burden of cancer in the United States: Goals for a high-performing health care system.

Between 1991 and 2015, the cancer mortality rate declined dramatically in the United States, reflecting improvements in cancer prevention, screening, treatment, and survivorship care. However, cancer outcomes in the United States vary substantially between populations defined by race/ethnicity, socioeconomic status, health insurance coverage, and geographic area of residence. Many potentially preventable cancer deaths occur in individuals who did not receive effective cancer prevention, screening, treatment, or survivorship care. At the same time, cancer care spending is large and growing, straining national, state, health insurance plans, and family budgets. Indeed, one of the most pressing issues in American medicine is how to ensure that all populations, in every community, derive the benefit from scientific research that has already been completed. Addressing these questions from the perspective of health care delivery is necessary to accelerate the decline in cancer mortality that began in the early 1990s. This article, part of the Cancer Control Blueprint series, describes challenges with the provision of care across the cancer control continuum in the United States. It also identifies goals for a high-performing health system that could reduce disparities and the burden of cancer by promoting the adoption of healthy lifestyles; access to a regular source of primary care; timely access to evidence-based care; patient-centeredness, including effective patient-provider communication; enhanced coordination and communication between providers, including primary care and specialty care providers; and affordability for patients, payers, and society.

Treatment patterns and burden of infection in patients with chronic lymphocytic leukemia and secondary immunodeficiency: a retrospective database study.

Patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) and secondary immunodeficiency disease (SID) are susceptible to severe, recurrent, or persistent infections. This retrospective cohort study assessed the burden of infection in patients with CLL/SLL with and without SID, and in immunoglobulin replacement therapy (IgRT)-treated and -untreated patients with CLL/SLL and SID. Anonymized data from the US Optum-Humedica database (Oct-1-2015-Mar-10-2020) were used. Eligible patients aged ≥ 18 years with a confirmed CLL/SLL diagnosis were assigned to cohorts (SID or no-SID) using an algorithm based on serum IgG levels < 5.0 g/L, hypogammaglobulinemia diagnosis codes, and ≥ 1 major infection. A further sub-categorization was made based on patients with SID who received IgRT and those who did not. During 12-month follow-up, patients with CLL/SLL and SID were significantly more likely to experience infections (70.1% vs. 30.4%), including severe bacterial infections (39.8% vs. 9.2%), and infections requiring hospitalization (27.7% vs. 5.8%) than patients without SID. The use of anti-infectives and healthcare resource utilization (HCRU) was also higher in the SID cohort versus the no-SID cohort. Overall survival was shorter in patients with SID than those without (12.3 vs. 16.9 months). In patients with CLL/SLL and SID, burden of infection and HCRU were greater in IgRT-treated patients than in no-IgRT patients, potentially highlighting the IgRT-treated cohort as a more vulnerable population. Increasing understanding of SID burden may help to improve outcomes in patients with CLL/SLL. Further research is needed to develop guidance for IgRT use and to assess the benefits of IgRT in this vulnerable population.

A quantitative cross-sectional study of the burden of caring for patients with Lennox-Gastaut syndrome, Dravet syndrome, and tuberous sclerosis complex-associated epilepsy in Japan.

INTRODUCTION: Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), and tuberous sclerosis complex (TSC)-associated epilepsy are rare conditions associated with severe childhood-onset epilepsy. Caregivers play a critical role in the patients' care and may experience significant psychosocial and socioeconomic burden. This cross-sectional study determined the burden of caring for patients with these rare epilepsy conditions in Japan. METHODS: A quantitative online survey was used to assess patients' and caregivers' characteristics and the caregivers' emotional state, among others. Several validated questionnaires were used: the Hospital Anxiety and Depression Scale (HADS; 0-21 score) assessed the caregivers' emotional wellbeing, the Pediatric Quality of Life Inventory Family Impact Module (PedsQL FIM; 0-100 score) assessed the health-related quality of life (HRQoL) of the caregivers and their families, and the Work Productivity and Activity Impairment General Health (WPAI:GH; 0-100 % score) questionnaire assessed work productivity. RESULTS: A total of 36 caregivers responded (median [interquartile range (IQR)] age 43.5 [39.5, 48.3] years; 33/36 [92 %] female; 13/36 [36 %] working part-time and 13/36 [36 %] not working). Participants cared for 7/36 (19 %), 19/36 (53 %), and 10/36 (28 %) patients with LGS, DS, and TSC, respectively (median [IQR] age, 11.0 [6.8, 16.3] years; age at first seizure, 0 [0, 0] years). Patients received a median (IQR) of 4 (3, 5) treatment drug types. Patients experienced median (IQR) 3.0 (0, 21.0) epileptic seizures in the previous week; 28/36 (78 %) had severe intellectual disabilities, and 34/36 (94 %) had developmental delays. Caregivers reported stress (17/36 [47 %]), sleep problems (13/36 [36 %]), and anxiety (12/36 [33 %]). They spent a median (IQR) of 50.0 (17.5, 70.0) hours caregiving in the previous week, with 3.0 (1.0, 11.0) hours of seizure-specific care. Caregivers reported that their lives would be easier with a median (IQR) of 1.5 (0, 5.0) hours fewer per week caring for patients during/following seizures. Median HADS scores were 9.5 ('suspected anxiety diagnosis') and 7.5 ('no depression') for caregivers, and PedsQL FIM Total median score was 60.1, indicating HRQoL impairment for the caregiver and their family. WPAI:GH scores for paid workers indicated important work impairment. Higher caregiving hours (≥ 21 h vs. < 21 h in the previous week) resulted in higher caregiver burden as indicated by the HADS Total score (p = 0.0062) and PedsQL FIM Total score (p = 0.0007). CONCLUSIONS: Caregivers of patients with LGS, DS, or TSC in Japan experience a significant time burden, reduced HRQoL, and high level of work/activity impairment. Caregivers provide round-the-clock care to patients and rely on family and specialized caring services to help manage the increased caregiving time, which tends to be associated with greater emotional burden and HRQoL impact.

Burden of Asthma Among a Sample of Primary Schoolchildren in Jordan Based on Child and Parental Report.

BACKGROUND: Asthma is a major health issue with an extensive burden to family and health systems. Different measures were used previously to assess disease burden, including prevalence, school performance, parental perceptions, and cost among others. AIM: The purpose of this study was to assess the burden of asthma among schoolchildren using parental and child reports and metered-dose inhaler (MDI) demonstration. METHODS: A descriptive exploratory design based on parent and child self-reports was employed. Parents of children with asthma completed a questionnaire about asthma management and burden including symptoms and health services use. A sample of children with asthma completed a questionnaire measuring their perception of asthma management at school. An assessment of children's proper use of MDI inhalers was also conducted. RESULTS: The burden of asthma was high in the current sample with the majority of children experiencing symptoms, asthma exacerbations, emergency department (ED) visits, and admission because of their asthma. The majority of participating children also performed incorrect inhaler technique, which reflects uncontrolled asthma and poor management. Parental perception of their child's health compared well with their burden of asthma. CONCLUSION: The outcomes of this study and previous reports show an alarmingly high burden of asthma. This calls for collaboration between professionals and stakeholders with children and caregivers in planning and implementing care, including improving the school environment.

The burden of nonalcoholic steatohepatitis (NASH) in the United States.

BACKGROUND: There is limited data on the comparative economic and humanistic burden of non-alcoholic steatohepatitis (NASH) in the United States. The objective was to examine the burden of disease comparing NASH to a representative sample of the general population and separately to a type 2 diabetes mellitus (T2DM) cohort by assessing health-related quality of life (HRQoL) measures, healthcare resource use (HRU) and work productivity and activity impairment (WPAI). METHODS: Data came from the 2016 National Health and Wellness Survey, a nationally representative patient-reported outcomes survey conducted in the United States. Respondents with physician-diagnosed NASH, physician-diagnosed T2DM, and respondents from the general population were compared. Humanistic burden was examined with mental (MCS) and physical (PCS) component summary scores from the Short-Form (SF)-36v2, concomitant diagnosis of anxiety, depression, and sleep difficulties. Economic burden was analysed based on healthcare professional (HCP) and emergency room (ER) visits, hospitalizations in the past six months; absenteeism, presenteeism, overall work impairment, and activity impairment scores on WPAI questionnaire. Bivariate and multivariable analysis were conducted for each outcome and matched comparative group. RESULTS: After adjusting for baseline demographics and characteristics, NASH (N = 136) compared to the matched general population cohort (N = 544), reported significantly lower (worse) mental (MCS 43.19 vs. 46.22, p = 0.010) and physical (PCS 42.04 vs. 47.10, p < 0.001) status, higher % with anxiety (37.5% vs 25.5%, p = 0.006) and depression (43.4% vs 30.1%, p = 0.004), more HCP visits (8.43 vs. 5.17), ER visits (0.73 vs. 0.38), and hospitalizations (0.43 vs. 0.2) all p's < 0.05, and higher WPAI scores (e.g. overall work impairment 39.64% vs. 26.19%, p = 0.011). NASH cohort did not differ from matched T2DM cohort (N = 272) on mental or work-related WPAI scores, but had significantly worse physical status (PCS 40.52 vs. 44.58, p = 0.001), higher % with anxiety (39.9% vs 27.8%, p = 0.043), more HCP visits (8.63 vs. 5.68, p = 0.003) and greater activity impairment (47.14% vs. 36.07%, p = 0.010). CONCLUSION: This real-world study suggests that burden of disease is higher for all outcomes assessed among NASH compared to matched general controls. When comparing to T2DM, NASH cohort has comparable mental and work-related impairment but worse physical status, daily activities impairment and more HRU.

Global critical care: a call to action.

Critical care is underprioritized. A global call to action is needed to increase equitable access to care and the quality of care provided to critically ill patients. Current challenges to effective critical care in resource-constrained settings are many. Estimates of the burden of critical illness are extrapolated from common etiologies, but the true burden remains ill-defined. Measuring the burden of critical illness is epidemiologically challenging but is thought to be increasing. Resources, infrastructure, and training are inadequate. Millions die unnecessarily due to critical illness. Solutions start with the implementation of first-step, patient care fundamentals known as Essential Emergency and Critical Care. Such essential care stands to decrease critical-illness mortality, augment pandemic preparedness, decrease postoperative mortality, and decrease the need for advanced level care. The entire healthcare workforce must be trained in these fundamentals. Additionally, physician and nurse specialists trained in critical care are needed and must be retained as leaders of critical care initiatives, researchers, and teachers. Context-specific research is mandatory to ensure care is appropriate for the patient populations served, not just duplicated from high-resourced settings. Governments must increase healthcare spending and invest in capacity to treat critically ill patients. Advocacy at all levels is needed to achieve universal health coverage for critically ill patients.

Economic burden of atherosclerotic cardiovascular disease: a matched case-control study in more than 450,000 Swedish individuals.

AIM: To examine direct and indirect costs, early retirement, cardiovascular events and mortality over 5 years in people with atherosclerotic cardiovascular disease (ASCVD) and matched controls in Sweden. METHODS: Individuals aged ≥ 16 years living in Sweden on 01 January 2012 were identified in an existing database. Individuals with ASCVD were propensity score matched to controls without ASCVD by age, sex and educational status. We compared direct healthcare costs (inpatient, outpatient and drug costs), indirect costs (resulting from work absence) and the risk of stroke, myocardial infarction (MI) and early retirement. RESULTS: After matching, there were 231,417 individuals in each cohort. Total mean per-person annual costs were over 2.5 times higher in the ASCVD group versus the controls (€6923 vs €2699). Indirect costs contributed to 60% and 67% of annual costs in the ASCVD and control groups, respectively. Inpatient costs accounted for ≥ 70% of direct healthcare costs. Cumulative total costs over the 5-year period were €32,011 in the ASCVD group and €12,931 in the controls. People with ASCVD were 3 times more likely to enter early retirement than controls (hazard ratio [HR] 3.02 [95% CI 2.76-3.31]) and approximately 2 times more likely to experience stroke (HR 1.83 [1.77-1.89]) or MI (HR 2.27 [2.20-2.34]). CONCLUSION: ASCVD is associated with both economic and clinical impacts. People with ASCVD incurred considerably higher costs than matched controls, with indirect costs resulting from work absence and inpatient admissions being major cost drivers, and were also more likely to experience additional ASCVD events.

Application of big data analyses to compare the impact of oral and gastrointestinal mucositis on risks and outcomes of febrile neutropenia and septicemia among patients hospitalized for the treatment of leukemia or multiple myeloma.

PURPOSE: Oral ulcerative mucositis (UM) and gastrointestinal mucositis (GIM) have been associated with increased likelihood of systemic infection (bacteremia and sepsis) in patients being treated for hematological malignancies. To better define and contrast differences between UM and GIM, we utilized the United States 2017 National Inpatient Sample and analyzed patients hospitalized for the treatment of multiple myeloma (MM) or leukemia. METHODS: We utilized generalized linear models to assess the association between adverse events-UM and GIM-among hospitalized MM or leukemia patients and the outcome of febrile neutropenia (FN), septicemia, burden of illness, and mortality. RESULTS: Of 71,780 hospitalized leukemia patients, 1255 had UM and 100 GIM. Of 113,915 MM patients, 1065 manifested UM and 230 had GIM. In an adjusted analysis, UM was significantly associated with increased risk of FN in both the leukemia (aOR = 2.87, 95% CI = 2.09-3.92) and MM cohorts (aOR = 4.96, 95% CI = 3.22-7.66). Contrastingly, UM had no effect on the risk of septicemia in either group. Likewise, GIM significantly increased the odds of FN in both leukemia (aOR = 2.81, 95% CI = 1.35-5.88) and MM (aOR = 3.75, 95% CI = 1.51-9.31) patients. Similar findings were noted when we restricted our analysis to recipients of high-dose condition regimens in preparation for hematopoietic stem-cell transplant. UM and GIM were consistently associated with higher burden of illness in all the cohorts. CONCLUSION: This first use of big data provided an effective platform to assess the risks, outcomes, and cost of care of cancer treatment-related toxicities in patients hospitalized for the management of hematologic malignancies.

Evaluating the burden of endogenous Cushing's syndrome using a web-based questionnaire and validated patient-reported outcome measures.

INTRODUCTION: Endogenous Cushing's syndrome (CS) is a rare endocrine condition caused by chronic oversecretion of cortisol, resulting in a diverse constellation of symptoms. This study examined the ongoing burden of illness (BOI), from the first appearance of symptoms through treatment, which is currently not well evaluated. METHODS: A quantitative, cross-sectional, web-enabled survey including 5 validated patient reported outcomes (PRO) measures was conducted in patients with CS who had been diagnosed ≥ 6 months prior and who had received ≥ 1 treatment for their endogenous CS at the time of the survey. RESULTS: Fifty-five patients participated in this study; 85% were women. The mean age was 43.4 ± 12.3 years (± standard deviation, SD). On average, respondents reported a 10-year gap between the first occurrence of symptoms and diagnosis; 80% underwent surgical treatment for CS. Respondents experienced symptoms on 16 days in a typical month, and their health-related quality of life was moderately impacted based on the CushingQoL score. Weight gain, muscle fatigue, and weakness were the most common symptoms and 69% percent of patients reported moderate or severe fatigue using the Brief Fatigue Inventory. Following treatment, the occurrence of most symptoms declined over time, although anxiety and pain did not significantly decrease. Overall, 38% of participants reported an annual average of 25 missed workdays due to CS symptoms. CONCLUSIONS: These results demonstrate a BOI in CS despite ongoing treatment and illustrate the need for interventions to address persistent symptoms, particularly weight gain, pain, and anxiety.

Comparison of the burden of self-reported bacterial sexually transmitted infections among men having sex with men across 68 countries on four continents.

BACKGROUND: Men who have sex with men (MSM) are in general more vulnerable to sexually transmitted infections (STIs) than the heterosexual men population. However, surveillance data on STI diagnoses lack comparability across countries due to differential identification of MSM, diagnostic standards and methods, and screening guidelines for asymptomatic infections. METHODS: We compared self-reported overall diagnostic rates for syphilis, gonorrhea, and chlamydia infections, and diagnostic rates for infections that were classified to be symptomatic in the previous 12 months from two online surveys. They had a shared methodology, were conducted in 68 countries across four continents between October 2017 and May 2018 and had 202,013 participants. RESULTS: Using multivariable multilevel regression analysis, we identified age, settlement size, number of sexual partners, condom use for anal intercourse, testing frequency, sampling rectal mucosa for extragenital testing, HIV diagnosis, and pre-exposure prophylaxis use as individual-level explanatory variables. The national proportions of respondents screened and diagnosed who notified some or all of their sexual partners were used as country-level explanatory variables. Combined, these factors helped to explain differences in self-reported diagnosis rates between countries. The following differences were not explained by the above factors: self-reported syphilis diagnoses were higher in Latin America compared with Europe, Canada, Israel, Lebanon, and the Philippines (aORs 2.30 - 3.71 for symptomatic syphilis compared to Central-West Europe); self-reported gonorrhea diagnoses were lower in Eastern Europe and in Latin America compared with all other regions (aORs 0.17-0.55 and 0.34 - 0.62 for symptomatic gonorrhea compared to Central-West Europe); and self-reported chlamydia diagnoses were lower in Central East and Southeast Europe, South and Central America, and the Philippines (aORs 0.25 - 0.39 for symptomatic chlamydia for Latin American subregions compared to Central West Europe). CONCLUSIONS: Possible reasons for differences in self-reported STI diagnosis prevalence likely include different background prevalence for syphilis and syndromic management without proper diagnosis, and different diagnostic approaches for gonorrhea and chlamydia.

Foodborne disease outbreaks in flour and flour-based food products from microbial pathogens in the United States, and their health economic burden.

The most comprehensive and inclusive estimates for the economic burden of foodborne illness yield values as high as $97.4 billion USD annually. However, broad incidence and cost estimates have limited use if they cannot be attributed to specific foods, for the purposes of food safety control. In this study, we estimated the economic burden of foodborne illnesses resulting from flour and flour-based food products in the United States from the years 2001 to 2021. The outbreak, illness burden, and health economic data are combined to generate these estimates. Our model combined outbreak data with published Centers for Disease Control and Prevention multipliers to estimate the annual number of illnesses associated with flour-borne pathogens. We then integrated illness severity data with an updated economic model that accounts for costs related to medical care, productivity loss, loss of life, along with the quality of life loss that entails pain and suffering. In total, 752 cases and 223 hospitalizations from flour-related illnesses were reported from 2001 to 2021, with an average of 37.6 cases of reported cases annually. However, the actual number of cases, accounting for underreporting and underdiagnosis, can be as high as 19,440 annually. Pathogens involved in these outbreaks are Salmonella, E. coli O157:H7, and E. coli O121. Our estimates suggest average annual economic losses, including healthy years of life lost, of $108 and $258 million using two alternative models.

Estimating the health burden of foodborne gastroenteritis caused by non-typhoidal Salmonella enterica and Vibrio parahaemolyticus in Zhejiang province, China.

As acute gastrointestinal (AGI) disease patients only sometimes seek medical care or submit stool samples for testing, and laboratories cannot detect or identify the pathogen, most cases of foodborne gastroenteritis still need to be identified through public health monitoring. We conducted a population survey and sentinel hospital surveillance to determine the burden of foodborne gastroenteritis caused by non-typhoidal Salmonella enterica (NTS) and Vibrio parahaemolyticus infection, from July 2018 to June 2019 in Zhejiang province, China, and a model for calculating disease burden established. Using the burden of illness pyramid model, we estimated that there were 140.3 cases of NTS infection and 136.2 cases of V. parahaemolyticus infection. We estimated annual incidence per 100,000 population in Zhejiang province as 236 (95% confidence interval [CI] 208-267) and 206 (95% CI 155-232) cases for foodborne NTS and V. parahaemolyticus gastroenteritis, respectively. The results show that AGI caused by these two pathogens constitutes a substantial burden in the Zhejiang population. The health burden of AGI estimations caused by NTS and V. parahaemolyticus in this study can serve as a strategic framework to direct policy and intervention.

The economic and humanistic burden of bipolar disorder in adults in the United States.

BACKGROUND: Bipolar disorder is associated with functional impairment and diminished health-related quality of life (HRQoL). The purpose of this study was to estimate the annual per patient direct healthcare costs, indirect costs, and HRQoL of patients with bipolar disorder by depressive symptom severity and overall compared to the general population in the US. METHODS: This cross-sectional study used self-reported data from the 2020 US National Health and Wellness Survey. Adult respondents who reported bipolar disorder symptoms in the past 12 months and/or a diagnosis of bipolar disorder were identified (bipolar disorder cohort) and were further classified by depressive symptom severity based on Patient Health Questionnaire (PHQ-9) scores (none/mild = 0-9, moderate = 10-14, severe = 15-27). Annualized direct healthcare costs and indirect costs were calculated from 6-month healthcare resource utilization and work productivity, respectively. A general population cohort was constructed using 2:1 propensity score matching. Multivariate regression models of all-cause hospitalizations in the past 6 months, annualized direct healthcare costs, annualized indirect costs, and HRQoL (eg, EuroQol 5-Dimension Health Questionnaire (EQ-5D)) controlled for confounders (demographic and clinical characteristics). RESULTS: Of 3583 adults meeting pre-specified criteria for bipolar disorder, 1401 (39.1%) reported none/mild, 889 (24.8%) moderate, and 1293 (36.1%) severe depressive symptom severity. Additionally, 3285 (91.7%) were matched to 6570 adults in the general population. Compared to the general population, adjusted mean hospitalizations (0.53 vs. 0.30), annualized per patient direct healthcare costs ($20,846 vs. $11,391), and indirect costs ($14,795 vs. $9274) were significantly greater for the bipolar disorder cohort (all p < 0.001); adjusted HRQoL (EQ-5D: 0.69 vs. 0.79) was significantly worse (p < 0.001). By depressive symptom severity, adjusted mean hospitalizations (none/mild = 0.30, moderate = 0.50, severe = 0.46), direct healthcare costs ($14,389, $22,302, $21,341), and indirect costs ($10,799, $17,109, $18,470) were significantly greater for moderate and severe compared to none/mild depressive symptom severity (all p < 0.01); adjusted HRQoL (EQ-5D: 0.77, 0.67, 0.59) was significantly worse (p < 0.001). CONCLUSIONS: Among respondents with bipolar disorder, those with moderate to severe depression had greater direct healthcare costs and indirect costs as well as worse HRQoL than those with mild or no depressive symptoms. Treatment targeting reduction in depressive symptoms may reduce the economic and humanistic burden of bipolar disorder.

Effects of Client-Centered Occupational Therapy on Behavioral Psychological Symptoms, Social Interaction, Occupational Performance, Quality of Life, and Caregiver Burden among the Individuals with Dementia.

This study aimed to verify the effects of client-centered occupational therapy on individuals with dementia. Twenty participants were randomly assigned to an experimental group (client-centered occupational therapy) and a control group (general occupational therapy). A pretest, post-test, and follow-up tests were used to compare the effects of the intervention. The experimental group had significant changes in all variables, and the control group showed significant differences in verbal social interaction, quality of life, and burden of caregivers (p < 0.05). As a result of repeated measurement variance analysis, it was found that there was a significant effect within the group except for the NPI-Q distress items (p < 0.05), but the effect was not verified in all areas between the groups. Client-centered customized occupational therapy can be more effective than general occupational therapy in problem behavior, social interaction, quality of life, caregiver burden, and occupational performance of individuals with dementia.

The burden of illness in patients with paroxysmal nocturnal hemoglobinuria receiving treatment with the C5-inhibitors eculizumab or ravulizumab: results from a US patient survey.

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening disease with symptoms of hemolysis and thrombosis. Current therapies for this complement-mediated disease rely predominantly on inhibition of the C5 complement protein. However, data on treatment responses and quality of life in C5-inhibitor (C5i)-treated PNH patients are scarce. The objective of this study was to determine C5i treatment effects on clinical parameters, PNH symptoms, quality of life, and resource use for PNH patients. This cross-sectional study surveyed 122 individuals in the USA receiving treatment for PNH with C5-targeted monoclonal antibodies, eculizumab (ECU) or ravulizumab (RAV). Despite most patients receiving C5i therapy for ≥ 3 months (ECU 100%, n = 35; RAV 95.4%, n = 83), many patients remained anemic with hemoglobin levels ≤ 12 g/dL in 87.5% (n = 28/32) and 82.9% (n = 68/82) of ECU and RAV recipients, respectively. A majority of patients on ECU (88.6%; n = 31/35) and RAV (74.7%; n = 65/87) reported fatigue symptoms. Among PNH patients receiving C5i therapy for ≥ 12 months, some still reported thrombotic events (ECU, 10.0%, n = 1/10; RAV, 23.5%, n = 4/17) and required transfusions within the past year (ECU, 52.2%, n = 12/23; RAV, 22.6%, n = 7/31). Other patient-reported PNH symptoms included breakthrough hemolysis, shortness of breath, and headaches. Patients reported scores below the average population norms on the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue and European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) scales. Overall, this study found that PNH patients receiving ECU or RAV therapy demonstrated a significant burden of illness, highlighting the need for improved PNH therapies.

Multicenter, cross-sectional study of the costs of illness and cost-driving factors in adult patients with epilepsy.

OBJECTIVE: This study was undertaken to quantify epilepsy-related costs of illness (COI) in Germany and identify cost-driving factors. METHODS: COI were calculated among adults with epilepsy of different etiologies and severities. Multiple regression analysis was applied to determine any epilepsy-related and sociodemographic factors that serve as cost-driving factors. RESULTS: In total, 486 patients were included, with a mean age of 40.5 ± 15.5 years (range = 18-83 years, 58.2% women). Mean 3-month COI were estimated at €4911, €2782, and €2598 for focal, genetic generalized, and unclassified epilepsy, respectively. The mean COI for patients with drug-refractory epilepsy (DRE; €7850) were higher than those for patients with non-DRE (€4720), patients with occasional seizures (€3596), or patients with seizures in remission for >1 year (€2409). Identified cost-driving factors for total COI included relevant disability (unstandardized regression coefficient b = €2218), poorer education (b = €2114), living alone (b = €2612), DRE (b = €1831), and frequent seizures (b = €2385). Younger age groups of 18-24 years (b = -€2945) and 25-34 years (b = -€1418) were found to have lower overall expenditures. A relevant disability (b = €441), DRE (b = €1253), frequent seizures (b = €735), and the need for specialized daycare (b = €749) were associated with higher direct COI, and poorer education (b = €1969), living alone (b = €2612), the presence of a relevant disability (b = €1809), DRE (b = €1831), and frequent seizures (b = €2385) were associated with higher indirect COI. SIGNIFICANCE: This analysis provides up-to-date COI data for use in further health economics analyses, highlighting the high economic impacts associated with disease severity, disability, and disease-related loss of productivity among adult patients with epilepsy. The identified cost drivers could be used as therapeutic and socioeconomic targets for future cost-containment strategies.

A retrospective longitudinal cohort study of the clinical burden in myasthenia gravis.

BACKGROUND: Patients with generalized myasthenia gravis (MG) often experience debilitating exacerbations, with the possibility of life-threatening respiratory crises requiring hospitalization. Long-term longitudinal studies are needed to understand the burden of MG, including in patients whose disease is refractory to conventional treatment. METHODS: A retrospective, longitudinal, cohort study was conducted of patients in England aged ≥ 18 years with treatment-refractory or non-refractory MG, using data recorded during 1997-2016 in the Clinical Practice Research Datalink and the Hospital Episode Statistics databases. A control cohort of patients without MG, matched to the patients in the treatment-refractory MG cohort, was also identified. Outcome measures included myasthenic crises, MG exacerbations, MG-related hospitalizations, comorbidities, and all-cause mortality. Descriptive statistics were calculated for the overall MG population. For continuous variables, between-cohort comparisons were made using t tests for normally distributed data and Mann-Whitney U tests for non-normally distributed data. For categorical data, the comparisons were made by chi-squared tests. Differences in clinical outcomes between cohorts were modeled using negative binomial regression. RESULTS: A total of 1149 patients with MG were included. Overall, 18.4% of patients experienced myasthenic crises, 24.6% experienced exacerbations, and 38.6% underwent MG-related hospitalizations. Most of these events occurred within 2-3 years of diagnosis. Patients with MG refractory to conventional treatment (n = 66) experienced more exacerbations and MG-related hospitalizations than patients with non-refractory disease (n = 1083). Patients with refractory MG experienced a higher frequency of renal disease and hypertension compared with patients with non-refractory MG, and with matched patients without MG. They were also more likely to have diabetes and congestive heart failure than the matched controls. Rates of all-cause mortality during the follow-up period did not differ between patients with refractory MG and non-refractory MG. CONCLUSIONS: These results show that conventional treatments for MG are not adequately managing patients' symptoms and that patients with refractory MG are more likely to experience certain comorbidities than those with non-refractory MG or matched controls without MG. Future research should focus on the impact of newer targeted therapies on long-term clinical outcomes and comorbid conditions.

An estimate of the economic burden of venous leg ulcers associated with deep venous disease.

INTRODUCTION: Venous leg ulcers (VLU) embody the most severe stage of the broad spectrum of chronic venous disease. Approximately 40% of patients with VLU present with the underlying deep venous disease (DVD). Although the data are scarce, these deep venous disease-related VLU (DRV) are thought to have higher recurrence rates and a substantial economic burden. The objective of this study was to assess the economic burden of DRV across Australia, France, Germany, Italy, Spain, the UK, and the USA. METHODS: A comprehensive literature review was undertaken to identify publications documenting the incidence and prevalence of VLU and DRV, medical resource utilization, and associated costs of DRV. Findings from this literature review were used to estimate the economic burden of illness, including direct medical costs over a 12-month interval following initial presentation of a newly formed DRV. RESULTS: Total annual incidence of new or recurrent DRV in Australia, France, Germany, Italy, Spain, UK, and the US are estimated at 122,000, 263,000, 345,000, 253,000, 85,000, 230,000, and 643,000 events, respectively, in 2019. Incidence ranges from 0.73 to 3.12 per 1000 persons per year. The estimated annual direct medical costs for patients managed conservatively in these geographies total ~ $10.73 billion (USD) or $5527 per person per year. CONCLUSION: The availability of published data on the costs of VLU care varies widely across countries considered in this analysis. Although country-specific VLU practice patterns vary, there is a uniform pattern of high-cost care.

Characterisation of burden of illness measures associated with human (Fluoro)quinolone-resistant Campylobacter spp. infections - a scoping review.

Campylobacter spp. are one of the most common causes of bacterial gastroenteritis in Canada and worldwide. Fluoroquinolones are often used to treat complicated human campylobacteriosis and strains of Campylobacter spp. resistant to these drugs are emerging along the food chain. A scoping review was conducted to summarise how human (fluoro)quinolone-resistant (FQR; quinolones including fluoroquinolones) Campylobacter spp. infections are characterised in the literature by describing how burden of illness (BOI) associated with FQR is measured and reported, describing the variability in reporting of study characteristics, and providing a narrative review of literature that compare BOI measures of FQR Campylobacter spp. infections to those with susceptible infections. The review identified 26 studies that yielded many case reports, a lack of recent literature and a lack of Canadian data. Studies reported 26 different BOI measures and the most common were hospitalisation, diarrhoea, fever and duration of illness. There were mixed results as BOI measures reported in literature were inconsistently defined and there were limited comparisons between resistant and susceptible infections. This presents a challenge when attempting to assess the magnitude of the BOI due to FQR Campylobacter spp., highlighting the need for more research in this area.

Epidemiology, healthcare resource use, and mortality in patients with probable Dravet syndrome: A population-based study on German health insurance data.

OBJECTIVE: Ten-year retrospective study to assess burden of illness in patients with probable Dravet syndrome (DS) identified from German healthcare data. METHODS: In the absence of an International Classification of Diseases code, patients with probable DS were identified using a selection algorithm considering diagnoses and drug prescriptions. Primary analyses were prevalence and demographics; secondary analyses included healthcare costs, annual hospitalization rate (AHR) and length of stay (LOS), medication use, and mortality. RESULTS: In the final study year, 64 patients with probable DS (mean [range] age: 33.2 [3-82] years; male: 48%) were identified. Prevalence: 4.7 per 100,000 people. During the study, 160 patients with probable DS were identified and followed up for 1,261 patient-years. Mean cost of healthcare was €11,048 per patient-year (PPY), mostly attributable to inpatient care (47%), medication (26%), and services and devices (19%). Annual healthcare costs were significantly greater for those with prescribed rescue medication (15% of patient-years) vs. without (€16,123 vs. €10,125 PPY, p < 0.001). Mean (standard deviation [SD]) AHR and LOS were 1.1 (1.7) and 17.5 (33.5) days PPY. AHR was significantly greater in patients with prescribed rescue medication vs. without (1.6 [2.0] vs. 1.0 [1.6] PPY, p < 0.001). Mean (SD) number of antiseizure medications prescribed was 2.6 (1.2) PPY and 5.0 (2.5) over the entire observable time for each patient. Mortality rate was significantly higher for probable DS vs. matched controls (11.88% [19 events] vs. 1.19% [172 events], p < 0.001). CONCLUSION: Probable DS is associated with substantial healthcare costs in Germany.