The burden of multiple sclerosis in Japan

Aims: Multiple sclerosis (MS) is a disabling autoimmune disease affecting the central nervous system. Few studies have examined the effect of MS on patient outcomes in Japan. The study aim was to quantify MS burden in Japan by comparing MS respondents to matched controls on patient outcomes.

Materials and methods: Data from seven administrations of the nationally representative Japan National Health and Wellness Survey (2009–2014 and 2016) were used (n?=?181,423). Respondents self-reporting MS diagnosis were compared with respondents not reporting MS. Matched controls were selected using propensity scores. Respondents with MS and matched controls were compared on health-related quality-of-life (HRQoL), work productivity and activity impairment, healthcare resource utilization, and costs. Comparisons were made using Chi-square tests or one-way ANOVAs.

Results: A total of 96 respondents with MS and 480 matched controls were included in the analyses. MS respondents reported worse mental (44.35 vs 47.51, p?p?p?p?p?p?p?Limitations: Japan NHWS data are cross-sectional, and causal relationships cannot be established. Due to the self-reported nature of the data, responses could not be independently verified.

Conclusions: Results suggest MS in Japan is associated with poorer HRQoL and greater work and activity impairment, healthcare resource use, and costs. Improved MS management could benefit both patients and society.     

A Unifying Framework for Marginalised Random‐Intercept Models of Correlated Binary Outcomes

We demonstrate that many current approaches for marginal modelling of correlated binary outcomes produce likelihoods that are equivalent to the copula‐based models herein. These general copula models of underlying latent threshold random variables yield likelihood‐based models for marginal fixed effects estimation and interpretation in the analysis of correlated binary data with exchangeable correlation structures. Moreover, we propose a nomenclature and set of model relationships that substantially elucidates the complex area of marginalised random‐intercept models for binary data. A diverse collection of didactic mathematical and numerical examples are given to illustrate concepts. Copyright © 2013 John Wiley & Sons, Ltd.     

The burden of preserved ejection fraction heart failure in a real-world Swedish patient population

Objectives:

To evaluate resource use and associated costs in patients with a diagnosis of heart failure with preserved ejection fraction (HF-PEF) in Sweden.

Methods:

This retrospective study identified real-world patients with an ICD-10 diagnosis code for heart failure (I50) for the period between July 1, 2005 and December 31, 2006 from electronic medical records of primary care centers in Uppsala County Council, and in the Swedish patient registry data. Patients were categorized as having HF-PEF (left ventricle ejection fraction [LVEF] > 50%) during the index period. The study assessed medication utilization, outpatient visits, hospitalizations, and associated healthcare costs, as well as the incidence rates and time to all-cause and heart failure mortality following the index period.

Results:

The study included 137 HF-PEF patients with a mean age of 77.1 (SD?=?9.1) years. Over 50% of HF-PEF patients were female and hypertensive. Nearly all patients received ≥1 medication post-index. Patients had an average of 1.5 heart failure related hospitalizations per follow-up year. The average annual per patient cost for the management of a HF-PEF patient was found in Sweden to be Swedish Krona (SEK) 108,246 (EURO [EUR] 11,344). Hospitalizations contributed to more than 80% of the total cost. All-cause mortality over the 18-month study period was 25.5%, and more than 50% of these deaths occurred within 1 year of index.

Limitations:

Due to the limitations of registry data, it is not possible to confirm the HF diagnosis, and therefore the accuracy of registry records must be assumed. Other factors such as short follow-up time, the study-mandated LVEF assessment, and a lack of drug duration data may also have an impact on the study results.

Conclusions:

All-cause mortality was high in the HF-PEF population, with more than half of patients dying within 1 year of study follow-up. Study results also indicate that 60% of HF-PEF patients have ≥1 hospitalization during follow-up. Hospitalizations, especially heart failure related admissions, represent a substantial proportion of the total healthcare burden of patients with HF-PEF in Sweden.     

The burden of immune thrombocytopenia in adults: Evaluation of the thrombopoietin receptor agonist romiplostim

Abstract

Background:

Immune thrombocytopenia (ITP) is a chronic, immune-mediated disease characterized by a transient or long-lasting decrease in platelet counts. ITP is associated with numerous serious clinical consequences. Discussed here are clinical aspects of ITP, the humanistic and economic burden of ITP, and current treatment options with a focus on romiplostim, a thrombopoietin (TPO) receptor agonist. The aim of this review is to provide decision-makers with the background information necessary to evaluate the value of romiplostim.

Scope:

PubMed was searched for relevant, English-language papers published from January 2006 through November 2011 relating to the epidemiology and treatment options of chronic ITP, and, focusing on the TPO mimetic romiplostim, patient-reported outcomes (PRO) and economic burden. Recent select conference abstracts were also reviewed.

Findings:

The initial clinical management of ITP (e.g., corticosteroids, immunoglobulins) is often associated with adverse events and recommended for short-term use only. Splenectomy, a potentially curative second-line treatment, is associated with increased risks of bleeding and infection, and patients often require additional long-term drug intervention. ITP and its sequelae are associated with a substantial burden on patients’ health-related quality-of-life (HRQoL) and increased medical costs. Use of TPO receptor agonists in ITP patients may represent a more efficient use of healthcare resources than existing therapies.

Conclusion:

While this literature review is not a systematic review, e.g., it considers only approved therapies and published literature written in English, it provides a comprehensive overview of the clinical, humanistic, and economic factors that should be considered in treating ITP, particularly with new agents such as romiplostim. Among the limited number of safe and effective therapies currently available for chronic ITP, highly effective and well-tolerated medications such as romiplostim may reduce the healthcare resource utilization associated with ITP while improving patients’ HRQoL.     

The Impact of Self-Efficacy on Job Outcomes of Hotel Employees

Abstract

This study aims to examine the effect of self-efficacy on job performance, job satisfaction, and affective organizational commitment using data collected from employees in three-, four-, and five-star hotels in Northern Cyprus as its setting. The research hypotheses were tested using LISREL 8.30 through path analysis. The model test results demonstrated that self-efficacy is a significant determinant of job performance. This study, however, failed to find a significant positive association between self-efficacy and job satisfaction. Although not hypothesized, the results of the present study revealed that job performance mediates the impact of self-efficacy on job satisfaction. In addition, the results of the path analysis showed that self-efficacy is among the significant predictors of affective organizational commitment. The model test results provided empirical support for the rest of the hypothesized relationships. Specifically, the path-analytic findings indicated that job satisfaction exerts a significant positive influence on affective organizational commitment. The model test results also demonstrated that job satisfaction and affective organizational commitment are negatively associated with intention to leave. Discussion of the results, implications for hotel managers, and future research directions are presented in the study.     

Clinical and economic benefit of enzymatic debridement of pressure ulcers compared to autolytic debridement with a hydrogel dressing

Abstract

Objective:

The purpose of this study was to determine the cost-effectiveness of enzymatic debridement using collagenase relative to autolytic debridement with a hydrogel dressing for the treatment of pressure ulcers.

Methods:

A 3-stage Markov model was used to determine the expected costs and outcomes of wound care for collagenase and hydrogel dressings. Outcome data used in the analysis were taken from a randomized clinical trial that directly compared collagenase and hydrogel dressings. The primary outcome in the clinical trial was the proportion of patients achieving a closed epithelialized wound. Transition probabilities for the Markov states were estimated from the clinical trial. A 1-year time horizon was used to determine the expected number of closed wound days and the expected costs for the two alternative debridement therapies. Resource utilization was based on the wound care treatment regimen used in the clinical trial. Resource costs were derived from standard cost references and medical supply wholesalers. The economic perspective taken was that of the long-term care facility. No cost discounting was performed due to the short time horizon of the analysis. A deterministic sensitivity analysis was conducted to analyze economic uncertainty.

Results:

The number of expected wound days for the collagenase and hydrogel cohorts are estimated at 48 and 147, respectively. The expected direct cost per patient for pressure ulcer care was $2003 for collagenase and $5480 for hydrogel debridement. The number of closed wound days was 1.5-times higher for collagenase (317 vs 218 days) than with the hydrogel. The estimated cost/closed wound day was 4-times higher for the hydrogel ($25) vs collagenase ($6).

Conclusions:

In this Markov model based on a randomized trial of pressure ulcer care in a long-term care setting collagenase debridement was economically dominant over autolytic debridement, yielding better outcomes at a lower total cost. Since it was a single institution study with a small sample size, the results should be interpreted with caution. Specifically, the findings may not necessarily be generalized to other hydrogel dressings, healthcare settings, age groups, or to wounds of other etiologies.     

Randomized,open-label study to evaluate patient-reported outcomes with fingolimod after changing from prior disease-modifying therapy for relapsing multiple sclerosis: EPOC study rationale and design

Abstract

Objective:

The study to Evaluate Patient OutComes, Safety, and Tolerability of Fingolimod (EPOC; NCT01216072) aimed to test the hypothesis that therapy change to oral Gilenya (Novartis AG, Stein, Switzerland) (fingolimod) improves patient-reported outcomes compared with standard-of-care disease-modifying therapy (DMT) in patients with relapsing multiple sclerosis; safety and tolerability were also assessed. This communication describes the study rationale and design.

Methods:

EPOC is a phase 4, open-label, multi-center study conducted in the US and Canada of patients with relapsing forms of multiple sclerosis who are candidates for therapy change. Therapy change eligibility was determined by the treating physician (US patients) or required an inadequate response to or poor tolerance for at least 1 MS therapy (Canadian patients). Patients were randomly assigned in a 3:1 ratio to 6 months of treatment with once-daily oral fingolimod 0.5?mg or standard-of-care DMTs. The primary study end-point was the change from baseline in treatment satisfaction as determined by the global satisfaction sub-scale of the Treatment Satisfaction Questionnaire for Medication. Secondary end-points included changes from baseline in perceived effectiveness and side-effects, and measures of activities of daily living, fatigue, depression, and quality-of-life. A 3-month open-label fingolimod extension was available for patients randomly assigned to the DMT group who successfully completed all study visits.

Results:

Enrollment has been completed with 1053 patients; the patient population is generally older and has a longer duration of disease compared with populations from phase 3 studies of fingolimod.

Limitations:

Inclusion criteria selected for patients with a sub-optimal experience with a previous DMT, limiting the collection of data on therapy change in patients who were satisfied with their previous DMT.

Conclusions:

Results of the EPOC study are anticipated in early 2013 and will inform treatment selection by providing patient-centered data on therapy switch to fingolimod or standard-of-care DMTs.

Trial Registration:

ClinicalTrials.gov NCT01216072.     

The importance of HR practices and workplace trust in achieving superior performance: A study of public-sector organizations

This paper assesses the impact of bundles of HR practices on workplace trust, job satisfaction, commitment, effort and perceived organizational performance. A theoretical model is developed and tested using data collected through a postal survey of UK local government employees. The results support the hypothesis that HR practices are powerful predictors of trust and organizational performance. These findings demonstrate the need for public organizations to re-evaluate their current battery of HR practices in an attempt to improve overall performance.     

Relationship between patient-reported disease severity and other clinical outcomes in osteoarthritis: a European perspective

Abstract

Objective:

To determine how patient-rated osteoarthritis (OA) severity correlates with other patient-reported and clinical outcomes in the European clinical setting.

Methods:

We used the Adelphi Arthritis VII (2008) Disease Specific Program (DSP). OA severity was patient-rated using the question ‘How bad would you say your arthritis is now?’ with responses of ‘mild,’ ‘moderate,’ and ‘severe.’ Patient-reported outcomes included a 0–100?mm pain visual analogue scale (VAS); questions on daily functioning; Work Productivity and Activity Impairment (WPAI) scale; and EuroQoL (EQ-5D). Regression models and chi-square analyses evaluated relationships between self-rated OA severity and other outcomes.

Results:

Patient-reported data were available from 1739 individuals (63.1% female, mean age 64.4 [standard deviation 11.9] years) from France, Germany, Italy, Spain, and the UK. With increasing OA severity; mild (24.5%), moderate (56.3%), severe (19.2%), statistically significant differences (p?<?0.05) were observed with higher pain VAS scores (28.3, 49.9, 69.2, respectively), reduced function, and greater overall work impairment due to OA (24.3%, 38.5%, 68.6%, respectively). Significant associations of patient-reported OA severity with function and health status were indicated, including the EQ-5D health state index; 0.77 (mild), 0.62 (moderate), 0.30 (severe) (p?<?0.0001). Physicians tended to overestimate patients who rated their OA as mild, and underrate patients who rated their OA as severe.

Conclusions:

In five European countries, patient-rated OA severity was associated with other patient-reported outcomes, and may be of benefit in the clinical setting when choosing treatment options aimed at improving pain, function and productivity, providing an accurate and tangible assessment of patient’s perceptions of their disease.