A randomized clinical trial of isolated ambulatory phlebectomy versus saphenous thermal ablation with concomitant phlebectomy (SAPTAP Trial).

BACKGROUND: Current treatment of patients with saphenous trunk and tributary incompetence consists of truncal ablation with concomitant, delayed or no treatment of the tributary. However, reflux of the saphenous trunk may be reversible after treatment of the incompetent tributary. The aim of this study was to determine whether single ambulatory phlebectomy with or without delayed endovenous truncal ablation (SAP) is non-inferior to thermal endovenous ablation with concomitant phlebectomy (TAP), and whether SAP is a cost-effective alternative to TAP. METHODS: A multicentre, non-inferiority RCT was conducted in patients with an incompetent great saphenous vein or anterior accessory saphenous vein with one or more incompetent tributaries. Participants were randomized to receive SAP or TAP. After 9 months, additional truncal treatment was considered for SAP patients with remaining symptoms. The primary outcome was VEnous INsufficiency Epidemiological and Economic Study Quality of Life/Symptoms (VEINES-QOL/Sym score) after 12 months. Secondary outcomes were, among others, cost-effectiveness, perceived improvement of symptoms, and anatomical success. RESULTS: Some 464 patients received the allocated treatment (SAP 227, TAP 237). VEINES-QOL scores were 52.7 (95 per cent c.i. 51.9 to 53.9) for SAP and 53.8 (53.3 to 55.1) for TAP; VEINES-Sym scores were 53.5 (52.6 to 54.4) and 54.2 (54.0 to 55.6) respectively. Fifty-eight patients (25.6 per cent) in the SAP group received additional truncal ablation. Treatment with SAP was less costly than treatment with TAP. CONCLUSION: One year after treatment, participants who underwent SAP had non-inferior health-related quality of life compared with those who had TAP. Treatment with SAP was a cost-effective alternative to TAP at 12 months. REGISTRATION NUMBER: NTR 4821 (www.trialregister.nl).

Stratification of COPD patients towards personalized medicine: reproduction and formation of clusters.

BACKGROUND: The global initiative for chronic obstructive lung disease (GOLD) 2020 emphasizes that there is only a weak correlation between FEV1, symptoms and impairment of the health status of patients with chronic obstructive pulmonary disease (COPD). Various studies aimed to identify COPD phenotypes by cluster analyses, but behavioral aspects besides smoking were rarely included. METHODS: The aims of the study were to investigate whether (i) clustering analyses are in line with the classification into GOLD ABCD groups; (ii) clustering according to Burgel et al. (Eur Respir J. 36(3):531-9, 2010) can be reproduced in a real-world COPD cohort; and (iii) addition of new behavioral variables alters the clustering outcome. Principal component and hierarchical cluster analyses were applied to real-world clinical data of COPD patients newly referred to secondary care (n = 155). We investigated if the obtained clusters paralleled GOLD ABCD subgroups and determined the impact of adding several variables, including quality of life (QOL), fatigue, satisfaction relationship, air trapping, steps per day and activities of daily living, on clustering. RESULTS: Using the appropriate corresponding variables, we identified clusters that largely reflected the GOLD ABCD groups, but we could not reproduce Burgel's clinical phenotypes. Adding six new variables resulted in the formation of four new clusters that mainly differed from each other in the following parameters: number of steps per day, activities of daily living and QOL. CONCLUSIONS: We could not reproduce previously identified clinical COPD phenotypes in an independent population of COPD patients. Our findings therefore indicate that COPD phenotypes based on cluster analysis may not be a suitable basis for treatment strategies for individual patients.

e-Monitoring of Asthma Therapy to Improve Compliance in children (e-MATIC): a randomised controlled trial.

Real-time medication monitoring (RTMM) is a promising tool for improving adherence to inhaled corticosteroids (ICS), but has not been sufficiently tested in children with asthma. We aimed to study the effects of RTMM with short message service (SMS) reminders on adherence to ICS, asthma control, asthma-specific quality of life and asthma exacerbation rate; and to study the associated cost-effectiveness.In a multicentre, randomised controlled trial, children (aged 4-11 years) using ICS were recruited from five outpatient clinics and were given an RTMM device for 12 months. The intervention group also received tailored SMS reminders, sent only when a dose was at risk of omission. Outcome measures were adherence to ICS (RTMM data), asthma control (childhood asthma control test questionnaire), quality of life (paediatric asthma quality of life questionnaire) and asthma exacerbations. Costs were calculated from a healthcare and societal perspective.We included 209 children. Mean adherence was higher in the intervention group: 69.3% versus 57.3% (difference 12.0%, 95% CI 6.7%-17.7%). No differences were found for asthma control, quality of life or asthma exacerbations. Costs were higher in the intervention group, but this difference was not statistically significant.RTMM with tailored SMS reminders improved adherence to ICS, but not asthma control, quality of life or exacerbations in children using ICS for asthma.

Mapping the clinical chronic obstructive pulmonary disease questionnaire onto generic preference-based EQ-5D values.

OBJECTIVES: To develop a model to predict EuroQol five-dimensional questionnaire (EQ-5D) values from clinical chronic obstructive pulmonary disease (COPD) questionnaire (CCQ) scores. METHODS: We used data from three clinical trials (the Randomized Clinical Trial on Effectiveness of Integrated COPD Management in Primary Care [RECODE], the Assessment Of Going Home Under Early Assisted Discharge [GO-AHEAD], and the Health Status Guided COPD Care [MARCH]). Data were randomly split into an estimation sample and a validation sample. The conceptual similarity between patient-reported CCQ and preference-based EQ-5D scores was assessed using correlation and principal-component analysis. Different types of models were estimated with increasing complexity. We selected the final models on the basis of mean absolute error and root mean square error when comparing predicted and observed values from the same population (internal validity) and from different trial populations (external validity). We also developed models for different country-specific EQ-5D value sets. RESULTS: The principal-component analysis showed that the CCQ domains functional state and mental state are associated with four dimensions of the EQ-5D. The EQ-5D dimension pain/discomfort formed a separate construct on which no CCQ item loaded. The mean observed EQ-5D values were not significantly different from the mean predicted EQ-5D values in internal validation samples but did significantly differ in external validation samples. The models underestimated EQ-5D values in milder health states and overestimated them in more severe health states. The predictive ability of the models was similar across different EQ-5D value sets. CONCLUSIONS: The models can predict mean EQ-5D values that are similar to observed mean values in a similar population. The overestimating/underestimating of the low/high EQ-5D values, however, limits its use in Markov models. Therefore, mapping should be used cautiously.

Should I stay or should I go home? A latent class analysis of a discrete choice experiment on hospital-at-home.

OBJECTIVES: This study aimed 1) to quantify the strength of patient preferences for different aspects of early assisted discharge in The Netherlands for patients who were admitted with a chronic obstructive pulmonary disease exacerbation and 2) to illustrate the benefits of latent class modeling of discrete choice data. This technique is rarely used in health economics. METHODS: Respondents made multiple choices between hospital treatment as usual (7 days) and two combinations of hospital admission (3 days) followed by treatment at home. The latter was described by a set of attributes. Hospital treatment was constant across choice sets. Respondents were patients with chronic obstructive pulmonary disease in a randomized controlled trial investigating the cost-effectiveness of early assisted discharge and their informal caregivers. The data were analyzed using mixed logit, generalized multinomial logit, and latent-class conditional logit regression. These methods allow for heterogeneous preferences across groups, but in different ways. RESULTS: Twenty-five percent of the respondents opted for hospital treatment regardless of the description of the early assisted discharge program, and 46% never opted for the hospital. The best model contained four latent classes of respondents, defined by different preferences for the hospital and caregiver burden. Preferences for other attributes were constant across classes. Attributes with the strongest effect on choices were the burden on informal caregivers and co-payments. Except for the number of visits, all attributes had a significant effect on choices in the expected direction. CONCLUSIONS: Considerable segments of respondents had fixed preferences for either treatment option. Applying latent class analysis was essential in quantifying preferences for attributes of early assisted discharge.

Does the 2013 GOLD classification improve the ability to predict lung function decline, exacerbations and mortality: a post-hoc analysis of the 4-year UPLIFT trial.

BACKGROUND: The 2013 GOLD classification system for COPD distinguishes four stages: A (low symptoms, low exacerbation risk), B (high symptoms, low risk), C (low symptoms, high risk) and D (high symptoms, high risk). Assessment of risk is based on exacerbation history and airflow obstruction, whatever results in a higher risk grouping. The previous system was solely based on airflow obstruction. Earlier studies compared the predictive performance of new and old classification systems with regards to mortality and exacerbations. The objective of this study was to compare the ability of both classifications to predict the number of future (total and severe) exacerbations and mortality in a different patient population, and to add an outcome measure to the comparison: lung function decline. METHODS: Patient-level data from the UPLIFT trial were used to analyze 4-year survival in a Weibull model, with GOLD stages at baseline as covariates. A generalized linear model was used to compare the numbers of exacerbations (total and severe) per stage. Analyses were repeated with stages C and D divided into substages depending on lung function and exacerbation history. Lung function decline was analysed in a repeated measures model. RESULTS: Mortality increased from A to D, but there was no difference between B and C. For the previous GOLD stages 2-4, survival curves were clearly separated. Yearly exacerbation rates were: 0.53, 0.72 and 0.80 for stages 2-4; and 0.35, 0.45, 0.58 and 0.74 for A-D. Annual rates of lung function decline were: 47, 38 and 26 ml for stages 2-4 and 44, 48, 38 and 39 for stages A-D. With regards to model fit, the new system performed worse at predicting mortality and lung function decline, and better at predicting exacerbations. Distinguishing between the sub-stages of high-risk led to substantial improvements. CONCLUSIONS: The new classification system is a modest step towards a phenotype approach. It is probably an improvement for the prediction of exacerbations, but a deterioration for predicting mortality and lung function decline. TRIAL REGISTRATION: ClinicalTrials.gov NCT00144339 (September 2, 2005).

Cost-effectiveness of early assisted discharge for COPD exacerbations in The Netherlands.

OBJECTIVES: Hospital admissions for exacerbations of chronic obstructive pulmonary disease are the main cost drivers of the disease. An alternative is to treat suitable patients at home instead of in the hospital. This article reports on the cost-effectiveness and cost-utility of early assisted discharge in The Netherlands. METHODS: In the multicenter randomized controlled Assessment of GOing Home under Early Assisted Discharge trial (n = 139), one group received 7 days of inpatient hospital treatment (HOSP) and one group was discharged after 3 days and treated at home by community nurses for 4 days. Health care resource use, productivity losses, and informal care were recorded in cost questionnaires. Microcosting was performed for inpatient day costs. RESULTS: Seven days after admission, mean change from baseline Clinical Chronic Obstructive Pulmonary Disease Questionnaire score was better for HOSP, but not statistically significantly: 0.29 (95% confidence interval [CI]-0.04 to 0.61). The difference in the probability of having a clinically relevant improvement was significant in favor of HOSP: 19.0%-point (95% CI 0.5%-36.3%). After 3 months of follow-up, differences in effectiveness had almost disappeared. The difference in quality-adjusted life-years was 0.0054 (95% CI-0.021 to 0.0095). From a health care perspective, early assisted discharge was cost saving:-€244 (treatment phase, 95% CI-€315 to-€168) and-€168 (3 months, 95% CI-€1253 to €922). Societal perspective:-€65 (treatment phase, 95% CI-€152 to €25) and €908 (3 months, 95% CI-€553 to €2296). The savings per quality-adjusted life-year lost were €31,111 from a health care perspective. From a societal perspective, HOSP was dominant. CONCLUSIONS: No clear evidence was found to conclude that either treatment was more effective or less costly.

Evaluation of health care providers' role transition and satisfaction in hospital-at-home for chronic obstructive pulmonary disease exacerbations: a survey study.

BACKGROUND: Hospital-at-home is an accepted alternative for usual hospital treatment for patients with a Chronic Obstructive Pulmonary Disease (COPD) exacerbation. The introduction of hospital-at-home may lead to changes in health care providers' roles and responsibilities. To date, the impact on providers' roles is unknown and in addition, little is known about the satisfaction and acceptance of care providers involved in hospital-at-home. METHODS: Objective of this survey study was to investigate the role differentiation, role transitions and satisfaction of professional care providers (i.e. pulmonologists, residents, hospital respiratory nurses, generic and specialised community nurses and general practitioners) from 3 hospitals and 2 home care organisations, involved in a community-based hospital-at-home scheme. A combined multiple-choice and open-end questionnaire was administered in study participants. RESULTS: Response rate was 10/17 in pulmonologists, 10/23 in residents, 9/12 in hospital respiratory nurses, 15/60 in generic community nurses, 6/10 in specialised community nurses and 25/47 in general practitioners. For between 66% and 100% of respondents the role in early discharge was clear and between 57% and 78% of respondents was satisfied with their role in early discharge. For nurses the role in early discharge was different compared to their role in usual care. 67% of generic community nurses felt they had sufficient knowledge and skills to monitor patients at home, compared to 100% of specialised community nurses. Specialised community nurses felt they should monitor patients. 60% of generic community nurses responded they should monitor patients at home. 78% of pulmonologists, 12% of general practitioners, 55% of hospital respiratory nurses and 48 of community nurses was satisfied with early discharge in general. For coordination of care 29% of community nurses had an unsatisfied response. For continuity of care this was 12% and 10% for hospital respiratory nurses and community nurses, respectively. CONCLUSION: A community-based early assisted discharge for COPD exacerbations is possible and well accepted from the perspective of health care providers' involved. Satisfaction with the different aspects is good and the transfer of patients in the community while supervised by generic community nurses is possible. Attention should be paid to coordination and continuity of care, especially information transfer between providers.

How Can a Bundled Payment Model Incentivize the Transition from Single-Disease Management to Person-Centred and Integrated Care for Chronic Diseases in the Netherlands?

To stimulate the integration of chronic care across disciplines, the Netherlands has implemented single-disease management programmes (SDMPs) in primary care since 2010; for example, for COPD, type 2 diabetes mellitus, and cardiovascular diseases. These disease-specific chronic care programmes are funded by bundled payments. For chronically ill patients with multimorbidity or with problems in other domains of health, this approach was shown to be less fit for purpose. As a result, we are currently witnessing several initiatives to broaden the scope of these programmes, aiming to provide truly person-centred integrated care (PC-IC). This raises the question if it is possible to design a payment model that would support this transition. We present an alternative payment model that combines a person-centred bundled payment with a shared savings model and pay-for-performance elements. Based on theoretical reasoning and results of previous evaluation studies, we expect the proposed payment model to stimulate integration of person-centred care between primary healthcare providers, secondary healthcare providers, and the social care domain. We also expect it to incentivise cost-conscious provider-behaviour, while safeguarding the quality of care, provided that adequate risk-mitigating actions, such as case-mix adjustment and cost-capping, are taken.

Effectiveness and cost-effectiveness of early assisted discharge for chronic obstructive pulmonary disease exacerbations: the design of a randomised controlled trial.

BACKGROUND: Exacerbations of chronic obstructive pulmonary disease (COPD) are the main cause for hospitalisation. These hospitalisations result in a high pressure on hospital beds and high health care costs. Because of the increasing prevalence of COPD this will only become worse. Hospital at home is one of the alternatives that has been proved to be a safe alternative for hospitalisation in COPD. Most schemes are early assisted discharge schemes with specialised respiratory nurses providing care at home. Whether this type of service is cost-effective depends on the setting in which it is delivered and the way in which it is organised. METHODS/DESIGN: GO AHEAD (Assessment Of Going Home under Early Assisted Discharge) is a 3-months, randomised controlled, multi-centre clinical trial. Patients admitted to hospital for a COPD exacerbation are either discharged on the fourth day of admission and further treated at home, or receive usual inpatient hospital care. Home treatment is supervised by general nurses. Primary outcome is the effectiveness and cost effectiveness of an early assisted discharge intervention in comparison with usual inpatient hospital care for patients hospitalised with a COPD exacerbation. Secondary outcomes include effects on quality of life, primary informal caregiver burden and patient and primary caregiver satisfaction. Additionally, a discrete choice experiment is performed to provide insight in patient and informal caregiver preferences for different treatment characteristics. Measurements are performed on the first day of admission and 3 days, 7 days, 1 month and 3 months thereafter. Ethical approval has been obtained and the study has been registered. DISCUSSION: This article describes the study protocol of the GO AHEAD study. Early assisted discharge could be an effective and cost-effective method to reduce length of hospital stay in the Netherlands which is beneficial for patients and society. If effectiveness and cost-effectiveness can be proven, implementation in the Dutch health care system should be considered. TRIAL REGISTRATION: Netherlands Trial Register NTR1129.

The risk of overestimating cost savings from hospital-at-home schemes: A literature review.

BACKGROUND: The concept of hospital-at-home means that home treatment is provided to patients who would otherwise have been treated in the hospital. This may lead to lower costs, but estimates of savings may be overstated if inpatient hospital costs are priced incorrectly. OBJECTIVE: The objective of this study was to evaluate the quality of cost analyses of hospital-at-home studies for acute conditions published from 1996 through 2019 and to present an overview of evidence. DESIGN: Literature review DATA SOURCES: The PubMed and NHS EED databases were searched. REVIEW METHODS: The overall quality of studies was evaluated based on Quality of Health Economic Studies (QHES) score, design, sample size, alignment of cost calculation with study perspective, time horizon, use of tariffs or real resource use and clarity of calculations. Furthermore, we systematically assessed whether cost savings were likely to be overestimated, based on criteria about the costing of inpatient hospital days, informal care costs and bias. RESULTS: We identified 48 studies. The average QHES score was 60 out of a maximum of 100 points. Almost all studies violated one or more criteria for the risk of overestimation of cost savings. The most frequent problems were the use of average unit prices per inpatient day (not taking into account the decreasing intensity of care) and biased designs. Most studies found cost differences in favour of hospital-at-home; the range varied from savings of €8773 to a cost increase of €2316 per patient. CONCLUSION: Overall quality of studies was not good, with some exceptions. Many cost savings were probably overestimated.

Summarizing Patient Preferences for the Competitive Landscape of Multiple Sclerosis Treatment Options.

Objective. Quantitatively summarize patient preferences for European licensed relapsing-remitting multiple sclerosis (RRMS) disease-modifying treatment (DMT) options. Methods. To identify and summarize the most important RRMS DMT characteristics, a literature review, exploratory physician interviews, patient focus groups, and confirmatory physician interviews were conducted in Germany, the United Kingdom, and the Netherlands. A discrete choice experiment (DCE) was developed and executed to measure patient preferences for the most important DMT characteristics. The resulting DCE data (n=799 and n=363 respondents in the United Kingdom and Germany, respectively) were analyzed using Bayesian mixed logit models. The estimated individual-level patient preferences were subsequently summarized using 3 additional analyses: the quality of the choice data was assessed using individual-level R2 estimates, individual-level preferences for the available DMTs were aggregated into DMT-specific preference shares, and a principal component analysis was performed to explain the patients' choice process. Results. DMT usage differed between RRMS patients in Germany and the United Kingdom but aggregate patient preferences were similar. Across countries, 42% of all patients preferred oral medications, 38% infusions, 16% injections, and 4% no DMT. The most often preferred DMT was natalizumab (26%) and oral DMT cladribine tablets (22%). The least often preferred were mitoxantrone and the beta-interferon injections (1%-3%). Patient preferences were strongly correlated with patients' MS disease duration and DMT experience, and differences in patient preferences could be summarized using 8 principle components that together explain 99% of the variation in patients' DMT preferences. Conclusion. This study summarizes patient preferences for the included DMTs, facilitates shared decision making along the dimensions that are relevant to RRMS patients, and introduces methods in the medical DCE literature that are ideally suited to summarize the impact of DMT introductions in preexisting treatment landscapes.

Protocolled practice nurse-led care for children with asthma in primary care: protocol for a cluster randomised trial.

INTRODUCTION: In children with asthma, daily symptoms and exacerbations have a significant impact on the quality of life of both children and parents. More effective use of asthma medication and, consequently, better asthma control is advocated, since both overtreatment and undertreatment are reported in primary care. Trials in adults suggest that asthma control is better when patients receive a regular medical review. Therefore, protocolled care by the general practitioner may also lead to better asthma control in children. However, such protocolled care by the general practitioner may be time consuming and less feasible. Therefore, this study aims to determine whether protocolled practice nurse-led asthma care for children in primary care provides more effective asthma control than usual care. METHODS AND ANALYSIS: The study will be a cluster-randomised open-label trial with an 18-month follow-up. Practice nurses will be the units of randomisation and children with asthma the units of analysis. It is planned to include 180 children aged 6-12 years. Primary outcome will be average asthma control during the 18-month follow-up measured by the Childhood Asthma Control Test (C-ACT). Secondary outcomes include C-ACT scores at t=3, t=6, t=12 and t=18 months; the frequency and severity of exacerbations; cost-effectiveness; quality of life; satisfaction with delivered care; forced expiratory volume in 1 s and forced expiratory flow at 75% and the association of high symptoms scores at baseline and baseline characteristics. Besides, we will conduct identical measurements in a non-randomised sample of children. ETHICS AND DISSEMINATION: This will be the first trial to evaluate the effectiveness of protocolled practice nurse-led care for children with asthma in primary care. The results may lead to improvements in asthma care for children and can be directly implemented in revisions of asthma guidelines.The study protocol was approved by the Medical Research Ethics Committee of the Erasmus Medical Centre in Rotterdam. TRIAL REGISTRATION: NTR6847.

Exploring the Impact of Adding a Respiratory Dimension to the EQ-5D-5L.

Objectives. To evaluate the impact of adding a respiratory dimension (a bolt-on dimension) to the EQ-5D-5L health state valuations. Methods. Based on extensive regression and principal component analyses, 2 respiratory bolt-on candidates were formulated: R1, limitations in physical activities due to shortness of breath, and R2, breathing problems. Valuation interviews for the selected bolt-ons were performed with a representative sample from the Dutch general public using the standardized interview protocol and software of the EuroQol group. Hybrid models based on the combined time-tradeoff (TTO) and discrete choice experiment (DCE) data were estimated to assess whether the 5 levels of the respiratory bolt-on led to significant changes in utility values. Results. For each bolt-on candidate, slightly more than 200 valuation interviews were conducted. Mean TTO values and DCE choice probabilities for health states with a level 4 or 5 for the respiratory dimension were significantly lower compared with the same health states in the Dutch EQ-5D-5L valuation study without the respiratory dimension. Results of hybrid models showed that for the bolt-on "limitations in physical activities," the utility decrements were significant for level 3 (-0.055), level 4 (-0.087), and level 5 (-0.135). For "breathing problems," the utility decrements for the same levels were greater (-0.086, -0.219, and -0.327, respectively). Conclusions. The addition of each of the 2 respiratory bolt-ons to the EQ-5D-5L had a significant effect on the valuation of health states with severe levels for the bolt-on. The bolt-on dimension "breathing problems" showed the greatest utility decrements and therefore seems the most appropriate respiratory bolt-on dimension.

The Fold-in, Fold-out Design for DCE Choice Tasks: Application to Burden of Disease.

Background In discrete-choice experiments (DCEs), choice alternatives are described by attributes. The importance of each attribute can be quantified by analyzing respondents' choices. Estimates are valid only if alternatives are defined comprehensively, but choice tasks can become too difficult for respondents if too many attributes are included. Several solutions for this dilemma have been proposed, but these have practical or theoretical drawbacks and cannot be applied in all settings. The objective of the current article is to demonstrate an alternative solution, the fold-in, fold-out approach (FiFo). We use a motivating example, the ABC Index for burden of disease in chronic obstructive pulmonary disease (COPD). Methods Under FiFo, all attributes are part of all choice sets, but they are grouped into domains. These are either folded in (all attributes have the same level) or folded out (levels may differ). FiFo was applied to the valuation of the ABC Index, which included 15 attributes. The data were analyzed in Bayesian mixed logit regression, with additional parameters to account for increased complexity in folded-out questionnaires and potential differences in weight due to the folding status of domains. As a comparison, a model without the additional parameters was estimated. Results Folding out domains led to increased choice complexity for respondents. It also gave domains more weight than when it was folded in. The more complex regression model had a better fit to the data than the simpler model. Not accounting for choice complexity in the models resulted in a substantially different ABC Index. Conclusion Using a combination of folded-in and folded-out attributes is a feasible approach for conducting DCEs with many attributes.

Cost-Utility Analysis of High Molecular Weight Hyaluronic Acid for Knee Osteoarthritis in Everyday Clinical Care in Patients at a Working Age: An Economic Evaluation of a Randomized Clinical Trial.

OBJECTIVE: Knee osteoarthritis (OA) is associated with high medical costs and especially with high productivity costs, in particular in patients in their working years. High molecular weight (HMW) hyaluronic acid (HA) is an alternative treatment for nonsteroidal antiinflammatory drugs, which are known for their serious side-effects. The cost-utility of intraarticular HMW-HA treatment in these patients is unknown, however, and was assessed in this study. METHODS: Secondary care patients ages 18-65 years with knee OA were randomized to usual care plus HMW-HA (intervention group) or to usual care only (control group). A cost-utility analysis over 52 weeks from the societal and health care perspective was performed. Uncertainty for costs, effects, and cost-utility ratio was analyzed by nonparametric bootstrapping. Baseline imbalance adjustment was done by inverse probability of treatment weighting. RESULTS: In total, 156 subjects were included (intervention group n = 77, control group n = 79). The total of productivity and medical costs was €475 higher in the intervention group at €7,754 (95% confidence interval [95% CI] 5,426, 10,436) versus €7,270 (95% CI 5,453, 9,262). The amount of quality-adjusted life years (QALYs) gained during followup was also higher in the intervention group (0.779 versus 0.727). This variation resulted in an incremental cost-effectiveness ratio of €9,100/QALY from a societal perspective and €8,700/QALY from a health care perspective. When the maximum willingness to pay for conditions similar to knee OA is considered, the probability on cost-effectiveness is 64% and 86%, respectively. CONCLUSION: Intraarticular HMW-HA added to usual care for knee OA is probably cost-effective in the treatment of knee OA.

Multisystemic Therapy and Functional Family Therapy Compared on their Effectiveness Using the Propensity Score Method.

Multisystemic Therapy (MST) and Functional Family Therapy (FFT) have overlapping target populations and treatment goals. In this study, these interventions were compared on their effectiveness using a quasi-experimental design. Between October, 2009 and June, 2014, outcome data were collected from 697 adolescents (mean age 15.3 (SD 1.48), 61.9% male) assigned to either MST or FFT (422 MST; 275 FFT). Data were gathered during Routine Outcome Monitoring. The primary outcome was externalizing problem behavior (Child Behavior Checklist and Youth Self Report). Secondary outcomes were the proportion of adolescents living at home, engaged in school or work, and who lacked police contact during treatment. Because of the non-random assignment, a propensity score method was used to control for observed pre-treatment differences. Because the risk-need-responsivity (RNR) model guided treatment assignment, effectiveness was also estimated in youth with and without a court order as an indicator of their risk level. Looking at the whole sample, no difference in effect was found with regard to externalizing problems. For adolescents without a court order, effects on externalizing problems were larger after MST. Because many more adolescents with a court order were assigned to MST compared to FFT, the propensity score method could not balance the treatment groups in this subsample. In conclusion, few differences between MST and FFT were found. In line with the RNR model, higher risk adolescents were assigned to the more intensive treatment, namely MST. In the group with lower risk adolescents, this more intensive treatment was more effective in reducing externalizing problems.

How much does a heart valve implantation cost and what are the health care costs afterwards?

Objective: In the era of limited healthcare budgets, healthcare costs of heart valve implantations need to be considered to inform cost-effectiveness analyses. We aimed to provide age group-specific costs estimates of heart valve implantations, related complications and other healthcare utilisation following the intervention. Methods: We performed retrospective analyses of healthcare costs of patients who had undergone heart valve implantations in 2010-2013 and controls using claims data from Dutch health insurers. Heart valve implantations included surgical valve replacement and transcatheter valve implantation in all heart valve positions. Patients were divided in four age groups. Control groups were created by taking random samples of the Dutch population stratified by age, gender, socioeconomic status and comorbidities. We applied non-parametric bootstrapping to address uncertainty of the cost estimates. The association of patient and intervention characteristics with costs was determined by (multilevel) generalised linear models. Results: The baseline characteristics of 18 903 patients and 188 925 controls were comparable. The annual healthcare costs were substantially higher for surgical heart valve replacement patients than for controls, especially in the year of heart valve implantation. Factors associated with increased annual healthcare costs for patients were older age, female gender, comorbidities, low socioeconomic status and complications. Conclusions: We provided a comprehensive overview of age group-specific incidence of heart valve implantations, subsequent survival and complications as well as associated healthcare costs of all patients in the Netherlands. Our results provide real-world costs estimates that can be used as a benchmark for costs of future innovative heart valve implantations.

Systematic Review and Quality Appraisal of Cost-Effectiveness Analyses of Pharmacologic Maintenance Treatment for Chronic Obstructive Pulmonary Disease: Methodological Considerations and Recommendations.

BACKGROUND: Worldwide, chronic obstructive pulmonary disease (COPD) is a highly prevalent chronic lung disease with considerable clinical and socioeconomic impact. Pharmacologic maintenance drugs (such as bronchodilators and inhaled corticosteroids) play an important role in the treatment of COPD. The cost effectiveness of these treatments has been frequently assessed, but studies to date have largely neglected the impact of treatment sequence and the exact stage of disease in which the drugs are used in real life. OBJECTIVE: We aimed to systematically review recently published articles that reported the cost effectiveness of COPD maintenance treatments, with a focus on key findings, quality and methodological issues. METHODS: We performed a systematic literature search in Embase, PubMed, the UK NHS Economic Evaluation Database (NHS-EED) and EURONHEED (European Network of Health Economics Evaluation Databases) and included all relevant articles published between 2011 and 2015 in either Dutch, English or German. Main study characteristics, methods and outcomes were extracted and critically assessed. The Quality of Health Economic Studies (QHES) instrument was used as basis for quality assessment, but additional items were also addressed. RESULTS: The search identified 18 recent pharmacoeconomic analyses of COPD maintenance treatments. Papers reported the cost effectiveness of long-acting muscarinic antagonist (LAMA) monotherapy (n = 6), phosphodiesterase (PDE)-4 inhibitors (n = 4), long-acting beta agonist/inhaled corticosteroid (LABA/ICS) combinations (n = 4), LABA monotherapy (n = 2) and LABA/LAMA combinations (n = 2). All but two studies were funded by the manufacturer, and all studies indicated favourable cost effectiveness; however, the number of quality-adjusted life-years (QALYs) gained was small. Less than half of the studies reported a COPD-specific outcome in addition to a generic outcome (mostly QALYs). Exacerbation and mortality rates were found to be the main drivers of cost effectiveness. According to the QHES, the quality of the studies was generally sufficient, but additional assessment revealed that most studies poorly represented the cost effectiveness of real-life medication use. CONCLUSIONS: The majority of studies showed that pharmacologic COPD maintenance treatment is cost effective, but most studies poorly reflected real-life drug use. Consistent and COPD-specific methodology is recommended.