[Effectiveness of the Assessment of Burden of COPD tool: a cluster-randomised controlled trial]. / Effectiviteit van de 'Ziektelastmeter COPD'.

OBJECTIVE: Assessment of the effectiveness of the Assessment of Burden of COPD (ABC) tool on disease-specific quality of life in patients with Chronic Obstructive Pulmonary Disease (COPD). DESIGN: Cluster-randomised controlled trial. METHOD: This concerned a trial in 39 Dutch primary care practices and 17 hospitals, involving 357 patients with COPD (postbronchodilator FEV1/FVC ratio < 0.7) aged ≥ 40 years. Healthcare providers were randomized to an intervention or control group. Patients in the intervention group were treated with the ABC tool. This innovative tool consists of a short validated questionnaire and a number of objective parameters, which collectively give a visual overview of the combined integral health; the tool subsequently produces an individualized treatment plan by means of a treatment algorithm. Patients in the control group received usual care. The primary outcome measure was the proportion of patients with a clinically relevant improvement in disease-specific quality of life measured, as measured by means of the St. George's Respiratory Questionnaire (SGRQ) score, between baseline and 18 months follow-up. Secondary outcomes included the SGRQ total score and the Patient Assessment of Chronic Illness Care (PACIC) score. RESULTS: At 18-month follow-up, a significant and clinically relevant improvement in the SGRQ score was seen in 34% of the patients (N=49) in the intervention group, and in the control group this figure was 22% (N=33). This difference between the two groups was significant (OR 1.85, 95% CI 1.08 to 3.16). Patients in the intervention group experienced a higher quality of care than patients in the control group (0.32 points difference in PACIC, 95% CI 0.14 to 0.50). CONCLUSION: Use of the ABC tool increases the disease-specific quality of life and the quality of care for COPD patients; it may therefore offer a valuable contribution to improvements in the daily care of COPD. Replication of this study in other (non-Dutch) health-care settings is recommended.

Cost-Effectiveness of Including a Nurse Specialist in the Treatment of Urinary Incontinence in Primary Care in the Netherlands.

OBJECTIVE: Incontinence is an important health problem. Effectively treating incontinence could lead to important health gains in patients and caregivers. Management of incontinence is currently suboptimal, especially in elderly patients. To optimise the provision of incontinence care a global optimum continence service specification (OCSS) was developed. The current study evaluates the costs and effects of implementing this OCSS for community-dwelling patients older than 65 years with four or more chronic diseases in the Netherlands. METHOD: A decision analytic model was developed comparing the current care pathway for urinary incontinence in the Netherlands with the pathway as described in the OCSS. The new care strategy was operationalised as the appointment of a continence nurse specialist (NS) located with the general practitioner (GP). This was assumed to increase case detection and to include initial assessment and treatment by the NS. The analysis used a societal perspective, including medical costs, containment products (out-of-pocket and paid by insurer), home care, informal care, and implementation costs. RESULTS: With the new care strategy a QALY gain of 0.005 per patient is achieved while saving €402 per patient over a 3 year period from a societal perspective. In interpreting these findings it is important to realise that many patients are undetected, even in the new care situation (36%), or receive care for containment only. In both of these groups no health gains were achieved. CONCLUSION: Implementing the OCSS in the Netherlands by locating a NS in the GP practice is likely to reduce incontinence, improve quality of life, and reduce costs. Furthermore, the study also highlighted that various areas of the continence care process lack data, which would be valuable to collect through the introduction of the NS in a study setting.

Costs and effects of new professional roles: Evidence from a literature review.

One way in which governments are seeking to improve the efficiency of the health care sector is by redesigning health services to contain labour costs. The aim of this study was to investigate the impact of new professional roles on a wide range of health service outcomes and costs. A systematic literature review was performed by searching in different databases for evaluation papers of new professional roles (published 1985-2013). The PRISMA checklist was used to conduct and report the systematic literature review and the EPHPP-Quality Assessment Tool to assess the quality of the studies. Forty-one studies of specialist nurses (SNs) and advanced nurse practitioners (ANPs) were selected for data extraction and analysis. The 25 SN studies evaluated most often quality of life (10 studies), clinical outcomes (8), and costs (8). Significant advantages were seen most frequently regarding health care utilization (in 3 of 3 studies), patient information (5 of 6), and patient satisfaction (4 of 6). The 16 ANP studies evaluated most often patient satisfaction (8), clinical outcomes (5), and costs (5). Significant advantages were seen most frequently regarding clinical outcomes (5 of 5), patient information (3 of 4), and patient satisfaction (5 of 8). Promoting new professional roles may help improve health care delivery and possibly contain costs. Exploring the optimal skill-mix deserves further attention from health care professionals, researchers and policy makers.

Comparison of EQ-5D and SF-6D utilities in Pompe disease.

PURPOSE: Comparative studies between Euroqol-5D (EQ-5D) and ShortForm 6D (SF-6D) utilities have been performed for a number of diseases, but not yet for orphan diseases. Pompe disease is an orphan disease with a prevalence of <5/10,000, characterized by impaired ambulatory and pulmonary functioning. We compared the psychometric properties of EQ-5D and SF-6D in patients with this disease and assessed their convergent validity, discriminative ability and sensitivity to change. METHODS: EQ-5D utilities and SF-6D utilities were computed using the UK value set. Dimensions and utilities of the two instruments were compared by correlation coefficients and descriptive statistics. We assessed whether EQ-5D and SF-6D were able to discriminate between different levels of severity and examined sensitivity to change for patients with multiple observations. RESULTS: Correlations between theoretically related dimensions of the EQ-5D and SF-6D were highly significant and were moderate to strong (range rho = 0.409-0.564). Utility values derived from the two instruments were similar (mean EQ-5D = 0.670; mean SF-6D = 0.699) and correlated strongly (rho = 0.591). Discriminative properties were somewhat better for EQ-5D; mean changes and effect sizes were better for SF-6D. CONCLUSIONS: Overall, we conclude that both instruments appear to be equally appropriate with respect to assessing utilities in Pompe disease, but neither of them performed excellently. The descriptive system of the SF-6D describes health states for Pompe disease more accurately. EQ-5D showed better discriminative properties. The SF-6D performed better with respect to sensitivity to change.

Which long-acting bronchodilator is most cost-effective for the treatment of COPD?

BACKGROUND: The aim of this study was to estimate the cost-effectiveness of tiotropium versus salmeterol to inform decision making within the Dutch healthcare setting. METHODS: A previously published, validated COPD progression model was updated with new exacerbation data and adapted to the Dutch setting by including Dutch estimates of healthcare use for COPD maintenance treatment and Dutch unit costs. Exacerbation data from the POET-COPD trial were combined with evidence from earlier tiotropium studies using Bayesian meta-analysis. The model-based analysis was performed using a one- and five-year time horizon. Main health outcomes were the number of exacerbations and quality-adjusted life years (QALYs). RESULTS: One-year costs per patient from the healthcare perspective were v1370 for tiotropium and v1359 for salmeterol; a difference of v11 (95% uncertainty interval (UI): -198-212). The annual number of exacerbations was 0.068 (-0.005-0.140) lower in the tiotropium group. The number of QALYs in the tiotropium group was 0.011 (-0.019-0.049) higher, resulting in an incremental cost-effectiveness ratio (ICER) of v1015 per QALY. After five years, the difference in exacerbations, QALYs and costs between the tiotropium and salmeterol group were -0.435 (-0.915-0.107), 0.079 (-0.272-0.520) and v-277 (-1586-1074), respectively, indicating that tiotropium was more effective and less costly. Using a societal perspective, tiotropium dominated salmeterol both after one and five years. CONCLUSION: Tiotropium reduced exacerbations and exacerbation-related costs. After one year the cost per QALY of tiotropium compared with salmeterol was very low, while after five years tiotropium was found to dominate salmeterol.

Bottom-up implementation of disease-management programmes: results of a multisite comparison.

OBJECTIVES: To evaluate the implementation of three regional disease-management programmes on chronic obstructive pulmonary disease (COPD) based on bottlenecks experienced in professional practice. METHODS: The authors performed a multisite comparison of three Dutch regional disease-management programmes combining patient-related, professional-directed and organisational interventions. Process (Assessing Chronic Illness Care survey) and outcome (disease specific quality of life (clinical COPD questionnaire (CCQ); chronic respiratory questionnaire (CRQ)), Medical Research Council dyspnoea and patients' experiences) data were collected for 370 COPD patients and their care providers. RESULTS: Bottlenecks in region A were mostly related to patient involvement, in region B to organisational issues and in region C to both. Selected interventions related to identified bottlenecks were implemented in all programmes, except for patient-related interventions in programme A. Within programmes, significant improvements were found on dyspnoea and patients' experiences with practice nurses. Outcomes on quality of life differed between programmes: programme A did not show any significant improvements; programme B did show any significant improvements on CCQ total (p<0.001), functional (p=0.011) and symptom (p<0.001), CRQ fatigue (p<0.001) and emotional scales (p<0.001); in programme C, CCQ symptom (p<0.001) improved significantly, whereas CCQ mental score (p<0.001) deteriorated significantly. Regression analyses showed that programmes with better implementation of selected interventions resulted in relatively larger improvements in quality of life (CCQ). CONCLUSIONS: Bottom-up implementation of COPD disease-management programmes is a feasible approach, which in multiple settings leads to significant improvements in outcomes of care. Programmes with a better fit between implemented interventions and bottlenecks showed more positive changes in outcomes.

Case fatality of COPD exacerbations: a meta-analysis and statistical modelling approach.

The aim of our study was to estimate the case fatality of a severe exacerbation from long-term survival data presented in the literature. A literature search identified studies reporting ≥1.5 yr survival after a severe chronic obstructive pulmonary disease (COPD) exacerbation resulting in hospitalisation. The survival curve of each study was divided into a critical and a stable period. Mortality during the stable period was then estimated by extrapolating the survival curve during the stable period back to the time of exacerbation onset. Case fatality was defined as the excess mortality that results from an exacerbation and was calculated as 1 minus the (backwardly) extrapolated survival during the stable period at the time of exacerbation onset. The 95% confidence intervals (CI) of the estimated case fatalities were obtained by bootstrapping. A random effect model was used to combine all estimates into a weighted average with 95% CI. The meta-analysis based on six studies that fulfilled the inclusion criteria resulted in a weighted average case-fatality rate of 15.6% (95% CI 10.9-20.3), ranging from 11.4% to 19.0% for the individual studies. A severe COPD exacerbation requiring hospitalisation not only results in higher mortality risks during hospitalisation, but also in the time-period after discharge and contributes substantially to total COPD mortality.

Short- and long-term efficacy of a community-based COPD management programme in less advanced COPD: a randomised controlled trial.

BACKGROUND: The effectiveness of pulmonary rehabilitation in advanced COPD is well established, but few data are available in less advanced disease. METHODS: In a 2 year randomised controlled trial, 199 patients with an average moderate airflow obstruction but impaired exercise capacity (mean (SD) forced expiratory volume in 1 s (FEV(1)) 60 (16)%, peak work load (Wmax) <70%) were randomised to the INTERdisciplinary COMmunity-based COPD management programme (INTERCOM) or usual care. Intervention consisted of 4 months multidisciplinary rehabilitation followed by a 20-month maintenance phase. Outcomes (4, 12, 24 months): health-related quality of life (St George's Respiratory Questionnaire (SGRQ)), exacerbation frequency, MRC dyspnoea score, cycle endurance time (CET), 6-minute walking distance (6MWD), skeletal muscle strength and patients' and caregivers' perceived effectiveness. RESULTS: Between-group comparison after 4 months revealed the following mean (SE) significant differences in favour of INTERCOM: SGRQ total score 4.06 (1.39), p = 0.004; activity and impact subscores, p<0.01; MRC score 0.33 (0.13), p = 0.01; Wmax 6.0 (2.3) Watt, p = 0.02; CET 221 (104) s, p = 0.04; 6MWD 13 (6) m, p = 0.02; hand grip force 4.3 (1.5) lb, p<0.01; and fat-free mass index 0.34 (0.13) kg/m(2), p = 0.01. Between-group differences over 2 years were as follows: SGRQ 2.60 (1.3), p = 0.04; MRC score 0.21 (0.10), p = 0.048; CET 253 (104) s, p = 0.0156; 6MWD 18 (8) m, p = 0.0155. Exacerbation frequency was not different (RR 1.29 (95% CI 0.89 to 1.87)). Patients' and caregivers' perceived effectiveness significantly favoured the INTERCOM programme (p<0.01). CONCLUSIONS: This study shows that a multidisciplinary community-based disease management programme is also effective in patients with COPD with exercise impairment but less advanced airflow obstruction.

Is INTERdisciplinary COMmunity-based COPD management (INTERCOM) cost-effective?

The study aimed to estimate the cost-effectiveness of interdisciplinary community-based chronic obstructive pulmonary disease (COPD) management in patients with COPD. We conducted a cost-effectiveness analysis alongside a 2-yr randomised controlled trial, in which 199 patients with less advanced airflow obstruction and impaired exercise capacity were assigned to the INTERCOM programme or usual care. The INTERCOM programme consisted of exercise training, education, nutritional therapy and smoking cessation counselling offered by community-based physiotherapists and dieticians and hospital-based respiratory nurses. All-cause resource use during 2 yrs was obtained by self-report and from hospital and pharmacy records. Health outcomes were the St George's Respiratory Questionnaire (SGRQ), exacerbations and quality-adjusted life years (QALYs). The INTERCOM group had 30% (95% CI 3-56%) more patients with a clinically relevant improvement in SGRQ total score, 0.08 (95% CI -0.01-0.18) more QALYs per patient, but a higher mean number of exacerbations, 0.84 (95% CI -0.07-1.78). Mean total 2-yr costs were euro2,751 (95% CI -euro632-euro6,372) higher for INTERCOM than for usual care, which resulted in an incremental cost-effectiveness ratio of euro9,078 per additional patient with a relevant improvement in SGRQ or euro32,425 per QALY. INTERCOM significantly improved disease-specific quality of life, but did not affect exacerbation rate. The cost per QALY ratio was moderate, but within the range of that generally considered to be acceptable.

Outcomes for COPD pharmacological trials: from lung function to biomarkers.

The American Thoracic Society/European Respiratory Society jointly created a Task Force on "Outcomes for COPD pharmacological trials: from lung function to biomarkers" to inform the chronic obstructive pulmonary disease research community about the possible use and limitations of current outcomes and markers when evaluating the impact of a pharmacological therapy. Based on their review of the published literature, the following document has been prepared with individual sections that address specific outcomes and markers, and a final section that summarises their recommendations.