Peter McMeekin

Health-related utility values of patients with primary Sjögren's syndrome and its predictors

Objectives EuroQoL-5 dimension (EQ-5D) is a standardised preference-based tool for measurement of health-related quality of life and EQ-5D utility values can be converted to quality-adjusted life years (QALYs) to aid cost-utility analysis. This study aimed to evaluate the EQ-5D utility values of 639 patients with primary Sjögrens syndrome (PSS) in the UK. Methods Prospective data collected using a standardised pro forma were compared with UK normative data. Relationships between utility values and the clinical and laboratory features of PSS were explored. Results The proportion of patients with PSS reporting any problem in mobility, self-care, usual activities, pain/discomfort and anxiety/depression were 42.2%, 16.7%, 56.6%, 80.6% and 49.4%, respectively, compared with 5.4%, 1.6%, 7.9%, 30.2% and 15.7% for the UK general population. The median EQ-5D utility value was 0.691 (IQR 0.587–0.796, range −0.239 to 1.000) with a bimodal distribution. Bivariate correlation analysis revealed significant correlations between EQ-5D utility values and many clinical features of PSS, but most strongly with pain, depression and fatigue (R values>0.5). After adjusting for age and sex differences, multiple regression analysis identified pain and depression as the two most important predictors of EQ-5D utility values, accounting for 48% of the variability. Anxiety, fatigue and body mass index were other statistically significant predictors, but they accounted for <5% in variability. Conclusions This is the first report on the EQ-5D utility values of patients with PSS. These patients have significantly impaired utility values compared with the UK general population. EQ-5D utility values are significantly related to pain and depression scores in PSS.

A validated clinical tool for the prediction of varices in PBC: The Newcastle Varices in PBC Score

BACKGROUND & AIMS Gastro-oesophageal varices (GOV) can occur in early stage primary biliary cirrhosis (PBC), making it difficult to identify the appropriate time to begin screening with oesophageo-gastro-duodenoscopy (OGD). Our aim was to develop and validate a clinical tool to predict the probability of finding GOV in PBC patients. METHODS A cross-sectional retrospective study analysing clinical data of 330 PBC patients who underwent an OGD at the Freeman Hospital, Newcastle was used to create a predictive tool, the Newcastle Varices in PBC (NVP) Score, that was externally validated in PBC patients from Cambridge (UK) and Toronto (Canada). RESULTS 48% of the Newcastle, 31% of the Cambridge, and 22% of the Toronto cohorts of PBC patients had GOV. Twenty-five percent (95% CI 18-32%) of the Newcastle cohort had GOV diagnosed at an index variceal bleed. Of the others, 37% (95% CI 28-46%) bled after a median of 1.5 years (IQR 3.75). Transplant-free survival was significantly better in those without GOV than in those with GOV (p<0.001), but similar in patients with GOV that bled and those that did not (p=0.1). The NVP score (%Probability)=1/[1+exp^-(9.186+0.001*alkaline phosphatase in IU-0.178*albumin in g/L-0.015*platelet × 10(9)) was validated in 2 external cohorts and was highly discriminant (AUROC 0.86). Cost consequences analyses revealed the NVP score to be as accurate as, but more economical than using either OGD directly or other risk scores for screening PBC patients. CONCLUSIONS The NVP score is an inexpensive, non-invasive, externally validated tool that accurately predicts GOV in PBC.

Can Aging in Place Be Cost Effective? A Systematic Review

Purpose of the Study To systematically review cost, cost-minimization and cost-effectiveness studies for assisted living technologies (ALTs) that specifically enable older people to ‘age in place’ and highlight what further research is needed to inform decisions regarding aging in place. Design People aged 65+ and their live-in carers (where applicable), using an ALT to age in place at home opposed to a community-dwelling arrangement. Methods Studies were identified using a predefined search strategy on two key economic and cost evaluation databases NHS EED, HEED. Studies were assessed using methods recommended by the Campbell and Cochrane Economic Methods Group and presented in a narrative synthesis style. Results Eight eligible studies were identified from North America spread over a diverse geographical range. The majority of studies reported the ALT intervention group as having lower resource use costs than the control group; though the low methodological quality and heterogeneity of the individual costs and outcomes reported across studies must be considered. Implications The studies suggest that in some cases ALTs may reduce costs, though little data were identified and what there were was of poor quality. Methods to capture quality of life gains were not used, therefore potential effects on health and wellbeing may be missed. Further research is required using newer developments such as the capabilities approach. High quality studies assessing the cost-effectiveness of ALTs for ageing in place are required before robust conclusion on their use can be drawn.

Quality compared to quantity of life in laryngeal cancer: A time trade‐off study

The purpose of this study was to use time trade‐off to assess the factors influencing patients’ decisions in advanced laryngeal cancer. Time trade‐off is a well‐established method of assessing how individuals value a particular health state.

The STRIDE (Strategies to Increase confidence, InDependence and Energy) study: cognitive behavioural therapy-based intervention to reduce fear of falling in older fallers living in the community - study protocol for a randomised controlled trial.

BackgroundAround 30% to 62% of older individuals fall each year, with adverse consequences of falls being by no means limited to physical injury and escalating levels of dependence. Many older individuals suffer from a variety of adverse psychosocial difficulties related to falling including fear, anxiety, loss of confidence and subsequent increasing activity avoidance, social isolation and frailty. Such ‘fear of falling’ is common and disabling, but definitive studies examining the effective management of the syndrome are lacking. Cognitive behavioural therapy has been trialed with some success in a group setting, but there is no adequately powered randomised controlled study of an individually based cognitive behavioural therapy intervention, and none using non-mental health professionals to deliver the intervention.Methods/DesignWe are conducting a two-phase study examining the role of individual cognitive behavioural therapy delivered by healthcare assistants in improving fear of falling in older adults. In Phase I, the intervention was developed and taught to healthcare assistants, while Phase II is the pragmatic randomised controlled study examining the efficacy of the intervention in improving fear of falling in community-dwelling elders attending falls services. A qualitative process evaluation study informed by Normalization Process Theory is being conducted throughout to examine the potential promoters and inhibitors of introducing such an intervention into routine clinical practice, while a health economic sub-study running alongside the trial is examining the costs and benefits of such an approach to the wider health economy.Trial registrationCurrent Controlled Trials ISRCTN78396615

Economic evaluations on centralisation of specialised healthcare services: a systematic review of methods

Objective To systematically review and appraise the quality of economic evaluations assessing centralisation of specialised healthcare services. Methods A systematic review to identify economic evaluations on centralisation of any specialised healthcare service. Full economic evaluations comparing costs and consequences of centralisation of any specialised healthcare service were eligible for inclusion. Methodological characteristics of included studies were appraised using checklists adapted from recommended guidelines. Results A total of 64 full-text articles met the inclusion criteria. Two studies were conducted in the UK. Most of the studies used volume of activity as a proxy measure of centralisation. The methods used to assess centralisation were heterogeneous. Studies differed in terms of study design used and aspect of centralisation they considered. There were major limitations in studies. Only 12 studies reported the study perspective. Charges which are not true representation of costs were used by 17 studies to assess cost outcomes. Only 10 reported the detailed breakdown of the cost components used in their analysis. Discounting was necessary in 14 studies but was reported only in 7 studies. Sensitivity analyses were included by less than one-third of the studies. The applicability of the identified studies to a setting other than the one they were conducted in is questionable, given variations in the organisation of services and healthcare costs. Centralisation as a concept has also been variably and narrowly defined as activity of specific services which may not reflect the wider aspects of centralisation. Conclusions Confounded and biased information coming from studies without standardised methods may mislead decision-makers towards making wrong decisions on centralisation. It is important to improve the methodology and reporting of economic evaluations in order to provide more robust and transferable evidence. Wider aspects of healthcare centralisation should be considered in the estimates of costs and health outcomes.

Development of a decision analytic model to support decision making and risk communication about thrombolytic treatment

BackgroundIndividualised prediction of outcomes can support clinical and shared decision making. This paper describes the building of such a model to predict outcomes with and without intravenous thrombolysis treatment following ischaemic stroke.MethodsA decision analytic model (DAM) was constructed to establish the likely balance of benefits and risks of treating acute ischaemic stroke with thrombolysis. Probability of independence, (modified Rankin score mRS ≤ 2), dependence (mRS 3 to 5) and death at three months post-stroke was based on a calibrated version of the Stroke-Thrombolytic Predictive Instrument using data from routinely treated stroke patients in the Safe Implementation of Treatments in Stroke (SITS-UK) registry. Predictions in untreated patients were validated using data from the Virtual International Stroke Trials Archive (VISTA). The probability of symptomatic intracerebral haemorrhage in treated patients was incorporated using a scoring model from Safe Implementation of Thrombolysis in Stroke-Monitoring Study (SITS-MOST) data.ResultsThe model predicts probabilities of haemorrhage, death, independence and dependence at 3-months, with and without thrombolysis, as a function of 13 patient characteristics. Calibration (and inclusion of additional predictors) of the Stroke-Thrombolytic Predictive Instrument (S-TPI) addressed issues of under and over prediction. Validation with VISTA data confirmed that assumptions about treatment effect were just. The C-statistics for independence and death in treated patients in the DAM were 0.793 and 0.771 respectively, and 0.776 for independence in untreated patients from VISTA.ConclusionsWe have produced a DAM that provides an estimation of the likely benefits and risks of thrombolysis for individual patients, which has subsequently been embedded in a computerised decision aid to support better decision-making and informed consent.

Do the EULAR Sjögren’s syndrome outcome measures correlate with health status in primary Sjögren’s syndrome?

OBJECTIVE This study sets out to investigate the relationship between health status [EuroQol five-dimensions questionnaire (EQ-5D)] in primary SS and three of the European League Against Rheumatism (EULAR) SS outcome measures-the disease activity index (ESSDAI), the patient reported index (ESSPRI) and the sicca score. In particular, the goal was to establish whether there is a relationship between the EULAR outcome measures and quality of life. METHODS Health status was evaluated using a standardized measure developed by the EuroQol Group-the EQ5D. This permits calculation of two measures of health status: time trade-off (TTO) values and the EQ-5D visual analogue scale (VAS) scores. We used Spearmans rank correlation analysis to investigate the strength of association between health status and three EULAR measures of physician- and patient-reported disease activity in 639 patients from the UK primary SS registry (UKPSSR) cohort. RESULTS This study demonstrates that the EULAR SS disease-specific outcome measures are significantly correlated with health outcome values (P < 0.001). Higher scores on the ESSDAI, EULAR sicca score and ESSPRI are associated with poorer health states-i.e. lower TTO values and lower VAS scores. While all three are significantly correlated with TTO values and EQ-5D VAS scores, the effect is strongest for the ESSPRI. CONCLUSION This study provides further evidence supporting the use of ESSDAI, EULAR sicca score and ESSPRI measures in the clinic. We also discuss the need for disease-specific measures of health status and their comparison with standardized health outcome measures.

An observational study of patient characteristics associated with the mode of admission to acute stroke services in North East, England

Objective Effective provision of urgent stroke care relies upon admission to hospital by emergency ambulance and may involve pre-hospital redirection. The proportion and characteristics of patients who do not arrive by emergency ambulance and their impact on service efficiency is unclear. To assist in the planning of regional stroke services we examined the volume, characteristics and prognosis of patients according to the mode of presentation to local services. Study design and setting A prospective regional database of consecutive acute stroke admissions was conducted in North East, England between 01/09/10-30/09/11. Case ascertainment and transport mode were checked against hospital coding and ambulance dispatch databases. Results Twelve acute stroke units contributed data for a mean of 10.7 months. 2792/3131 (89%) patients received a diagnosis of stroke within 24 hours of admission: 2002 arrivals by emergency ambulance; 538 by private transport or non-emergency ambulance; 252 unknown mode. Emergency ambulance patients were older (76 vs 69 years), more likely to be from institutional care (10% vs 1%) and experiencing total anterior circulation symptoms (27% vs 6%). Thrombolysis treatment was commoner following emergency admission (11% vs 4%). However patients attending without emergency ambulance had lower inpatient mortality (2% vs 18%), a lower rate of institutionalisation (1% vs 6%) and less need for daily carers (7% vs 16%). 149/155 (96%) of highly dependent patients were admitted by emergency ambulance, but none received thrombolysis. Conclusion Presentations of new stroke without emergency ambulance involvement were not unusual but were associated with a better outcome due to younger age, milder neurological impairment and lower levels of pre-stroke dependency. Most patients with a high level of pre-stroke dependency arrived by emergency ambulance but did not receive thrombolysis. It is important to be aware of easily identifiable demographic groups that differ in their potential to gain from different service configurations.

Estimating the number of UK stroke patients eligible for endovascular thrombectomy

Introduction Endovascular thrombectomy is a highly effective treatment for acute ischemic stroke due to large arterial occlusion. Routine provision will require major changes in service configuration and workforce. An important first step is to quantify the population of stroke patients that could benefit. We estimated the annual UK population suitable for endovascular thrombectomy using standard or advanced imaging for patient selection. Patients and methods Evidence from randomised control trials and national registries was combined to estimate UK stroke incidence and define a decision-tree describing the endovascular thrombectomy eligible population. Results Between 9620 and 10,920 UK stroke patients (approximately 10% of stroke admissions) would be eligible for endovascular thrombectomy annually. The majority (9140–9620) would present within 4 h of onset and be suitable for intravenous thrombolysis. Advanced imaging would exclude 500 patients presenting within 4 h, but identify an additional 1310 patients as eligible who present later. Discussion Information from randomised control trials and large registry data provided the evidence criterion for 9 of the 12 decision points. The best available evidence was used for two decision points with sensitivity analyses to determine how key branches of the tree affected estimates. Using the mid-point estimate for eligibility (9.6% of admissions) and assuming national endovascular thrombectomy coverage, 4280 patients would have reduced disability. Conclusion A model combining published trials and register data suggests approximately 10% of all stroke admissions in the UK are eligible for endovascular thrombectomy. The use of advanced imaging based on current published evidence did not have a major impact on overall numbers but could alter eligibility status for 16% of cases.