The relationship between EQ-5D, HAQ and pain in patients with rheumatoid arthritis: further validation and development of the limited dependent variable, mixture model approach

Objective: To provide robust estimates of EQ-5D as a function of the Health Assessment Questionnaire (HAQ) and pain in patients with rheumatoid arthritis. Method: Repeated observations of patients diagnosed with RA in a US observational cohort (n=100,398 observations) who provided data on HAQ, pain on a visual analogue scale and the EQ-5D questionnaire. We use a bespoke mixture modelling approach to appropriately reflect the characteristics of the EQ-5D instrument and compare this to results from linear regression. Results: The addition of pain alongside HAQ as an explanatory variable substantially improves explanatory power. The preferred model is a four component mixture. Unlike the linear regression it exhibits very good fit to the data, does not suffer from problems of bias or predict values outside the feasible range. Conclusions: It is appropriate to model the relationship between HAQ and EQ-5D but only if suitable statistical methods are applied. Linear models underestimate the QALY benefits, and therefore the cost effectiveness, of therapies. The bespoke mixture model approach outlined here overcomes this problem. The addition of pain as an explanatory variable greatly improves the estimates

Access to and use of clinical services and disease-modifying therapies by people with progressive multiple sclerosis in the United Kingdom

Background: According to current UK guidelines everyone with progressive MS should have access to an MS Specialist but levels of access and use of clinical services is unknown. Our objective was to investigate access to MS Specialists, use of clinical services and disease-modifying therapies (DMTs) by people with progressive MS in the United Kingdom. Methods: A UK wide, online survey was conducted via the UK MS Register. Inclusion criteria: age over 18 years, primary or secondary progressive MS and a member of the UK MS Register. Participants were asked about access to MS Specialists; recent clinical service use; receipt of regular review and current and previous DMT use. Participant demographics; quality of life and disease impact measures were supplied from the UK MS Register. Results: In total 1298 participants responded: 5% were currently taking DMT; 23% had previously taken DMT; and 95% reported access to an MS Specialist. Most utilised services were: MS Doctor/Nurse (50%), General Practitioner (45%), and Physiotherapist (40%). Seventy-four percent received a regular review although 37% received theirs less than annually. Current DMT use was associated with better quality of life but past DMT use was associated with poorer quality of life and higher impact of disease. Conclusions: Access to, and use of, MS Specialists was high. However a gap in service provision was highlighted in both receiving and frequency of regular reviews

The development and validation of a general measure of well-being: the BBC well-being scale

Purpose: The concept of maximising well-being, as opposed to merely treating mental disorder, is a powerful current theme in the area of mental health. Clearly this emphasises the need for appropriate valid and reliable measures of general well-being. This paper examines the appropriateness of a number of measures in this area and concludes that existing assessment tools fail to address the full range of aspects of personal well-being. This paper therefore presents the psychometric properties, validity and reliability of a new measure of well-being-the BBC Well-being Scale. Methods: A total of 1,940 participants completed the new measure, the Goldberg scales of anxiety and depression, the 'List of Threatening Experiences' life events scale, a modified version of the Response Styles Questionnaire and a modified version of the Internal, Personal and Situational Attributions Questionnaire presented via the internet. Results: Exploratory factor-analysis suggested a three-factor solution including themes of psychological well-being, physical health and well-being and relationships. The total 24-item scale had good internal consistency (α = .935) and correlated significantly with key demographic variables and measures of concurrent validity. Conclusions: The new measure-the BBC Well-being Scale-is recommended for research and clinical purposes. © 2011 Springer Science+Business Media B.V

Factors Influencing the Participation of Older People in Clinical Trials : Data Analysis from the MAVIS Trial

Peer reviewedPostprin

Hydroxymethylglutaryl-CoA reductase inhibition with simvastatin in acute lung injury to reduce pulmonary dysfunction (HARP-2) trial : study protocol for a randomized controlled trial

Acute lung injury (ALI) is a common devastating clinical syndrome characterized by life-threatening respiratory failure requiring mechanical ventilation and multiple organ failure. There are in vitro, animal studies and pre-clinical data suggesting that statins may be beneficial in ALI. The Hydroxymethylglutaryl-CoA reductase inhibition with simvastatin in Acute lung injury to Reduce Pulmonary dysfunction (HARP-2) trial is a multicenter, prospective, randomized, allocation concealed, double-blind, placebo-controlled clinical trial which aims to test the hypothesis that treatment with simvastatin will improve clinical outcomes in patients with ALI

The Relative Effectiveness of Pumps Over MDI and Structured Education (REPOSE): study protocol for a cluster randomised controlled trial

Introduction People with type 1 diabetes (T1DM) require insulin therapy to sustain life, and need optimal glycaemic control to prevent diabetic ketoacidosis and serious long-term complications. Insulin is generally administered using multiple daily injections but can also be delivered using an infusion pump (continuous subcutaneous insulin infusion), a more costly option with benefits for some patients. The UK National Institute for Health and Care Excellence (NICE) recommend the use of pumps for patients with the greatest need, citing insufficient evidence to approve extension to a wider population. Far fewer UK adults use pumps than in comparable countries. Previous trials of pump therapy have been small and of short duration and failed to control for training in insulin adjustment. This paper describes the protocol for a large randomised controlled trial comparing pump therapy with multiple daily injections, where both groups are provided with high-quality structured education. Methods and analysis A multicentre, parallel group, cluster randomised controlled trial among 280 adults with T1DM. All participants attended the week-long dose adjustment for normal eating (DAFNE) structured education course, and receive either multiple daily injections or pump therapy for 2 years. The trial incorporates a detailed mixed-methods psychosocial evaluation and cost-effectiveness analysis. The primary outcome will be the change in glycosylated haemoglobin (HbA1c) at 24 months in those participants whose baseline HbA1c is at or above 7.5% (58 mmol/mol). The key secondary outcome will be the proportion of participants reaching the NICE target of an HbA1c of 7.5% (58 mmol/mol) or less at 24 months. Ethics and dissemination The protocol was approved by the Research Ethics Committee North West, Liverpool East and received Medicines and Healthcare products Regulatory Agency (MHRA) clinical trials authorisation. Each participating centre gave National Health Service R&D approval. We shall disseminate study findings to study participants and through peer reviewed publications and conference presentations, including lay user groups. Trial registration number ISRCTN 61215213

The relationship between changes in quality of life outcomes and progression of Alzheimer's disease: results from the Dependence in AD in England 2 longitudinal study

Objective: The relationship between conventional indicators of Alzheimer’s disease (AD) progression and quality of life (QoL) outcomes is unclear. Dependence on others has been recommended as a unifying construct in defining AD severity. This study examined the relationship between indicators of disease severity (including dependence) and changes in QoL and utility over 18-months. Methods: A multi-centre, cohort study was conducted across 18 UK sites. One hundred and forty five patients with possible/probable AD and their caregivers completed assessments of disease severity (Dependence Scale, Mini-Mental State Examination, Neuropsychiatric Inventory, Disability Assessment for Dementia), dementia-specific QoL (DEMQOL, DEMQOL-Proxy) and generic health-related utility (EQ-5D) at both time points. Results: There was evidence of individual change in QoL over 18 months, with over 50% of patients reporting either maintenance or improvement of life quality. The EQ-5D proxy suggested a mean decline in QoL whilst the DEMQOL-Proxy indicated overall improvement. In the subsample of people who self-reported QoL and utility, no mean change was evident. Changes in dependence did not explain changes on any QoL or utility outcome. There was a weak association between the EQ-5D proxy and changes in cognition, whereas changes on the DEMQOL-Proxy were partly explained by changes in behavioural disturbance. Conclusions: The natural progression of AD over 18-months does not lead to inevitable decline in QoL or utility. There are no clear or consistent direct relationships between changes in disease severity and QoL outcomes. The impact of increasing dependence and worsening disease severity is likely buffered by a combination of psychological, social and environmental factors

Supervised exercise training as an adjunct therapy for venous leg ulcers: a randomised controlled feasibility trial

Background Venous leg ulcers (VLUs) are typically painful and heal slowly. Compression therapy offers high healing rates; however, improvements are not usually sustained. Exercise is a low‐cost, low‐risk and effective strategy for improving physical and mental health. Little is known about the feasibility and efficacy of supervised exercise training used in combination with compression therapy patients with VLUs. Objectives To assess the feasibility of a 12‐week supervised exercise programme as an adjunct therapy to compression in patients with VLUs. Methods This was a two‐centre, two‐arm, parallel‐group, randomized feasibility trial. Thirty‐nine patients with venous ulcers were recruited and randomized 1 : 1 either to exercise (three sessions weekly) plus compression therapy or compression only. Progress/success criteria included exercise attendance rate, loss to follow‐up and patient preference. Baseline assessments were repeated at 12 weeks, 6 months and 1 year, with healing rate and time, ulcer recurrence and infection incidents documented. Intervention and healthcare utilization costs were calculated. Qualitative data were collected to assess participants’ experiences. Results Seventy‐two per cent of the exercise group participants attended all scheduled exercise sessions. No serious adverse events and only two exercise‐related adverse events (both increased ulcer discharge) were reported. Loss to follow‐up was 5%. At 12 months, median ulcer healing time was lower in the exercise group (13 vs. 34·7 weeks). Mean National Health Service costs were £813·27 for the exercise and £2298·57 for the control group. Conclusions The feasibility and acceptability of both the supervised exercise programme in conjunction with compression therapy and the study procedures is supported

A RCT of telehealth for COPD patient's Quality of life: The Whole System Demonstrator Evaluation

INTRODUCTION/OBJECTIVES: Despite some concerns that the introduction of telehealth (TH) may lead to reductions in quality of life (QoL), lower mood and increased anxiety in response to using assistive technologies to reduce health care utilisation and manage long term conditions, this research focuses on the extent to which providing people with tools to monitor their condition can improve QoL. METHODS: The Chronic Obstructive Pulmonary Disease cohort of the Whole Systems Demonstrator Trial is a pragmatic General Practitioner (GP) clustered RCT evaluating TH in the UK from three regions in England. All patients at a participating GP practice were deemed eligible for inclusion in the study if they were diagnosed with COPD. RESULTS: 447 participants completed baseline and either a short (4 months) or long term (12 months) follow up. There was a trend of improved QoL and mood in the TH group at longer-term follow up, but not short term follow up. Emotional functioning (g= 0.280 95% CI, 0.051- 0.510) and mastery reached (g= 2.979 95%CI, 0- 0.46) significance at P<0.05 (all Hedges g <0.3). CONCLUSIONS: TH showed minimal benefit to QoL in COPD patients who were not preselected to be at increased risk of acute exacerbations. Benefits were more likely in disease specific measures at longer term follow up. TH is a complex intervention and should be embedded in a service that is evidenced based. Outcome measures must be sensitive enough to detect changes in the target population for the specific intervention. This article is protected by copyright. All rights reserved