Requirements for Quality of Life Instruments in Clinical Research

AbstractThe ability to produce high quality instruments for the assessment of quality of life has advanced considerably in recent years. As the science progresses it has become clear that certain standards must be met if outcome measures are to be capable of providing useful, reliable, and valid information within the context of clinical studies and trials. This paper specifies what these standards are with particular reference to theoretical basis, practicality, acceptability to respondents, unidimensionality, scaling and psychometric properties, and cultural validity and equivalence. The paper also indicates how failure to achieve such standards results in measures that are inaccurate and insensitive to true changes in outcome

Development and validation of a preference based measure derived from the Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) for use in cost utility analyses

<p>Abstract</p> <p>Background</p> <p>Pulmonary Hypertension is a severe and incurable disease with poor prognosis. A suite of new disease-specific measures – the Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) – was recently developed for use in this condition. The purpose of this study was to develop and validate a preference based measure from the CAMPHOR that could be used in cost-utility analyses.</p> <p>Methods</p> <p>Items were selected that covered major issues covered by the CAMPHOR QoL scale (activities, travelling, dependence and communication). These were used to create 36 health states that were valued by 249 people representative of the UK adult population, using the time trade-off (TTO) technique. Data from the TTO interviews were analysed using both aggregate and individual level modelling. Finally, the original CAMPHOR validation data were used to validate the new preference based model.</p> <p>Results</p> <p>The predicted health state values ranged from 0.962 to 0.136. The mean level model selected for analyzing the data had good explanatory power (0.936), did not systematically over- or underestimate the observed mean health state values and showed no evidence of auto correlation in the prediction errors. The value of less than 1 reflects a background level of ill health in state 1111, as judged by the respondents. Scores derived from the new measure had excellent test-retest reliability (0.85) and construct validity. The CAMPHOR utility score appears better able to distinguish between WHO functional classes (II and III) than the EQ-5D and SF-6D.</p> <p>Conclusion</p> <p>The tariff derived in this study can be used to classify an individual into a health state based on their responses to the CAMPHOR. The results of this study widen the evidence base for conducting economic evaluations of interventions designed to improve QoL for patients with PH.</p

Testing a workplace physical activity intervention: a cluster randomized controlled trial

<p>Abstract</p> <p>Background</p> <p>Increased physical activity levels benefit both an individuals' health and productivity at work. The purpose of the current study was to explore the impact and cost-effectiveness of a workplace physical activity intervention designed to increase physical activity levels.</p> <p>Methods</p> <p>A total of 1260 participants from 44 UK worksites (based within 5 organizations) were recruited to a cluster randomized controlled trial with worksites randomly allocated to an intervention or control condition. Measurement of physical activity and other variables occurred at baseline, and at 0 months, 3 months and 9 months post-intervention. Health outcomes were measured during a 30 minute health check conducted in worksites at baseline and 9 months post intervention. The intervention consisted of a 3 month tool-kit of activities targeting components of the Theory of Planned Behavior, delivered in-house by nominated facilitators. Self-reported physical activity (measured using the IPAQ short-form) and health outcomes were assessed.</p> <p>Results and discussion</p> <p>Multilevel modelling found no significant effect of the intervention on MET minutes of activity (from the IPAQ) at any of the follow-up time points controlling for baseline activity. However, the intervention did significantly reduce systolic blood pressure (B = -1.79 mm/Hg) and resting heart rate (B = -2.08 beats) and significantly increased body mass index (B = .18 units) compared to control. The intervention was found not to be cost-effective, however the substantial variability round this estimate suggested that further research is warranted.</p> <p>Conclusions</p> <p>The current study found mixed support for this worksite physical activity intervention. The paper discusses some of the tensions involved in conducting rigorous evaluations of large-scale randomized controlled trials in real-world settings.</p> <p>Trial registration</p> <p>Current controlled trials <a href="http://www.controlled-trials.com/ISRCTN08807396">ISRCTN08807396</a></p

Development and psychometric assessment of the COPD and Asthma Sleep Impact Scale (CASIS)

<p>Abstract</p> <p>Background</p> <p>Patients with respiratory disease experience disturbed sleep, but there is no widely accepted measure of sleep impairment due to respiratory disease. We developed and evaluated the psychometric performance of a patient-reported measure to assess the impact on sleep due to respiratory disease, the COPD and Asthma Sleep Impact Scale (CASIS).</p> <p>Methods</p> <p>Identification of the items forming the CASIS was guided by patient interviews and focus groups. An observational study involving patients from the US and UK was then conducted to assess the psychometric characteristics of the measure.</p> <p>Results</p> <p>Qualitative data from 162 patients were used to develop the CASIS (n = 78 COPD; n = 84 asthma). The observational study included 311 patients with COPD and 324 patients with asthma. The final seven items used in the CASIS were identified based on factor and item response theory analyses. Internal consistency was 0.90 (COPD) and 0.92 (asthma), and test-retest reliability was 0.84 (both groups). In the COPD sample, CASIS scores were significantly correlated with the Saint George's Respiratory Questionnaire scores (all p < 0.0001) and differed significantly by patient-reported disease severity, exacerbation status, and overall health status (all p ≤ 0.005). In the asthma sample, CASIS scores were significantly correlated with the Asthma Quality of Life Questionnaire scores (all p < 0.0001) and differed significantly by clinician and patient-reported disease severity, exacerbation status, and overall health status (all p ≤ 0.0005).</p> <p>Conclusion</p> <p>The CASIS shows good internal consistency, test-retest reliability, and construct validity and may be useful in helping to understand the impact that COPD and asthma have on sleep outcomes.</p

Breast cancer in lesbians and bisexual women: Systematic review of incidence, prevalence and risk studies

This article is made available through the Brunel Open Access Publishing Fund. © 2013 Meads and Moore; licensee BioMed Central Ltd. This is an open access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.Background: The UK Parliamentary Enquiry and USA Institute of Medicine state that lesbians may be at a higher risk of breast cancer but there is insufficient information. Lesbians and bisexual (LB) women have behavioural risk-factors at higher rates compared to heterosexuals such as increased alcohol intake and higher stress levels. Conversely, breast cancer rates are higher in more affluent women yet income levels in LB women are relatively low. This systematic review investigated all evidence on whether there is, or likely to be, higher rates of breast cancer in LB women. Methods: Cochrane library (CDSR, CENTRAL, HTA, DARE, NHSEED), MEDLINE, EMBASE, PsychINFO, CAB abstracts, Web of Science (SCI, SSCI), SIGLE and Social Care Online databases were searched to October 2013. Unpublished research and specific lesbian, gay and bisexual websites were checked, as were citation lists of relevant papers. Included were studies in LB populations reporting breast cancer incidence or prevalence rates, risk model results or risk-factor estimates. Inclusions, data-extraction and quality assessment were by two reviewers with disagreements resolved by discussion. Results: Searches found 198 references. No incidence rates were found. Nine studies gave prevalence estimates - two showed higher, four showed no differences, one showed mixed results depending on definitions, one had no comparison group and one gave no sample size. All studies were small with poor methodological and/or reporting quality. One incidence modelling study suggested a higher rate. Four risk modelling studies were found, one Rosner-Colditz and three Gail models. Three suggested higher and one lower rate in LB compared to heterosexual women. Six risk-factor estimates suggested higher risk and one no difference between LB and heterosexual women. Conclusions: The only realistic way to establish rates in LB women would be to collect sexual orientation within routine statistics, including cancer registry data, or from large cohort studies

Development and validation of the Asthma Life Impact Scale (ALIS)

SummaryBackgroundCurrent asthma patient-reported outcome (PRO) measures focus on symptoms and functioning and may not capture the holistic impact of asthma on the quality of life of the patient.ObjectiveTo develop a PRO measure capturing the overall impact of asthma on patient's quality of life.MethodsItems for the Asthma Life Impact Scale (ALIS) were generated from patients with asthma during interviews in the UK and focus groups in the US. The ALIS was tested with UK and US asthma patients during cognitive debriefing interviews and included in large, two-administration, validation studies in the UK and US.ResultsIssues raised by asthma patients during interviews (n = 39 patients) and focus groups (n = 16 patients) were included in the draft ALIS. Cognitive debriefing interviews with 29 UK and US asthma patients showed that the scale was relevant and comprehensive. 140 UK and 185 US asthma patients participated in the validation study. The analysis showed that the ALIS measures a single construct, namely the overall impact of asthma on patients' quality of life. Internal consistency (Cronbach's Alpha) was high (UK = 0.94; US = 0.92) as was test-retest reliability (UK = 0.93; US = 0.83). Patients reporting worse general health or more severe asthma had significantly higher ALIS scores (p < 0.001) (indicating greater negative impact of asthma). Correlations with the Asthma Quality of Life Questionnaire were moderate to high.ConclusionsThe final 22-item ALIS is unidimensional, reliable and valid, and a valuable tool for comprehensively assessing the holistic impact of asthma from the patient's perspective

Can We Rely on the Dermatology Life Quality Index as a Measure of the Impact of Psoriasis or Atopic Dermatitis?

The Dermatology Life Quality Index (DLQI) is a widely used health-related quality of life measure. However, little research has been conducted on its dimensionality. The objectives of the current study were to apply Rasch analysis to DLQI data to determine whether the scale is unidimensional, to assess its measurement properties, test the response format, and determine whether the measure exhibits differential item functioning (DIF) by disease (atopic dermatitis versus psoriasis), gender, or age group. The results show that there were several problems with the scale, including misfitting items, DIF by disease, age, and gender, disordered response thresholds, and inadequate measurement of patients with mild illness. As the DLQI did not benefit from the application of Rasch analysis in its development, it is argued that a new measure of disability related to dermatological disease is required. Such a measure should use a coherent measurement model and ensure that items are relevant to all potential respondents. The current use of the DLQI as a guide to treatment selection is of concern, given its inadequate measurement properties

Recruiting in surgical trial in the emergency setting: understanding clinician and patient perspectives: a mixed methods study

Background. Undertaking randomised controlled trials (RCT) in emergency surgical setting is associated with methodological and practical challenges. This study explored patients and clinicians’ perspectives associated with the conduct of a RCT comparing laparoscopic and open colorectal surgery in the acute setting.Method. All eligible patients screened/enrolled for the ‘Laparoscopic versus Open Colorectal Surgery in the Acute Setting multicentre, randomised controlled feasibility (LaCeS) trial’ in five NHS Trusts were invited to respond to a survey. Also, patients and healthcare professionals were invited to take part in semi-structured interviews. Survey and interviews explored acceptability of the feasibility trial. Interviews were audio-recorded, transcribed verbatim and analysed using thematic analysis. Survey data were analysed descriptively to assess patient views of the trial and intervention. Results. Out of 72 patients enrolled for the LaCeS RCT, survey data was collected from 28 patients (38.9%), and interviews were conducted with 16 patients and 14 healthcare professionals. Thirteen out of 28 patients (46%) had treatment preferences but these were not strong enough to deter participation. Twelve of the patients interviewed believed their surgeon preferred laparoscopic surgery, but this did not deter them from participating in the trial. Half of the surgeons interviewed expressed the view that laparoscopic surgery was of benefit in this setting, but recognised that the need for research evidence outweighed their personal treatment preferences. Eight of the 14 recruiters reported that the emergency setting affected recruitment, especially in centres with fewer recruiting surgeons. Interviewees reported that recruitment was helped significantly by using surgical trainees to consent patients. Conclusion. This study identified specific challenges for the LaCeS2 trial design to address, and adds to our understanding of recruiting to emergency surgical trials more broadly

Efficacy and safety of co-careldopa as an add-on therapy to occupational and physical therapy in patients after stroke (DARS): a randomised, double-blind, placebo controlled trial

Background Dopamine is a key modulator of striatal function and learning and might improve motor recovery after stroke. Previous small trials of dopamine agonists after stroke provide equivocal evidence of effectiveness on improving motor recovery. We aimed to assess the safety and efficacy of co-careldopa plus routine occupational and physical therapy during early rehabilitation after stroke. Methods This double-blind, multicentre, randomised controlled trial of co-careldopa versus placebo in addition to routine NHS occupational and physical therapy was done at 51 UK NHS acute inpatient stroke rehabilitation services. We recruited patients with new or recurrent clinically diagnosed ischaemic or haemorrhagic (excluding subarachnoid haemorrhage) stroke 5–42 days before randomisation, who were unable to walk 10 m or more, had a score of less than 7 points on the Rivermead Mobility Index, were expected to need rehabilitation, and were able to access rehabilitation after discharge from hospital. Participants were assigned (1:1) using stratified random blocks to receive 6 weeks of oral co-careldopa or matched placebo in addition to routine NHS physiotherapy and occupational therapy. The initial two doses of co-careldopa were 62·5 mg (50 mg of levodopa and 12·5 mg of carbidopa) and the remaining doses were 125 mg (100 mg of levodopa and 25 mg of carbidopa). Participants were required to take a single oral tablet 45–60 min before physiotherapy or occupational therapy session. The primary outcome was ability to walk independently, defined as a Rivermead Mobility Index score of 7 or more, at 8 weeks. Primary and safety analyses were done in the intention-to-treat population. The trial is registered on the ISRCTN registry, number ISRCTN99643613.Findings Between May 30, 2011, and March 28, 2014, of 1574 patients found eligible, 593 (mean age 68·5 years) were randomly assigned to either the co-careldopa group (n=308) or to the placebo group (n=285), on an average 18 days after stroke onset. Primary outcome data were available for all 593 patients. We found no evidence that the ability to walk independently improved with co-careldopa (125 [41%] of 308 patients) compared with placebo (127 [45%] of 285 patients; odds ratio 0·78 [95% CI 0·53–1·15]) at 8 weeks. Mortality at 12 months did not differ between the two groups (22 [7%] vs 17 [6%]). Serious adverse events were largely similar between groups. Vomiting during therapy sessions, after taking the study drug, was the most frequent adverse event and was more frequent in the co-careldopa group than the placebo group (19 [6·2%] vs 9 [3·2%]).Interpretation Co-careldopa in addition to routine occupational and physical therapy does not seem to improve walking after stroke. Further research might identify subgroups of patients with stroke who could benefit from dopaminergic therapy at different doses or times after stroke with more intensive motor therap

The effectiveness of early lens extraction with intraocular lens implantation for the treatment of primary angle-closure glaucoma (EAGLE) : study protocol for a randomized controlled trial

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