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Background: Regulatory guidance recommends anchor-based methods for interpretation of treatment effects measured by PRO endpoints. Methodological pros and cons of patient global ratings of change vs. patient global ratings of concept have been discussed but empirical evidence in support of either approach is lacking. This study evaluated the performance of patient global ratings of change and patient global ratings of concept for interpreting patient stability and patient improvement.Methods: Patient global ratings of change and patient global ratings of concept were included in a psychometric validation study of an osteoporosis-targeted PRO instrument (the OPAQ-PF) to assess its ability to detect change and to derive responder definitions. 144 female osteoporosis patients with (n = 37) or without (n = 107) a recent (within 6 weeks) fragility fracture completed the OPAQ-PF and global items at baseline, 2 weeks (no recent fracture), and 12 weeks (recent fracture) post-baseline.Results: Results differed between the two methods. Recent fracture patients reported more improvement while patients without recent fracture reported more stability on ratings of change than ratings of concept. However, correlations with OPAQ-PF score change were stronger for ratings of concept than ratings of change (both groups). Effect sizes for OPAQ-PF score change increased consistently with level of change in ratings of concept but inconsistently with ratings of change, with the mean AUC for prediction of a one-point change being 0.72 vs. 0.56.Conclusions: This study provides initial empirical support for methodological and regulatory recommendations to use patient global ratings of concept rather than ratings of change when interpreting change captured by PRO instruments in studies evaluating treatment effects. These findings warrant being confirmed in a purpose-designed larger scale analysis. [ABSTRACT FROM AUTHOR]

Objective: To conduct a systematic review and meta-analysis of the equivalence between electronic and paper administration of patient reported outcome measures (PROMs) in studies conducted subsequent to those included in Gwaltney et al's 2008 review.Methods: A systematic literature review of PROM equivalence studies conducted between 2007 and 2013 identified 1,997 records from which 72 studies met pre-defined inclusion/exclusion criteria. PRO data from each study were extracted, in terms of both correlation coefficients (ICCs, Spearman and Pearson correlations, Kappa statistics) and mean differences (standardized by the standard deviation, SD, and the response scale range). Pooled estimates of correlation and mean difference were estimated. The modifying effects of mode of administration, year of publication, study design, time interval between administrations, mean age of participants and publication type were examined.Results: Four hundred thirty-five individual correlations were extracted, these correlations being highly variable (I2 = 93.8) but showing generally good equivalence, with ICCs ranging from 0.65 to 0.99 and the pooled correlation coefficient being 0.88 (95% CI 0.87 to 0.88). Standardised mean differences for 307 studies were small and less variable (I2 = 33.5) with a pooled standardised mean difference of 0.037 (95% CI 0.031 to 0.042). Average administration mode/platform-specific correlations from 56 studies (61 estimates) had a pooled estimate of 0.88 (95% CI 0.86 to 0.90) and were still highly variable (I2 = 92.1). Similarly, average platform-specific ICCs from 39 studies (42 estimates) had a pooled estimate of 0.90 (95% CI 0.88 to 0.92) with an I2 of 91.5. After excluding 20 studies with outlying correlation coefficients (≥3SD from the mean), the I2 was 54.4, with the equivalence still high, the overall pooled correlation coefficient being 0.88 (95% CI 0.87 to 0.88). Agreement was found to be greater in more recent studies (p < 0.001), in randomized studies compared with non-randomised studies (p < 0.001), in studies with a shorter interval (<1 day) (p < 0.001), and in respondents of mean age 28 to 55 compared with those either younger or older (p < 0.001). In terms of mode/platform, paper vs Interactive Voice Response System (IVRS) comparisons had the lowest pooled agreement and paper vs tablet/touch screen the highest (p < 0.001).Conclusion: The present study supports the conclusion of Gwaltney's previous meta-analysis showing that PROMs administered on paper are quantitatively comparable with measures administered on an electronic device. It also confirms the ISPOR Taskforce´s conclusion that quantitative equivalence studies are not required for migrations with minor change only. This finding should be reassuring to investigators, regulators and sponsors using questionnaires on electronic devicesafter migration using best practices. Although there is data indicating that migrations with moderate changes produce equivalent instrument versions, hence do not require quantitative equivalence studies, additional work is necessary to establish this. Furthermore, there is the need to standardize migration practices and reporting practices (i.e. include copies of tested instrument versions and screenshots) so that clear recommendations regarding equivalence testing can be made in the future.raising questions about the necessity of conducting equivalence testing moving forward. [ABSTRACT FROM AUTHOR]

Background Enhancing quality of life for people with long-term conditions by monitoring patient-reported outcome measure scores is a key domain of health care policy. This study investigated the responsiveness of patient-reported outcome measures for long-term conditions. Methods A cohort survey was conducted in 33 primary care practices and 4485 patients (1334 asthma, 567 chronic obstructive pulmonary disease, 1121 diabetes, 525 epilepsy, 520 heart failure and 418 stroke) were sent a baseline survey containing a generic (EQ-5D) and a disease-specific measure. Baseline respondents were sent a follow-up after 1 year. Differences in scores for each long-term condition were assessed by paired t-tests. The relationship between scores and self-reported 'change in health' was assessed by analysis of variance. Results The baseline achieved a 38.4% response rate and the follow-up 71.5%. The only significant difference for the EQ-5D was found for the Visual Analogue Scale in heart failure between baseline and follow-up, and for change in health. Significant differences between baseline and follow-up scores were found on the disease-specific measures for 1 asthma dimension and 1 stroke dimension. No significant differences were found for other conditions. Significant differences between self-reported change in health and the disease-specific measures were found for 4 asthma dimensions and 2 stroke dimensions. Conclusions Few significant differences were found between the baseline and follow up or between 'change in health' and PROMs scores. This could be explained by the time frame of one year being too short for change to occur or by the PROMs not being responsive enough to change in a primary care sample. The latter is unlikely as the PROMs were in part chosen for their responsiveness to change. The baseline response rates may mean that the sample is not representative, and stable patients may have been more likely to participate. If PROMs are to be used routinely to monitor outcomes in LTCs, further research is needed to maximize response rates, to ensure that the PROMs used are reliable, valid and sensitive enough to detect change and that the time frame for data collection is appropriate. [ABSTRACT FROM AUTHOR]

Background: The Whole Systems Demonstrator was a large, pragmatic, cluster randomised trial that compared telehealth with usual care among 3,230 patients with long-term conditions in three areas of England. Telehealth involved the regular transmission of physiological information such as blood glucose to health professionals working remotely. We examined whether telehealth led to changes in glycosylated haemoglobin (HbA1c) among the subset of patients with type 2 diabetes. Methods: The general practice electronic medical record was used as the source of information on HbA1c. Effects on HbA1c were assessed using a repeated measures model that included all HbA1c readings recorded during the 12-month trial period, and adjusted for differences in HbA1c readings recorded before recruitment. Secondary analysis averaged multiple HbA1c readings recorded for each individual during the trial period. Results: 513 of the 3,230 participants were identified as having type 2 diabetes and thus were included in the study. Telehealth was associated with lower HbA1c than usual care during the trial period (difference 0.21% or 2.3 mmol/mol, 95% CI, 0.04% to 0.38%, p = 0.013). Among the 457 patients in the secondary analysis, mean HbA1c showed little change for controls following recruitment, but fell for intervention patients from 8.38% to 8.15% (68 to 66 mmol/mol). A higher proportion of intervention patients than controls had HbA1c below the 7.5% (58 mmol/mol) threshold that was targeted by general practices (30.4% vs. 38.0%). This difference, however, did not quite reach statistical significance (adjusted odds ratio 1.63, 95% CI, 0.99 to 2.68, p = 0.053). Conclusions: Telehealth modestly improved glycaemic control in patients with type 2 diabetes over 12 months. The scale of the improvements is consistent with previous meta-analyses, but was relatively modest and seems unlikely to produce significant patient benefit. [ABSTRACT FROM AUTHOR]

Background: In the majority of patients with osteoarthritis of the knee the disease originates in the medial compartment. There are two fundamentally different approaches to knee replacement for patients with unicompartmental disease: some surgeons feel that it is always best to replace both the knee compartments with a total knee replacement (TKR); whereas others feel it is best to replace just the damaged component of the knee using a partial or unicompartment replacement (UKR). Both interventions are established and well-documented procedures. Little evidence exists to prove the clinical and cost-effectiveness of either management option. This provides an explanation for the high variation in treatment of choice by individual surgeons for the same knee pathology. The aim of the TOPKAT study will be to assess the clinical and cost effectiveness of TKRs compared to UKRs in patients with medial compartment osteoarthritis. Methods/Design: The design of the study is a single layer multicentre superiority type randomised controlled trial of unilateral knee replacement patients. Blinding will not be possible as the surgical scars for each procedure differ. We aim to recruit 500 patients from approximately 28 secondary care orthopaedic units from across the UK including district general and teaching hospitals. Participants will be randomised to either UKR or TKR. Randomisation will occur using a web-based randomisation system. The study is pragmatic in terms of implant selection for the knee replacement operation. Participants will be followed up for 5 years. The primary outcome is the Oxford Knee Score, which will be collected via questionnaires at 2 months, 1 year and then annually to 5 years. Secondary outcomes will include cost-effectiveness, patient satisfaction and complications data. [ABSTRACT FROM AUTHOR]

Background: Telehealth is increasingly used in the care of people with long term conditions. Whilst many studies look at the impacts of the technology on hospital use, few look at how it changes contacts with primary care professionals. The aim of this paper was to assess the impacts of home-based telehealth interventions on general practice contacts. Method: Secondary analysis of data from a Department of Health funded cluster-randomised trial with 179 general practices in three areas of England randomly assigned to offer telehealth or usual care to eligible patients. Telehealth included remote exchange of vitals signs and symptoms data between patients and healthcare professionals as part of the continuing management of patients. Usual care reflected the range of services otherwise available in the sites, excluding telehealth. Anonymised data from GP systems were used to construct person level histories for control and intervention patients. We tested for differences in numbers of general practitioner and practice nurse contacts over twelve months and in the number of clinical readings recorded on general practice systems over twelve months. Results: 3,230 people with diabetes, chronic obstructive pulmonary disease or heart failure were recruited in 2008 and 2009. 1219 intervention and 1098 control cases were available for analysis. No statistically significant differences were detected in the numbers of general practitioner or practice nurse contacts between intervention and control groups during the trial, or in the numbers of clinical readings recorded on the general practice systems. Conclusions: Telehealth did not appear associated with different levels of contact with general practitioners and practice nurses. We note that the way that telehealth impacts on primary care roles may be influenced by a number of other features in the health system. The challenge is to ensure that these systems lead to better integration of care than fragmentation. [ABSTRACT FROM AUTHOR]

Background: Neurological conditions have a substantial impact on carers, with carer well-being having been shown to be influenced by a number of demographic, patient and caregiving factors. Support given to carers can lead to better coping. This study investigated the relationship between carer well-being and experiences with health and social services. Methods: A cross-sectional survey was conducted of 1910 (37.4%) of carers of 5109 people with motor neuron disease (MND) (n=434, 54.9%), multiple sclerosis (MS) (n=721, 30.7%) and Parkinson's disease (PD) (n=755, 38.2%). Carers completed a generic health status measure (SF-12), a carer strain measure (Carer Strain Index- CSI) and a newly developed questionnaire on health and social care experiences. Data were analysed by analysis of variance with p set at <0.05. Results: Carer well-being was found to be compromised and differed significantly between the three conditions. Furthermore, a considerable number of carers experienced problems with aspects of health and social care, although there was no clear pattern according to the condition that was cared for. The total number of problems reported did not differ significantly between conditions but was significantly (all p<0.001) associated with carer quality of life (both physical and mental health) and strain, even when other influencing factors (demographic and caregiving variables) were corrected for. The association was particularly strong for carer strain, and less strong (but still significant) for quality of life. Conclusions: The results show that carer well-being is compromised, in line with previous studies. Furthermore, the link of carer well-being to the number of problems reported suggests that minimizing problems experienced could improve carer well-being. This stresses the importance of health and social services appropriately supporting carers. [ABSTRACT FROM AUTHOR]

Background: It is expected that increased demands on services will result from expanding numbers of older people with long-term conditions and social care needs. There is significant interest in the potential for technology to reduce utilisation of health services in these patient populations, including telecare (the remote, automatic and passive monitoring of changes in an individual's condition or lifestyle) and telehealth (the remote exchange of data between a patient and health care professional). The potential of telehealth and telecare technology to improve care and reduce costs is limited by a lack of rigorous evidence of actual impact., Methods/design: We are conducting a large scale, multi-site study of the implementation, impact and acceptability of these new technologies. A major part of the evaluation is a cluster-randomised controlled trial of telehealth and telecare versus usual care in patients with long-term conditions or social care needs. The trial involves a number of outcomes, including health care utilisation and quality of life. We describe the broad evaluation and the methods of the cluster randomised trial, Discussion: If telehealth and telecare technology proves effective, it will provide additional options for health services worldwide to deliver care for populations with high levels of need., Trial Registration: Current Controlled Trials ISRCTN43002091.

Background: The compulsory treatment of anorexia nervosa is a contentious issue. Research suggests that psychiatrists have a range of attitudes towards patients suffering from anorexia nervosa, and towards the use of compulsory treatment for the disorder., Methods: A postal self-completed attitudinal questionnaire was sent to senior psychiatrists in the United Kingdom who were mostly general adult psychiatrists, child and adolescent psychiatrists, or psychiatrists with an interest in eating disorders., Results: Respondents generally supported a role for compulsory measures under mental health legislation in the treatment of patients with anorexia nervosa. Compared to 'mild' anorexia nervosa, respondents generally were less likely to feel that patients with 'severe' anorexia nervosa were intentionally engaging in weight loss behaviours, were able to control their behaviours, wanted to get better, or were able to reason properly. However, eating disorder specialists were less likely than other psychiatrists to think that patients with 'mild' anorexia nervosa were choosing to engage in their behaviours or able to control their behaviours. Child and adolescent psychiatrists were more likely to have a positive view of the use of parental consent and compulsory treatment for an adolescent with anorexia nervosa. Three factors emerged from factor analysis of the responses named: 'Support for the powers of the Mental Health Act to protect from harm'; 'Primacy of best interests'; and 'Autonomy viewed as being preserved in anorexia nervosa'. Different scores on these factor scales were given in terms of type of specialist and gender., Conclusion: In general, senior psychiatrists tend to support the use of compulsory treatment to protect the health of patients at risk and also to protect the welfare of patients in their best interests. In particular, eating disorder specialists tend to support the compulsory treatment of patients with anorexia nervosa independently of views about their decision-making capacity, while child and adolescent psychiatrists tend to support the treatment of patients with anorexia nervosa in their best interests where decision-making is impaired.