Modelling treatment, age- and gender-specific recovery in acute injury studies

Background: Acute injury studies often measure physical ability repeatedly over time through scores that have a finite range. This can result in a faster score change at the beginning of the study than towards the end, motivating the investigation of the rate of change. Additionally, the bounds of the score and their dependence on covariates are often of interest. Methods: We argue that transforming bounded data is not satisfactory in some settings. Motivated by the Collaborative Ankle Support Trial (CAST), which investigated different methods of immobilisation for severe ankle sprains, we developed a model under the assumption that the recovery rate at a specific time is proportional to the current score and the remaining score. This model enables a direct interpretation of the covariate effects. We have re-analyzed the CAST data using these improved methods, and explored novel relationships between age, gender and recovery rate. Results: We confirm that using below knee cast is advantageous compared with a tubular bandage in relation with the recovery rate. An age and gender effect on the recovery rate and the maximum achievable score is demonstrated, with older female patients recovering less fast (age-effect: -0.21, 95% confidence interval (CI) [-0.28,- 0.14]; gender effect: -0.06, CI [-0.12,-0.004]) and achieving a lower maximum score (age-effect: -8.07, CI [-11.68,-4.01]; gender-effect: -5.34, CI [-8.18, -2.50]) than younger male patients. Conclusions: Our model is able to accurately model repeated measurements on the original scale, while accounting for the bounded nature of a score. We demonstrate that recovery in acute injury trials can differ substantially by age and gender. Older female patients are less likely to recover well from a sprain

Can lay-led walking programmes increase physical activity in middle aged adults? : a randomised controlled trial

Study objective: To compare health walks, a community based lay-led walking scheme versus advice only on physical activity and cardiovascular health status in middle aged adults. Design: Randomised controlled trial with one year follow up. Physical activity was measured by questionnaire. Other measures included attitudes to exercise, body mass index, cholesterol, aerobic capacity, and blood pressure. Setting: Primary care and community. Participants: 260 men and women aged 40–70 years, taking less than 120 minutes of moderate intensity activity per week. Main results: Seventy three per cent of people completed the trial. Of these, the proportion increasing their activity above 120 minutes of moderate intensity activity per week was 22.6% in the advice only and 35.7% in the health walks group at 12 months (between group difference =13% (95% CI 0.003% to 25.9%) p=0.05). Intention to treat analysis, using the last known value for missing cases, demonstrated smaller differences between the groups (between group difference =6% (95% CI -5% to 16.4%)) with the trend in favour of health walks. There were improvements in the total time spent and number of occasions of moderate intensity activity, and aerobic capacity, but no statistically significant differences between the groups. Other cardiovascular risk factors remained unchanged. Conclusions: There were no significant between group differences in self reported physical activity at 12 month follow up when the analysis was by intention to treat. In people who completed the trial, health walks was more effective than giving advice only in increasing moderate intensity activity above 120 minutes per week

The Achilles tendon total rupture score : a study of responsiveness, internal consistency and convergent validity on patients with acute Achilles tendon ruptures

Background The Achilles tendon Total Rupture Score was developed by a research group in 2007 in response to the need for a patient reported outcome measure for this patient population. Beyond this original development paper, no further validation studies have been published. Consequently the purpose of this study was to evaluate internal consistency, convergent validity and responsiveness of this newly developed patient reported outcome measure within patients who have sustained an isolated acute Achilles tendon rupture. Methods Sixty-four eligible patients with an acute rupture of their Achilles tendon completed the Achilles tendon Total Rupture Score alongside two further patient reported outcome measures (Disability Rating Index and EQ 5D). These were completed at baseline, six weeks, three months, six months and nine months post injury. The Achilles tendon Total Rupture Score was evaluated for internal consistency, using Cronbach's alpha, convergent validity, through correlation analysis and responsiveness, by analysing floor and ceiling effects and calculating its relative efficiency in comparison to the Disability Rating Index and EQ 5D scores. Results The Achilles tendon Total Rupture Score demonstrated high internal consistency (Cronbachs alpha > 0.8) and correlated significantly (p < 0.001) with the Disability Rating Index at five time points (pre-injury, six weeks, three, six and nine months) with correlation coefficients between -0.5 and -0.9. However, the confidence intervals were wide. Furthermore, the ability of the new score to detect clinically important changes over time (responsiveness) was shown to be greater than the Disability Rating Index and EQ 5D. Conclusions A universally accepted outcome measure is imperative to allow comparisons to be made across practice. This is the first study to evaluate aspects of validity of this newly developed outcome measure, outside of the developing centre. The ATRS demonstrated high internal consistency and responsiveness, with limited convergent validity. This research provides further support for the use of this outcome measure, however further research is required to advocate its universal use in patients with acute Achilles tendon ruptures. Such areas include inter-rater reliability and research to determine the minimally clinically important difference between scores

An empirical evaluation of the SF-12, SF-6D, EQ-5D and Michigan Hand Outcome Questionnaire in patients with rheumatoid arthritis of the hand

Background The aim of this study was to assess the psychometric properties, namely acceptability, validity, reliability, interpretability and responsiveness of the EuroQol EQ-5D (EQ-5D visual analogue (VAS) and EQ-5D (utility)), Short Form 12 Dimensions (SF-12), SF-6D and Michigan Hand Outcome Questionnaire (MHQ) in patients with rheumatoid arthritis (RA) of the hand. Methods The empirical investigation was based upon data from a randomised controlled trial of 488 adults with rheumatoid arthritis who had pain and dysfunction of the hands and/or wrists. Participants completed the EQ-5D, SF-12 and MHQ at baseline and at 4 and 12 months follow up. Acceptability was measured using completion rates over time; construct validity using the “known groups” approach, based on pain troublesomeness; convergent validity using spearman’s rho correlation (ρ); reliability using internal consistency (Cronbach’s alpha); interpretability using minimal important differences (MID); and responsiveness using effect sizes and standardised response means (SRM) stratified by level of self-rated improvement in hand and wrist function or level of self-rated benefit and satisfaction from trial treatments. Results At baseline, the study population had a mean age of 62.4 years, a mean MHQ score of 52.1 and included 76% women. The EQ-5D (utility) had the highest completion rates across time points. All instruments discriminated between pre-specified groups based on pain troublesomeness. Convergent validity analysis indicated that the MHQ score correlated strongly with the EQ-5D (ρ = 0.65) and SF-6D (ρ = 0.63) utility scores. The MHQ was most responsive at detecting change in indicators of RA pain severity between baseline and 4 months, whilst minimal important differences varied considerably across PROMs. Conclusions The instruments evaluated in this study displayed varying psychometric properties in the context of RA of the hand. The selection of a preferred instrument in evaluative studies should ultimately depend on the relative importance placed on individual psychometric properties and the importance placed on generation of health utilities for economic evaluation purposes

Can mapping algorithms based on raw scores overestimate QALYs gained by treatment? A comparison of mappings between the Roland–Morris Disability Questionnaire and the EQ-5D-3L based on raw and Differenced Score Data

Introduction Mapping algorithms are increasingly being used to predict health-utility values based on responses or scores from non-preference-based measures, thereby informing economic evaluations. Objectives We explored whether predictions in the EuroQol 5-dimension 3-level instrument (EQ-5D-3L) health-utility gains from mapping algorithms might differ if estimated using differenced versus raw scores, using the Roland–Morris Disability Questionnaire (RMQ), a widely used health status measure for low back pain, as an example. Methods We estimated algorithms mapping within-person changes in RMQ scores to changes in EQ-5D-3L health utilities using data from two clinical trials with repeated observations. We also used logistic regression models to estimate response mapping algorithms from these data to predict within-person changes in responses to each EQ-5D-3L dimension from changes in RMQ scores. Predicted health-utility gains from these mappings were compared with predictions based on raw RMQ data. Results Using differenced scores reduced the predicted health-utility gain from a unit decrease in RMQ score from 0.037 (standard error [SE] 0.001) to 0.020 (SE 0.002). Analysis of response mapping data suggests that the use of differenced data reduces the predicted impact of reducing RMQ scores across EQ-5D-3L dimensions and that patients can experience health-utility gains on the EQ-5D-3L ‘usual activity’ dimension independent from improvements captured by the RMQ. Conclusion Mappings based on raw RMQ data overestimate the EQ-5D-3L health utility gains from interventions that reduce RMQ scores. Where possible, mapping algorithms should reflect within-person changes in health outcome and be estimated from datasets containing repeated observations if they are to be used to estimate incremental health-utility gains

A longitudinal, qualitative study exploring sustained adherence to a hand exercise programme for Rheumatoid Arthritis evaluated in the SARAH Trial

Purpose: This study explores the experience of participants taking part in a hand exercise programme for people with rheumatoid arthritis with a focus on adherence. The exercise programme was tested in a randomised controlled trial. This parallel qualitative study will inform future implementation into clinical practice. Method: Twenty-seven semi-structured interviews from 14 participants were undertaken at 2 time points (4 and 12 months after randomisation). We collected data of participants’ experiences over time. This was guided by an interview schedule. Interview data were analysed using interpretative phenomenological analysis which is informed by phenomenological and hermeneutic theory. We recruited participants from National Health Service rheumatology and therapy departments. Results: At 4 months, 11/14 participants reported continuing with the exercises. By 12 months, 7/13 participants still reported exercising. The ability to establish a routine determined whether participants adhered to the exercise programme. This was sometimes influenced by practical issues. We also identified facilitators and barriers to regular exercise in the themes of – the therapeutic encounter, perceived benefit of exercises, attitude of mind, confidence and unpredictability. Conclusions: Establishing a routine was an important step towards participants being able to exercise independently. Therapists provided participants with skills to continue to exercise while dealing with changes in symptoms and schedules. Potential barriers to long term exercise adherence need to be taken into account and addressed for successful implementation of this programme

Does a monetary incentive improve the response to a postal questionnaire in a randomised controlled trial? : the MINT incentive study

Background: Sending a monetary incentive with postal questionnaires has been found to improve the proportion of responders, in research in non-healthcare settings. However, there is little research on use of incentives to improve follow-up rates in clinical trials, and existing studies are inconclusive. We conducted a randomised trial among participants in the Managing Injuries of the Neck Trial (MINT) to investigate the effects on the proportion of questionnaires returned and overall non-response of sending a £5 gift voucher with a follow-up questionnaire. Methods: Participants in MINT were randomised to receive either: (a) a £5 gift voucher (incentive group) or (b) no gift voucher (no incentive group), with their 4 month or 8 month follow-up questionnaire. We recorded, for each group, the number of questionnaires returned, the number returned without any chasing from the study office, the overall number of non-responders (after all chasing efforts by the study office), and the costs of following up each group. Results: 2144 participants were randomised, 1070 to the incentive group and 1074 to the no incentive group. The proportion of questionnaires returned (RR 1.10 (95% CI 1.05, 1.16)) and the proportion returned without chasing (RR 1.14 (95% CI 1.05, 1.24) were higher in the incentive group, and the overall non-response rate was lower (RR 0.68 (95% CI 0.53, 0.87)). Adjustment for injury severity and hospital of recruitment to MINT made no difference to these results, and there were no differences in results between the 4-month and 8-month follow up questionnaires. Analysis of costs suggested a cost of £67.29 per additional questionnaire returned. Conclusion: Monetary incentives may be an effective way to increase the proportion of postal questionnaires returned and minimise loss to follow-up in clinical trials

Participation in a trial in the emergency situation : a qualitative study of patient experience in the UK WOLLF trial

Background Patients can struggle to make sense of trials in emergency situations. This study examines patient experience of participating in the United Kingdom, Wound management of Open Lower Limb Fractures (UK WOLLF) study, a trial of standard wound management versus Negative Pressure Wound Therapy (NPWT). Methods The aim of the study was to understand the patient’s lived experience of taking part in a trial of wound dressings. Interviews drawing on Phenomenology were undertaken with a purposive sample of 20 patients, on average 12 days into their hospital stay from July 2012–July 2013. Results The participants were vulnerable due to the emotional and physical impact of injury. They expressed their trial experience through the theme of being compromised identified in categories of being dependent, being trusting, being grateful and being without experience. Participants felt dependent on and trusted the team to make the right decisions for them and not cause them harm. Their hopes for future recovery were also invested within the expertise of the team. Despite often not being well enough to consent to the study prior to surgery, they wished to be involved as much as possible. In agreeing to take part they expressed gratitude for their care, wanted to be helpful to others and considered the trial interventions to be a small component in relation to the enormity of their injury and broader treatment. In making sense of the trial they felt they could not understand the interventions without experience of them but if they received NPWT they developed a strong technological preference for this intervention. Conclusions Patients prefer to be involved in studies within the limits of their capacity, despite not being able to provide informed consent. A variety of sources of knowledge may enable participants to feel that they have a better understanding of the interventions. Professional staff need to be aware of the situated nature of decision making where participants invest their hopes for recovery in the team

Physiotherapy rehabilitation for osteoporotic vertebral fracture (PROVE) : study protocol for a randomised controlled trial

Background: Osteoporosis and vertebral fracture can have a considerable impact on an individual’s quality of life. There is increasing evidence that physiotherapy including manual techniques and exercise interventions may have an important treatment role. This pragmatic randomised controlled trial will investigate the clinical and cost-effectiveness of two different physiotherapy approaches for people with osteoporosis and vertebral fracture, in comparison to usual care. Methods/Design: Six hundred people with osteoporosis and a clinically diagnosed vertebral fracture will be recruited and randomly allocated to one of three management strategies, usual care (control - A), an exercise-based physiotherapy intervention (B) or a manual therapy-based physiotherapy intervention (C). Those in the usual care arm will receive a single session of education and advice, those in the active treatment arms (B + C) will be offered seven individual physiotherapy sessions over 12 weeks. The trial is designed as a prospective, adaptive single-blinded randomised controlled trial. An interim analysis will be completed and if one intervention is clearly superior the trial will be adapted at this point to continue with just one intervention and the control. The primary outcomes are quality of life measured by the disease specific QUALLEFO 41 and the Timed Loaded Standing test measured at 1 year. Discussion: There are a variety of different physiotherapy packages used to treat patients with osteoporotic vertebral fracture. At present, the indication for each different therapy is not well defined, and the effectiveness of different modalities is unknown

Group treatments for sensitive health care problems : a randomised controlled trial of group versus individual physiotherapy sessions for female urinary incontinence

Background: The aim was to compare effectiveness of group versus individual sessions of physiotherapy in terms of symptoms, quality of life, and costs, and to investigate the effect of patient preference on uptake and outcome of treatment. Methods: A pragmatic, multi-centre randomised controlled trial in five British National Health Service physiotherapy departments. 174 women with stress and/or urge incontinence were randomised to receive treatment from a physiotherapist delivered in a group or individual setting over three weekly sessions. Outcome were measured as Symptom Severity Index; Incontinence-related Quality of Life questionnaire; National Health Service costs, and out of pocket expenses. Results: The majority of women expressed no preference (55%) or preference for individual treatment (36%). Treatment attendance was good, with similar attendance with both service delivery models. Overall, there were no statistically significant differences in symptom severity or quality of life outcomes between the models. Over 85% of women reported a subjective benefit of treatment, with a slightly higher rating in the individual compared with the group setting. When all health care costs were considered, average cost per patient was lower for group sessions (Mean cost difference £52.91 95%, confidence interval (£25.82 - £80.00)). Conclusion: Indications are that whilst some women may have an initial preference for individual treatment, there are no substantial differences in the symptom, quality of life outcomes or non-attendance. Because of the significant difference in mean cost, group treatment is recommended