Search

Introduction: A significant paucity of literature exists relating to the impact on children of parental neurological disorder, with the exception of multiple sclerosis. The wider literature in this field (parental cancer, depression, alcoholism, HIV/AIDS) exhibits the many potential challenges young people might experience during serious parental illness. Given this, a literature review of parental neurological disorder is long overdue. Methods: This review is structured around the World Health Organisation (WHO) classification of neurological disorders. The WHO identifies ten common neurological disorders; dementia, epilepsy, headache, multiple sclerosis, neuroinfections, neurological disorders associated with malnutrition, pain associated with neurological disorders, Parkinson’s disease, stroke, and traumatic brain injury. A comprehensive search of the MEDLINE database was performed using key terms for each of the ten conditions. Results for each condition were divided in to ‘negative’, ‘positive and/or neutral’ and ‘other’ child responses. Results: The search yielded a total of 6247 titles, of which 184 underwent a full-text assessment. Sixty-five met all eligibility criteria and were thus included in the review. A number of negative issues emerged across parental conditions including the prevalence of child mood disorders, parent-child role reversal, children’s need for information on the parental condition, the importance of family cohesion, the negative effect of parental psychopathology and differences between male and female children. A limited number of positive outcomes were evident in a minority of parental conditions. Outcomes measured and methodologies employed were highly heterogeneous. Conclusion: Children generally respond negatively to parental neurological disorder. Responses varied between neurological disorders, suggesting the need for parental disease-specific guidance and clinical management where required.

Importance: Many people with Parkinson disease (PD) develop motor complications that are uncontrolled by levodopa dose adjustment. Among these patients, it is uncertain which drug class is more effective as adjuvant therapy. Objective: To compare the long-term effects on patient-rated quality of life of adding a dopamine agonist vs a dopamine reuptake inhibitor (DRI), either a monoamine oxidase type B (MAO-B) inhibitor or a catechol-O-methyltransferase (COMT) inhibitor, to levodopa therapy for the treatment of patients with motor complications of PD. Design, Setting, and Participants: This pragmatic semifactorial (2 × 1) randomized clinical trial recruited from 64 neurology and geriatric clinics (62 in the United Kingdom, 1 in the Czech Republic, and 1 in Russia) between February 23, 2001, and December 15, 2009. A total of 500 patients with idiopathic PD who developed uncontrolled motor complications and did not have dementia were randomly assigned on a 1:1:1 basis using a computerized minimization program. Data were analyzed between 2017 and 2020. Interventions: Open-label dopamine agonist, MAO-B inhibitor, or COMT inhibitor. Main Outcomes and Measures: Primary outcomes were scores on the 39-item Parkinson's Disease Questionnaire (PDQ-39) mobility domain and cost-effectiveness. Outcomes were assessed before study entry, at 6 and 12 months after randomization, and annually thereafter. Repeated-measures and log rank analyses were used in an intention-to-treat approach. Results: Among 500 participants, the mean (SD) age was 73.0 (8.2) years; 314 participants (62.8%) were men. Over a median of 4.5 years (range, 0-13.3 years) of follow-up, the participants in the dopamine agonist group had a mean PDQ-39 mobility score that was 2.4 points (95% CI, -1.3 to 6.0 points) better than that of the combined MAO-B and COMT groups; however, this difference was not significant (P=.20). With regard to DRIs, participants in the MAO-B group had mean PDQ-39 mobility scores that were 4.2 points (95% CI, 0.4-7.9 points; P=.03) better than those of the COMT group and EuroQol 5-dimension 3-level (EQ-5D-3L) utility scores that were 0.05 points (95% CI, 0.003-0.09 points; P=.04) better than the COMT group. Nonsignificant improvements were found in the PDQ-39 summary index (mean difference, 2.2 points; 95% CI, -0.2 to 4.5 points; P=.07) along with nonsignificant reductions in dementia (rate ratio [RR], 0.70; 95% CI, 0.47-1.03; P =.07) and mortality (RR, 0.76; 95% CI, 0.56-1.03; P=.07). When dopamine agonists were compared with MAO-B inhibitors only, the outcomes were similar. Conclusions and Relevance: In this study, patient-rated quality of life was inferior when COMT inhibitors were used as adjuvant treatment compared with MAO-B inhibitors or dopamine agonists among people with PD who experienced motor complications that were uncontrolled by levodopa therapy. The MAO-B inhibitors produced equivalent disease control, suggesting that these agents may be underused as adjuvant therapy. Trial Registration: isrctn.org Identifier: ISRCTN69812316; EU Clinical Trials Register Identifier: 2005-001813-16.

Additional file 1. Carers’ Experience of Assistive Technology use in dementia - Participant Information Sheet.

Additional file 3. Carers’ Experience of Assistive Technology use in dementia – Explanatory interviews: Interview topic guide.

Additional file 2. Carers’ Experience of Assistive Technology use in dementia - Participant Consent Form.

Background: \ud The PD MED study reported small but persistent benefits in patient‐rated mobility scores and quality of life from initiating therapy with levodopa compared with levodopa‐sparing therapies in early Parkinson's disease (PD).\ud \ud Objectives: \ud The objective was to estimate the cost‐effectiveness of levodopa‐sparing therapy (dopamine agonists or monoamine oxidase type B inhibitors compared with levodopa alone.\ud \ud Methods: \ud PD MED is a pragmatic, open‐label randomized, controlled trial in which patients newly diagnosed with PD were randomly assigned between levodopa‐sparing therapy (dopamine agonists or monoamine oxidase type B inhibitors ) and levodopa alone. Mean quality‐adjusted life‐years and costs were calculated for each participant. Differences in mean quality‐adjusted life‐years and costs between levodopa and levodopa‐sparing therapies and between dopamine agonists and monoamine oxidase type B inhibitors were estimated using linear regression.\ud \ud Results: \ud Over a mean observation period of 4 years, levodopa was associated with significantly higher quality‐adjusted life‐years (difference, 0.18; 95% CI, 0.05–0.30; P

Supplemental material, sj-docx-3-chi-10.1177_17423953211060253 for Burden of cancer trial participation: A qualitative sub-study of the INTERIM feasibility RCT by Chidiebere Nwolise, Pippa Corrie, Ray Fitzpatrick, Avinash Gupta, Crispin Jenkinson, Mark Middleton and Rubeta Matin in Chronic Illness

Supplemental material, sj-docx-3-chi-10.1177_17423953211060253 for Burden of cancer trial participation: A qualitative sub-study of the INTERIM feasibility RCT by Chidiebere Nwolise, Pippa Corrie, Ray Fitzpatrick, Avinash Gupta, Crispin Jenkinson, Mark Middleton and Rubeta Matin in Chronic Illness

Additional file 1.

Supplemental material, sj-docx-1-chi-10.1177_17423953211060253 for Burden of cancer trial participation: A qualitative sub-study of the INTERIM feasibility RCT by Chidiebere Nwolise, Pippa Corrie, Ray Fitzpatrick, Avinash Gupta, Crispin Jenkinson, Mark Middleton and Rubeta Matin in Chronic Illness

Supplemental material, sj-docx-2-chi-10.1177_17423953211060253 for Burden of cancer trial participation: A qualitative sub-study of the INTERIM feasibility RCT by Chidiebere Nwolise, Pippa Corrie, Ray Fitzpatrick, Avinash Gupta, Crispin Jenkinson, Mark Middleton and Rubeta Matin in Chronic Illness

Lilian Hartman,1 Crispin Jenkinson,2 David Morley2 1Lincoln College, University of Oxford, Oxford OX1 3DR, UK; 2Nuffield Department of Population Health, University of Oxford, Oxford OX3 7LF, UKCorrespondence: David Morley Tel +44 1865 289432Email david.morley@ndph.ox.ac.ukIntroduction: A significant paucity of literature exists relating to the impact on children of parental neurological disorder, with the exception of multiple sclerosis. The wider literature in this field (parental cancer, depression, alcoholism, HIV/AIDS) exhibits the many potential challenges young people might experience during serious parental illness. Given this, a literature review of parental neurological disorder is long overdue.Methods: This review is structured around the World Health Organisation (WHO) classification of neurological disorders. The WHO identifies 10 common neurological disorders; dementia, epilepsy, headache, multiple sclerosis, neuroinfections, neurological disorders associated with malnutrition, pain associated with neurological disorders, Parkinson’s disease, stroke, and traumatic brain injury. A comprehensive search of the MEDLINE database was performed using key terms for each of the 10 conditions. Results for each condition were divided in to “negative”, “positive and/or neutral” and “other” child responses.Results: The search yielded a total of 6247 titles, of which 184 underwent a full-text assessment. Sixty-five met all eligibility criteria and were thus included in the review. A number of negative issues emerged across parental conditions including the prevalence of child mood disorders, parent-child role reversal, children’s need for information on the parental condition, the importance of family cohesion, the negative effect of parental psychopathology and differences between male and female children. A limited number of positive outcomes were evident in a minority of parental conditions. Outcomes measured and methodologies employed were highly heterogeneous.Conclusion: Children generally respond negatively to parental neurological disorder. Responses varied between neurological disorders, suggesting the need for parental disease-specific guidance and clinical management where required.Keywords: parental illness, neurological disorder, young people, children, parent, World Health Organisation, outcomes, review

Background Missing data can introduce bias in the results of randomised controlled trials (RCTs), but are typically unavoidable in pragmatic clinical research, especially when patient reported outcome measures (PROMs) are used. Traditionally applied to the composite PROMs score of multi-item instruments, some recent research suggests that multiple imputation (MI) at the item level may be preferable under certain scenarios. This paper presents practical guidance on the choice of MI models for handling missing PROMs data based on the characteristics of the trial dataset. The comparative performance of complete cases analysis, which is commonly used in the analysis of RCTs, is also considered. Methods Realistic missing at random data were simulated using follow-up data from an RCT considering three different PROMs (Oxford Knee Score (OKS), EuroQoL 5 Dimensions 3 Levels (EQ-5D-3L), 12-item Short Form Survey (SF-12)). Data were multiply imputed at the item (using ordinal logit and predicted mean matching models), sub-scale and score level; unadjusted mean outcomes, as well as treatment effects from linear regression models were obtained for 1000 simulations. Performance was assessed by root mean square errors (RMSE) and mean absolute errors (MAE). Results Convergence problems were observed for MI at the item level. Performance generally improved with increasing sample sizes and lower percentages of missing data. Imputation at the score and subscale level outperformed imputation at the item level in small sample sizes (n ≤ 200). Imputation at the item level is more accurate for high proportions of item-nonresponse. All methods provided similar results for large sample sizes (≥500) in this particular case study. Conclusions Many factors, including the prevalence of missing data in the study, sample size, the number of items within the PROM and numbers of levels within the individual items, and planned analyses need consideration when choosing an imputation model for missing PROMs data. Electronic supplementary material The online version of this article (10.1186/s12874-018-0542-6) contains supplementary material, which is available to authorized users.

MEDLINE (OVID) search strategy for this review. (DOCX 13 kb)

Data extraction forms. (DOCX 15 kb)

Table of inclusion and exclusion criteria for this review. (DOCX 15 kb)

Search strategy. (DOCX 18 kb)

Data from included studies. (DOCX 114 kb)

PRISMA-P checklist. (DOCX 35 kb)

Data extraction form for this review. (DOCX 15 kb)

PRISMA checklist. (DOCX 27 kb)

Table of contents S1 Using computerized adaptive testing Tim Croudace S2 Well-being: what is it, how does it compare to health and what are the implications of using it to inform health policy John Brazier O1 “Am I going to get better?”—Using PROMs to inform patients about the likely benefit of surgery Nils Gutacker, Andrew Street O2 Identifying Patient Reported Outcome Measures for an electronic Personal Health Record Dan Robotham, Samantha Waterman, Diana Rose, Safarina Satkunanathan, Til Wykes O3 Examining the change process over time qualitatively: transformative learning and response shift Nasrin Nasr, Pamela Enderby O4 Developing a PROM to evaluate self-management in diabetes (HASMID): giving patients a voice Jill Carlton, Donna Rowen, Jackie Elliott, John Brazier, Katherine Stevens, Hasan Basarir, Alex Labeit O5 Development of the Primary Care Outcomes Questionnaire (PCOQ) Mairead Murphy, Sandra Hollinghurst, Chris Salisbury O6 Developing the PKEX score- a multimodal assessment tool for patients with shoulder problems Dominic Marley, James Wilson, Amy Barrat, Bibhas Roy O7 Applying multiple imputation to multi-item patient reported outcome measures: advantages and disadvantages of imputing at the item, sub-scale or score level Ines Rombach, Órlaith Burke, Crispin Jenkinson, Alastair Gray, Oliver Rivero-Arias O8 Integrating Patient Reported Outcome Measures (PROMs) into routine primary care for patients with multimorbidity: a feasibility study Ian Porter, Jaheeda Gangannagaripalli, Charlotte Bramwell, Jose M. Valderas O9 eRAPID: electronic self-report and management of adverse-events for pelvic radiotherapy (RT) patients Patricia Holch, Susan Davidson, Jacki Routledge, Ann Henry, Kevin Franks, Alex Gilbert, Kate Absolom & Galina Velikova O10 Patient reported outcomes (PROMs) based recommendation in clinical guidance for the management of chronic conditions in the United Kingdom Ian Porter, Jose M.Valderas O11 Cross-sectional and longitudinal parameter shifts in epidemiological data: measurement invariance and response shifts in cohort and survey data describing the UK’s Quality of Life Jan R. Boehnke O12 Patient-reported outcomes within health technology decision making: current status and implications for future policy Andrew Trigg, Ruth Howells O13 Can social care needs and well-being be explained by the EQ-5D? Analysis of Health Survey for England dataset Jeshika Singh, Subhash Pokhrel, Louise Longworth O14 Where patients and policy meet: exploring individual-level use of the Long-Term Conditions Questionnaire (LTCQ) Caroline Potter, Cheryl Hunter, Laura Kelly, Elizabeth Gibbons, Julian Forder, Angela Coulter, Ray Fitzpatrick, Michele Peters

Background A better understanding of coeliac disease can be achieved by assessing health‐related quality of life alongside clinical factors. Existing patient‐reported outcome measures (PROMs) evaluating quality of life in coeliac disease have not been developed in accordance with the US Food and Drug Administration guidelines. Aim To develop a PROM in accordance with best practice guidelines, capturing all aspects of quality of life important to adults with coeliac disease. Methods Candidate items for the Coeliac Disease Assessment Questionnaire (CDAQ) were refined through item appraisal, expert review, cognitive interviews, and a translatability assessment. A cross‐sectional survey determined further item reduction and the CDAQ's structure. The final CDAQ was administered alongside the Short Form Health Survey Version 2 (SF‐36v2) in a second survey to assess construct validity and test‐retest reliability. Results Pre‐testing the 64 candidate items revealed a range of issues which guided their refinement and reduction, resulting in the final CDAQ with 32 items representing 5 subscales: stigma (eight items), dietary burden (eight items), symptoms (five items), social isolation (five items), and worries and concerns (six items). Cronbach's alpha ranged between 0.82 and 0.88 for all domains. Further results showed CDAQ scores were more strongly correlated with the SF‐36v2's mental health dimensions, as expected. Intraclass correlation coefficients ranged from 0.79 to 0.89. Conclusions The CDAQ is a reliable and valid coeliac‐specific measure that captures all aspects of quality of life important to adults with coeliac disease. Further work is underway to assess the CDAQ's responsiveness to change.

Background: The prevalence of type 2 diabetes is rising, placing increasing strain on health care services. Web-based and mobile technologies can be an important source of information and support for people with type 2 diabetes and may prove beneficial with respect to reducing complications due to mismanagement. To date, little research has been performed to gain an insight into people’s perspectives of using such technologies in their daily management. Objective: The purpose of this study was to understand the impact of using Web-based and mobile technologies to support the management of type 2 diabetes. Methods: In-depth interviews were conducted with 15 people with type 2 diabetes to explore experiences of using Web-based and mobile technologies to manage their diabetes. Transcripts were analyzed using the framework method. Results: Technology supported the users to maintain individualized and tailored goals when managing their health. A total of 7 themes were identified as important to participants when using technology to support self-management: (1) information, (2) understanding individual health and personal data, (3) reaching and sustaining goals, (4) minimizing disruption to daily life, (5) reassurance, (6) communicating with health care professionals, and (7) coordinated care. Conclusions: Patients need to be supported to manage their condition to improve well-being and prevent diabetes-related complications from arising. Technologies enabled the users to get an in-depth sense of how their body reacted to both lifestyle and medication factors—something that was much more difficult with the use of traditional standardized information alone. It is intended that the results of this study will inform a new questionnaire designed to assess self-management in people using Web-based and mobile technology to manage their health.

Supplementary material for Multiple imputation for patient reported outcome measures in randomised controlled trials: advantages and disadvantages of imputing at the item, subscale or composite score level. Table S1. Overview of correlations between OKS outcome variables and covariates used in imputation models. Table S2. Overview of correlations between EQ-5D-3L outcome variables and covariates used in imputation models. Table S3. Overview of correlations between SF-12 outcome variables and covariates used in imputation models. Figures S1-S9. Supplementary graphs to Figs. 3, 4, 5, 6, 7, 8, 9, 10, 11 and 12 in the manuscript, showing mean absolute errors instead of root mean square errors. (DOCX 1613 kb)

The proceedings contain 36 papers. The topics discussed include: using patient reported outcome measures (PROMs) in cancer care; validation of the long-term conditions questionnaire (LTCQ) in a diverse sample of health and social care users in England; the national institutes of health patient-reported outcomes measurement information system (PROMIS): a view from the UK; constructing and validating the short recovering quality of life (ReQoL) measure for use in a mental health population; developing preference based measures for diabetes for calculating QALYs: DHP-3D and DHP-5D; development of a novel patient reported outcome measure for patients with haematological malignancy: a qualitative study; and examining the relevance of PROMs to patients: a review of qualitative data capturing which HRQoL domains are important to patients.

Floor and ceiling effects for OxCAP-MH items scored on a 1 to 5 Likert scale. (DOCX 13Â kb)

Correlation of individual items of the OxCAP-MH with established measures of illness severity, functioning and social outcomes. (DOCX 13Â kb)

Background: The Stroke Impact Scale (SIS) is a stroke-specific, quality of life measure recommended for research and clinical practice. Completion rates are suboptimal and could relate to test burden. We derived and validated a short form-SIS.\ud Methods and Results: We examined data from the Virtual International Stroke Trial Archive, generating derivation and validation populations. We derived a short form (SF-SIS) by selecting one item per domain of SIS, choosing items most highly correlated with total domain score. Our validation described agreement of SF-SIS with original SIS and the SIS-16, and correlation with Barthel Index, modified Rankin Scale, NIHSS, and EQ-5D visual analogue scales. We assessed discriminative validity, (associations between SF-SIS and factors known to influence outcome [age, physiological parameters and comorbidity]). We assessed face validity and acceptability by sharing the SF-SIS with a focus group of stroke survivors and multidisciplinary stroke healthcare staff. \ud From 5549 acute study patients (mean age: 68.5 (SD:13) years; mean SIS :64 [SD:32]) and 332 rehabilitation patients (mean age 65.7 [SD:11]; mean SIS:61 [SD:11]), we derived an 8-item SF-SIS that demonstrated good agreement with original SIS and good correlation with our chosen functional and QOL measures (all rho>0.70; p

The identification of suitable outcome measures will improve the evaluation of integrated NHS care for the large number of children affected by neurodisability, and has the potential to encourage the provision of more appropriate and effective health care. This research sought to appraise the potential of patient-reported outcome measures (PROMs) for children and young people with neurodisability. This research aimed (i) to identify key outcomes of health care for children with neurodisability, beyond morbidity and mortality, from the perspectives of children, parents and professionals; (ii) to critically appraise existing generic multidimensional PROMs; and (iii) to examine whether or not the key outcomes might be measured by existing PROMs. We also sought agreement on a definition of neurodisability. Data were gathered in three main ways, (i) a systematic review identified eligible generic multidimensional PROMs and peer-reviewed studies evaluating psychometric performance using English-language questionnaires. Studies were appraised for methodological quality and psychometric performance was appraised using standard criteria. (ii) Focus groups and interviews with children and young people with neurodisability, and separately with parents, sought to identify important outcomes of NHS care, and their feedback on example PROM questionnaires. (iii) An online Delphi survey was conducted with a multidisciplinary sample of health professionals to seek agreement on appropriate NHS outcomes. In addition, we convened a consensus meeting with a small nominal group of young people, parents and professionals; the group sought agreement on a core set of important health outcomes. From the systematic review, we identified 126 papers that reported eligible evidence regarding the psychometric performance of 25 PROMs. Evidence of psychometric robustness was more favourable for a small number of PROMs: KIDSCREEN (generic), DISABKIDS (chronic-generic) and Child Health Utility 9D (preference-based measure). The Pediatric Quality of Life Inventory and KINDL offer both self-report and a proxy report version for a range of age bands, but evidence of their psychometric performance was weaker. Evidence was lacking in one or more respects for all candidate PROMs, in both general populations and those with neurodisability. Proxy reporting was found generally to be poorly correlated with self-report. Focus groups and interviews included 54 children and young people, and 53 parents. The more important health outcomes were felt to be communication, emotional well-being, pain, mobility, independence/self-care, worry/mental health, social activities and sleep. In addition, parents of children with intellectual impairment identified behaviour, toileting and safety as important outcomes. Participants suggested problems with the face validity of example PROM questionnaires for measuring NHS care. In the Delphi survey, 276 clinicians from a wide range of professions contributed to at least one of four rounds. Professionals rated pain, hearing, seeing, sleep, toileting, mobility and communication as key goals for the NHS but also identified treating neurological symptoms as important. Professionals in the Delphi survey and parents working with the research team agreed a proposed definition for neurodisability. The consensus meeting confirmed overlap between the outcomes identified as important by young people, parents and professionals, but not complete agreement. There was agreement between young people, parents and professionals regarding a core suite of more important health outcomes: communication, emotional well-being, pain, mobility, independence/self-care, worry/mental health, social activities and sleep. In addition, behaviour, toileting and safety were identified as important by parents. This research suggests that it would be appropriate to measure these constructs using PROMs to assess health care. None of the candidate PROMs in the review adequately captures all of the identified constructs, and there is inadequate evidence that candidate PROMs are psychometrically robust for use across children with neurodisability. Further consultation with young people, families and professionals is warranted to support the use of PROMs to measure NHS outcomes. Research to test potential PROMs with different age groups and conditions would be valuable. The National Institute for Health Research Health Services and Delivery Research programme.

Background: Long-term conditions have a significant impact on individuals, their families, and the health service. As people with these conditions represent a high proportion of hospital admissions, investigating their experiences of inpatient care has become an important area of investigation. We conducted a secondary analysis of the NHS adult inpatient survey for England to compare the hospital experiences of three groups of patients: those without long-term conditions, those with a single long-term condition, and those with multiple long-term conditions. We were particularly interested in the extent to which these patients received self-management support from hospital staff, so we developed a brief summary tool drawn from salient questions in the survey to aid the comparison. Methods. Analysis of data from the 2011 national adult inpatient survey (n = 65,134) to compare the experiences of three groups of patients: those with no limiting long-term conditions (No-LLTC), those with one limiting long-term condition (S-LLTC), and those with two or more limiting long-term conditions (M-LLTC). The main outcome measure was patients' self-reports of their experience of inpatient care, including staff-patient interactions, information provision, involvement in decisions and support for self-care and overall ratings of care. A short form scale, the Oxford Patient Involvement and Experience scale (OxPIE) was developed from the adult inpatient survey and used to compare the groups using logistic regression. Results: There were significant differences between the No-LLTC group in comparison to both the S-LLTC and M-LLTC groups. Patients with limiting long-term conditions reported significantly worse hospital experiences than those without, as measured by OxPIE: S-LLTC odds ratio = 1.23, 95% CI 1.03-1.48; M-LLTC odds ratio = 1.64, 95% CI 1.19 - 2.26. Responses to a single global rating question were more positive but not strongly correlated with OxPIE. Conclusions: Patients with LLTCs were more critical of their inpatient care than those with no LLTCs. Those with more than one long-term condition reported worse experiences than those with a single limiting condition. Simple rating questions may not be sufficiently sensitive to reflect important aspects of patients' experience. © 2014 Hewitson et al.; licensee BioMed Central Ltd.

Background and purpose A questionnaire was introduced by the New Zealand Arthroplasty Registry for use when evaluating the outcome of total ankle replacement surgery. We evaluated the reliability, validity, and responsiveness of the modified Swedish version of the questionnaire (SEFAS) in patients with osteoarthritis or inflammatory arthritis before and/or after their ankle was replaced or fused. Patients and methods The questionnaire was translated into Swedish and cross-culturally adapted according to a standardized procedure. It was sent to 135 patients with ankle arthritis who were scheduled for or had undergone surgery, together with the foot and ankle outcome score (FAOS), the short form 36 (SF-36) score, and the EuroQol (EQ-5D) score. Construct validity was evaluated with Spearman’s correlation coefficient when comparing SEFAS with FAOS, SF-36, and EQ-5D, content validity by calculating floor and ceiling effects, test-retest reliability with intraclass correlation coefficient (ICC), internal consistency with Cronbach’s alpha (n = 62), agreement by Bland-Altman plot, and responsiveness by effect size and standardized response mean (n = 37). Results For construct validity, we correlated SEFAS with the other scores and 70% or more of our predefined hypotheses concerning correlations could be confirmed. There were no floor or ceiling effects. ICC was 0.92 (CI 95%: 0.88–0.95), Cronbach’s alpha 0.96, effect size was 1.44, and the standardized response mean was 1.00. Interpretation SEFAS is a self-reported foot and ankle score with good validity, reliability and responsiveness, indicating that the score can be used to evaluate patients with osteoarthritis or inflammatory arthritis of the ankle and outcome of surgery.

This thesis is a report of a study on patients suffering one of two painful chronic illnesses (rheumatoid arthritis (RA) or migraine), and attempts to discover possible determinants of psychological reactions to long term painful illness. One hundred and sixty chronically ill individuals were interviewed, (80 migraine and 80 RA sufferers). In keeping with other evidence, the extent of psychological disturbance was found to be higher for chronically ill individuals than for general population samples. There were no zero order correlations between reported pain and psychological distress. Age and length of suffering have often been cited as possible factors influencing mood state, but no evidence was found for this in the data gained in this survey. The results provide no evidence for the use of coping strategies as a method of adapting to the demands of the painful chronic illnesses studied here. Beliefs in control over illness have been posited as possible factors that may influence psychological state, and were measured in this research using a health locus of control scale. However locus of control scores were not found to be associated with mood state, although, in keeping with other research, scores were found to be associated with age and social class, with both lower social class and older sufferers scoring higher on externality than those from higher social classes, or whom were younger. The strongest association was found between aspects of subjective health status and mood state. The major finding of this study is that patients assessment of their own health state, in both illnesses, is the major factor associated with psychological state.

Peer reviewed: True, Funder: National Institute for Health Research (NIHR) Research for Patient Benefit (RfPB); Grant(s): Grant Reference Number: PB-PG-0815-20048, OBJECTIVE: A qualitative sub-study was carried out within a larger phase II feasibility trial, to identify and describe the burden experienced by advanced melanoma patients participating in a clinical trial and the factors affecting their capacity to cope with the burden. METHODS: Semi-structured interviews were conducted with fourteen patients with advanced melanoma recruited from National Health Service hospitals in the United Kingdom. Qualitative analysis was undertaken using a framework analysis approach. Normalisation process theory was applied to the concept of research participation burden in order to interpret and categorise findings. RESULTS: Burdens of participation were identified as arising from making sense of the trial and treatment; arranging transport, appointment and prescriptions; enacting management strategies and enduring side effects; reflecting on trial documents and treatment efficacy, and emotional and mental effects of randomisation and treatment side effects. Factors reported as influencing capacity include personal attributes and skills, physical and cognitive abilities and support network. DISCUSSION: This is the first study to highlight the substantial burden faced by patients with advanced melanoma in a clinical trial and factors that may lessen or worsen the burden. Consideration of identified burdens during trial design and execution will reduce the burden experienced by research participants.