Patient-reported outcome measures in older people with hip fracture: a systematic review of quality and acceptability.

PURPOSE: Hip fracture is the most common serious injury of older people, often resulting in reduced mobility and loss of independence. However, guidance for the use of patient-reported outcome measures (PROMs) does not exist: we describe the first review to apply internationally endorsed criteria in support of PROM quality and acceptability in this group, and make recommendations for future applications. METHODS: Systematic literature searches of major databases (1980-2015) to identify published evidence of the application and quality of clearly defined measures. Evidence of measurement and practical properties, and the extent of active patient involvement, was sought. Study and PROM quality was assessed against recommended criteria. RESULTS: Seventy-one articles relating to 28 PROMs (Generic n = 12; Specific n = 16) were included. The SF-36 (v1) and EuroQoL EQ-5D 3L were the most widely evaluated measures with acceptable evidence of measurement properties, but limited evaluations of practical properties or relevance to this group. Evidence was mostly limited for the remaining measures. Hypothesized associations between variables were infrequently evaluated. Evidence of data quality, test-retest reliability, responsiveness, interpretation, acceptability and feasibility was also limited. Active patient involvement in PROM development or evaluation was not reported. There was limited evaluation of proxy completions. CONCLUSIONS: The paucity of robust evaluations is disappointing and prevents clear recommendations for PROM-based assessment. Further research must urgently seek to identify which outcomes really matter to this group. Future PROM selection must be underpinned by research which focuses on methodological quality, including issues of acceptability, relevance, feasibility of application, and proxy completion, whilst seeking to actively incorporate the perspective of patients and their advocates.

A systematic review of the outcomes reported in cardiac arrest clinical trials: the need for a core outcome set.

BACKGROUND: There is a limited guidance for outcome reporting for cardiac arrest trials. This review was conducted to explore the degree of variation and identify trends in outcome reporting. METHODS: Randomised controlled trials enrolling patients with cardiac arrest (2002-2012) were identified by applying a search strategy to four databases. Titles, abstracts and short-listed studies were independently assessed for eligibility. Data on the primary and secondary outcome measures, details of outcome reporting and reproducibility were extracted. RESULTS: 61 studies matched the inclusion criteria. There was wide variation in the focus, method and timing of assessment. Outcomes most commonly reported across studies were: survival (85.2%), activities (52.5%), body structure or function (41.0%), and processes of care (26.2%). Over 160 individual outcomes were reported including 39 different reports of survival measures of which 11 were measurements of ROSC (return of spontaneous circulation). Twenty different assessments of activity limitation were reported; only one was patient-reported. Many assessments were poorly defined or non-reproducible. The majority of outcomes were assessed up to hospital discharge (89.3%). There was no one outcome measure that was assessed across all trials. CONCLUSIONS: Outcome reporting in cardiac arrest RCTs lacks consistency and transparency. Guidance for improved outcome reporting is urgently required to reduce this heterogeneity in reporting, improve the quality of assessment in clinical trials, and to support the synthesis of trial data. The results highlight the importance of working towards a core outcome set for cardiac arrest clinical trials to maximise the utility of future research.

Developing a core outcome set for hip fracture trials.

The lack of a consensus for core health outcomes that should be reported in clinical research has hampered study design and evidence synthesis. We report a United Kingdom consensus for a core outcome set (COS) for clinical trials of patients with a hip fracture. We adopted a modified nominal group technique to derive consensus on 1) which outcome domains should be measured, and 2) methods of assessment. Participants reflected a diversity of perspectives and experience. They received an evidence synthesis and postal questionnaire in advance of the consensus meeting, and ranked the importance of candidate domains and the relevance and suitability of short-listed measures. During the meeting, pre-meeting source data and questionnaire responses were summarised, followed by facilitated discussion and a final plenary session. A COS was determined using a closed voting system: a 70% consensus was required. Consensus supported a five-domain COS: mortality, pain, activities of daily living, mobility, and health-related quality of life (HRQL). Single-item measures of mortality and mobility (indoor/outdoor walking status) and a generic multi-item measure of HRQL - the EuroQoL EQ-5D - were recommended. These measures should be included as a minimum in all hip fracture trials. Other outcome measures should be added depending on the particular interventions being studied. Cite this article:

Assessing severity of illness and outcomes of treatment in children with Chronic Fatigue Syndrome/Myalgic Encephalomyelitis (CFS/ME): a systematic review of patient-reported outcome measures (PROMs).

Chronic Fatigue Syndrome or Myalgic Encephalomyelitis (CFS/ME) in children is characterized by persistent or recurrent debilitating fatigue which results in a substantial reduction in activity. There is a growing interest in the use of questionnaires, or patient-reported outcome measures (PROMs), to assess how patients function and feel in relation to their health and associated healthcare. However, guidance for PROM selection for children with CFS/ME does not exist. We reviewed the quality and acceptability of PROMs used with children with CFS/ME to inform recommendations for practice. We conducted a systematic review of PROMs completed by children with CFS/ME. The quality of the evaluative studies and the reviewed measures were assessed against recommended criteria using an appraisal framework and the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) checklist. We sought evidence of measurement (reliability, validity, responsiveness, interpretability, data quality) and practical properties (acceptability, relevance, feasibility). Sixteen articles were included in the review, providing evidence of reliability and/or validity for 13 PROMs. Of these, five were child-specific (one health-related quality-of-life; four emotional well-being) and eight were not (four emotional well-being, three fatigue-specific; and one generic). All measures had limited evidence of measurement properties and no evidence of practical properties. Recommendations for patient-reported assessment are difficult to make because of limited evidence of the quality and acceptability of PROMs for children with CFS/ME. The appraisal method highlighted significant methodological and quality issues which must be addressed in future research. There is a lack of qualitative evidence describing the outcomes of healthcare that are important to children with CFS/ME, and the relevance or appropriateness of available measures. Future PROM development and evaluation in this group must seek to involve children collaboratively to ensure that the outcomes that children care about are assessed in an acceptable way.

Patients with well-established ankylosing spondylitis show limited deterioration in a ten-year prospective cohort study.

This study aims to describe the deterioration of well-established ankylosing spondylitis (AS) over a 10-year period. Patients with well-established AS under secondary care review were assessed at baseline and re-assessed after a 10-year period. Data on patient characteristics, spinal mobility and self-reported health status (AS Quality of Life, Bath AS Disease Activity Index, EuroQoL, Revised Leeds Disability Questionnaire and Short Form-12) were compared between the two time points. One hundred fifty-nine patients took part in the initial assessment. After 10 years, 69/117 patients still under secondary care follow-up attended a second assessment. At the second assessment, this cohort was predominately male (92.8 %), mean age of 48.7 years (SD 9.7) and mean disease and symptom duration of 14.9 (SD 9.0) and 21.9 years (SD 13.3), respectively. Amongst the physical and patient-reported measures, only tragus to wall (p < 0.001), cervical rotation (p = 0.001) and disability (p = 0.02) had significantly deteriorated over time. The percentage of patients who had withdrawn from the workforce before normal retirement age increased from 37 to 53 % over the 10-year period. Many of the measures used in the assessment of AS do not generally deteriorate over time in those with well-established disease. This suggests that deterioration may plateau in established disease. However, AS has a long-term social impact with levels of employment in this cohort 30 % lower than the general population. Interventions directed at preventing deterioration may be more effective earlier in their disease course, before this plateau is reached.

Evaluation of a modified arthritis self-efficacy scale for an ankylosing spondylitis UK population.

OBJECTIVES: To evaluate an Ankylosing Spondylitis-specific Arthritis Self-Efficacy Scale (ASES-AS) United Kingdom (UK) secondary care population. METHODS: The ASES-AS is based on the 8-item ASES with minor alterations in phraseology. Patients from ten secondary care rheumatology centres across England were asked to complete a postal questionnaire concerning sociodemographic and clinical characteristics: Bath AS Functional Index (BASFI), Bath AS Disease Activity Index (BASDAI), numerical pain rating scale (NRS), Hospital Anxiety and Depression Scale (HADS), Short Form 36 (SF-36), Evaluation of AS Quality of Life questionnaire (EASi-QoL) and ASES-AS. Respondents received repeat questionnaires at 2 weeks and 6 months including health transition questions assessing change in AS-specific and general health. The ASES-AS was assessed for data quality, reliability, validity, and responsiveness. RESULTS: Response rate was 64% (n=612), 72% (n=438) were male, mean age 50.8yrs (SD 12.2 yrs), mean disease duration 17.3 yrs (SD 11.7 yrs) and mean symptom duration 22.4 yrs (SD 12.4 yrs). Missing data for each item/total score range was 0.7%-3.1%. Item-total correlations range was 0.66 to 0.83. Cronbach's alpha was 0.93 and test-retest reliability (intraclass correlation coefficient) 0.77. A priori hypothesised associations between ASAS-AS and disease status measures were supported. Social variables potentially related to self-efficacy demonstrated evidence of convergent validity (employment p<0.001, educational level p<0.005). A Modified Standard Response Mean (MSRM) of 0.44 and 0.26 in AS-specific and general health respectively at 6 months indicates moderate responsiveness. CONCLUSIONS: ASES-AS has good evidence supporting its application as an AS-specific self-efficacy measure in research including clinical trials at a group level.

Impact of ankylosing spondylitis on work in patients across the UK.

OBJECTIVES: To examine the impact of ankylosing spondylitis (AS) on patients across the UK and to identify factors associated with unemployment, absenteeism, and presenteeism. METHODS: One thousand patients with AS from 10 specialist rheumatology centres across the UK were invited to participate in a study evaluating a new outcome measure. Patients completed a questionnaire, which included questions relating to their work, sociodemographic and clinical characteristics. RESULTS: The questionnaire was completed by 612 patients (438 males; 72%). The mean age of the participants was 50.8 (SD 12.2) years, mean disease duration was 17.3 (SD 11.7) years, and mean symptom duration 22.4 (SD 12.4) years. A total of 206 (40%) patients of working age were not employed. Factors associated with not being employed were social deprivation [odds ratio (OR) 3.52, 95% confidence interval (CI) 2.14-5.80], poor function (OR 3.42, 95% CI 1.90-6.13), depression (OR 2.05, 95% CI 1.12-3.78), increasing age (OR 1.05 per year, 95% CI 1.02-1.08), and longer disease duration (OR 1.03 per year, 95% CI 1.01-1.06). Disease activity (OR 3.24, 95% CI 1.11-9.48) and depression (OR 3.22, 95% CI 1.22-8.48) were associated with absenteeism, while depression (OR 5.69, 95% CI 1.77-18.27, disease activity (OR 3.97, 95% CI 1.76-8.98), anxiety (OR 3.90, 95% CI 1.83-8.31), self-efficacy (OR 0.71, 95% CI 0.58-0.86), and increasing age (OR 1.04 per year, 95% CI 1.00-1.08) were associated with presenteeism. CONCLUSION: Psychological, sociodemographic, and disease-related factors were all found to be related to work status. These factors should be taken into account when considering early treatment and management. Depression, in particular, appears to be associated with employment, absenteeism, and presenteeism, and should therefore be prioritized in clinical practice.

The direct healthcare costs associated with ankylosing spondylitis patients attending a UK secondary care rheumatology unit.

OBJECTIVES: To explore the direct healthcare resources associated with ankylosing spondylitis (AS) in the UK. A secondary objective was to establish if resources, and thus healthcare costs, vary by disease severity. METHODS: Medical records of 147 sequential AS patients attending a UK secondary care rheumatology unit were examined to assess the direct healthcare resources used over the previous 12 months. Starting with a detailed inventory and measurement of resources consumed, unit cost multipliers were applied to the quantity of each type of resource consumed. The mean cost per patient was estimated using the total cost divided by the number of patients included. RESULTS: The mean (median) annual cost per patient was 1852 pounds sterling (892 pounds sterling). The distribution of cost data was skewed, with 11% of patients incurring 50% of the total costs. The three most relevant cost domains were physiotherapy, hospitalization and medication costs at 32, 21 and 20% of the total costs, respectively. Twenty percent of the patients received physiotherapy, 13% received inpatient care and almost all incurred medication costs. Thirty-four percent of patients were prescribed disease-modifying anti-rheumatic drugs and 85% non-steroidal anti-inflammatory drugs. Over 50% of patients had at least one comorbidity. CONCLUSION: Direct costs accelerate steeply with disease activity (Bath Ankylosing Spondylitis Disease Activity Index >6.0) and increasing loss of function (Bath Ankylosing Spondylitis Functional Index >6.0) in patients with AS. The most severely affected patients incur 50% of the total costs, and physiotherapy accounts for 32% of the total healthcare costs in the UK.

Quality of life in older people: a structured review of generic self-assessed health instruments.

OBJECTIVE: To review evidence relating to the measurement properties of multi-item generic patient or self-assessed measures of health in older people. METHODS: Systematic literature searches to identify instruments. Pre-defined criteria relating to reliability, validity and responsiveness. RESULTS: 122 articles relating to 15 instruments met the inclusion criteria. The most extensive evidence was found for the SF-36, COOP Charts, EQ-5D, Nottingham Health Profile (NHP) and SIP. Four instruments have evidence of both internal consistency and test-retest reliability--NHP, SF-12, SF-20, SF-36. Four instruments lack evidence of reliability--HSQ-12, IHQL, QWB, SQL. Most instruments were assessed for validity through comparisons with other instruments, global judgements of health, or clinical and socio-demographic variables. Five instruments lack evidence of responsiveness--GQL, HSQ-12, IHQL, QLI and QWB. CONCLUSION: There is good evidence for reliability, validity and responsiveness for the SF-36, EQ-5D and NHP. There is more limited evidence for the COOP, SF-12 and SIP. The SF-36 is recommended where a detailed and broad ranging assessment of health is required, particularly in community dwelling older people with limited morbidity. The EQ-5D is recommended where a more succinct assessment is required, particularly where a substantial change in health is expected. Instrument content should be assessed for relevance before application. The concurrent evaluation of generic instruments in older people is recommended.

Patient-assessed health in ankylosing spondylitis: a structured review.

OBJECTIVE: To review evidence relating to the measurement properties for all disease-specific, multi-item, patient-assessed health instruments in patients with ankylosing spondylitis (AS). METHODS: Systematic literature searches were made to identify instruments, using predefined criteria relating to reliability, validity, responsiveness and precision. RESULTS: Twelve AS-specific and three arthritis-specific instruments met the inclusion criteria. Three AS-specific instruments that measure health-related quality of life (HRQL) were reviewed. The Bath Ankylosing Spondylitis Disease Activity Index (BASDAI), the Bath Ankylosing Spondylitis Functional Index (BASFI) and the Dougados Functional Index (DFI) had the greatest amount of evidence for reliability, validity and responsiveness across a range of settings. Four instruments lacked evidence for test-retest or internal consistency reliability. Most were assessed for validity through comparisons with other instruments, global judgements of health, mobility or clinical and sociodemographic variables. Most were assessed for responsiveness through mean score changes. Three instruments lacked evidence of responsiveness. CONCLUSION: This review provides a contribution to AS assessment. AS-specific multi-item measures specific to the assessment of pain, stiffness, fatigue and global health were not identified; where assessed, these domains were largely measured with single-item visual analogue scales. Single items may provide a limited reflection of these important domains. The BASFI and DFI remain the instruments of choice for functional assessment. HRQL is recommended as a core assessment domain. Further concurrent evaluation is recommended.

Reviewing measures of outcome: reliability of data extraction.

RATIONALE, AIMS AND OBJECTIVES: Although there is wide acceptance of the necessary domains for the evaluation of measures of health outcome, there has been little development or evaluation of methods of data extraction, such as checklists. This study aimed to assess the inter-rater reliability of data extraction from published articles using a new electronic database developed for structured reviews of health outcome measures. METHODS: Domains considered important in the evaluation of measures of health outcome provided the foundation for a checklist for data extraction using a new electronic database. The database utilized Microsoft Access 2000 software (Microsoft Corporation, 2000). Following training, three independent reviewers extracted data from seven selected articles using the electronic database, and assessed article and measure quality against pre-defined criteria. Data extraction was compared for consistency and item content. Inter-reviewer agreement for categorical data was assessed by multiple kappa correlation. RESULTS: Analysis demonstrated strong agreement between reviewers for all aspects of data extraction. However, lack of clarity in published articles affected the ability to clearly identify measures of outcome. CONCLUSIONS: The high level of inter-reviewer agreement supports the use of multiple trained reviewers in data extraction for reviews of measures of outcome using the checklist and Access software described. The electronic database supports standardized data extraction from published articles, benefiting from the combination of data extraction and data entry in a single step. Adaptation of the database to support structured reviews of measures of outcome adopted in the evaluation of other health states is proposed.

Multi-item outcome measures for lateral ligament injury of the ankle: a structured review.

OBJECTIVE: To identify and review evidence relating to the measurement properties of published multi-item outcome measures for the conservative management of lateral ligament injuries of the ankle. METHODS: Systematic literature searches were used to identify measures, which were then assessed against pre-defined criteria relating to development, item content, reliability, validity and responsiveness. RESULTS: Seven disease-specific measures of ankle status [Ankle Joint Functional Assessment Tool, Clinical Trauma Severity Score, Composite Inversion Injury Scale, Kaikkonen Functional Scale (KFS), Karlsson Ankle Function Score (KAFS), Olerud and Molander Ankle Score (OMAS), and the Point System] and two generic measures of health (McGill Pain Questionnaire, Sickness Impact Profile) met the review inclusion criteria. While all measures had been used in acute injuries, only two had also been applied during later stages of recovery (>6 months). The studies covered a comprehensive range of graded ligament injuries. Expert opinion dominated item generation for all measures. All measures lack evidence of test-retest or internal consistency reliability in patients with ankle sprain. Several measures were assessed for validity through comparison with other measures, but there was limited evidence of construct validity and no formal assessment of responsiveness for any measure. CONCLUSION: The disappointing lack of evidence for measurement properties suggests that any measure should be used with caution until appropriate evidence is provided. On the basis of limited evidence, the KFS offers the most promising approach to a combined clinician- and patient-assessment of ankle function, and the KAFS or OMAS if a patient-assessed evaluation of function is required.

Spinal mobility in ankylosing spondylitis: reliability, validity and responsiveness.

OBJECTIVE: To evaluate the measurement properties of an evidence-based selection of measures of spinal mobility in patients with ankylosing spondylitis (AS). METHODS: Measurements of spinal mobility were taken by trained observers within a UK rheumatology centre. Inter-observer reliability was assessed. Intra-observer reliability was assessed in patients reporting no change in AS-specific health at 2 weeks. Validity was assessed and scores were correlated with responses to health transition questions. Responsiveness was evaluated for patients reporting change in health at 6 months. RESULTS: Reliability estimates support the use of all measures in individual evaluation (intraclass correlation>0.90). Correlations between measures of spinal mobility were in the hypothesized direction; the largest was between the modified Schober index (15 cm) (MSI) and the other measures. As hypothesized, small to moderate levels of correlation were found between mobility measures and patient-assessed health status. There was no significant linear relationship between mobility measures and self-reported health transition. Fingertip-to-floor distance following trunk forward flexion (FFD) was the most responsive mobility measure but was not as responsive as two AS-specific patient-assessed instruments, the Ankylosing Spondylitis Quality of Life Questionnaire (ASQoL) and the Bath Ankylosing Spondylitis Disease Activity Index (BASDAI). The MSI and cervical rotation (Crot) also had evidence of responsiveness. Low levels of responsiveness were found for the remaining measures. CONCLUSION: All mobility measures had adequate levels of reliability and validity. The MSI had a strong relationship with all mobility measures, and the FFD and Crot were the most responsive to self-perceived changes in health at 6 months. The MSI, FFD and Crot are recommended for clinical practice and research.

Generic measures of health-related quality of life in ankylosing spondylitis: reliability, validity and responsiveness.

OBJECTIVE: To assess the acceptability and measurement properties of two generic measures of health-related quality of life (HRQL): the EuroQol and the Short Form 12-item Health Survey Questionnaire (SF-12) in ankylosing spondylitis (AS). METHODS: Instruments were administered by means of a self-completed questionnaire to AS patients recruited from across the United Kingdom. Instruments were assessed for data quality and scaling assumptions. Test-retest reliability was assessed in those patients reporting no change in general health at 2 weeks. The convergent validity of both instruments was assessed and scores were correlated with responses to health transition questions. Responsiveness was assessed for patients reporting change in health at 6 months. RESULTS: The instruments had high completion rates. Although slightly skewed towards better levels of health, scores covered the available range for both sections of the EuroQol [EQ-5D and EQ-visual analogue scale (EQ-VAS)]. Score distributions approximated normality for the SF-12. Test-retest reliability estimates support the use of both instruments in group evaluation and the SF-12 Physical Component Summary score (PCS) in individual evaluation (>0.90). Correlations between instruments were in the hypothesized direction and were of a moderate level. The EQ-VAS had the strongest linear relationship, with responses to both specific and general health transition questions (P<0.01). The EQ-VAS and SF-12 PCS were the most responsive instruments. The EQ-5D was the least responsive instrument. CONCLUSION: The instruments have undergone a comprehensive comparative evaluation to assess the measurement properties required for patient-assessed measures of health outcome in AS. Adequate levels of acceptability, reliability and validity were found for both instruments. Although evidence supporting instrument responsiveness was strong for the EQ-VAS and SF-12 PCS, it was very weak for the EQ-5D and SF-12 Mental Component Summary Scale (MCS). The EQ-VAS and SF-12 PCS can both be recommended for use in group evaluation, and the SF-12 PCS is recommended in routine practice or research. However, the lower reliability of the SF-12 MCS and the limited ability of both the EQ-5D and SF-12 MCS to detect change in health may restrict these roles.

Disease-specific, patient-assessed measures of health outcome in ankylosing spondylitis: reliability, validity and responsiveness.

OBJECTIVE: To assess the acceptability and measurement properties of four ankylosing spondylitis (AS)-specific, patient-assessed measures of health outcome: AS Quality of Life Questionnaire (ASQoL), Bath AS Disease Activity Index (BASDAI), the Body Chart and the Revised Leeds Disability Questionnaire (RLDQ). METHODS: Instruments were administered by means of a self-completed questionnaire to patients recruited from across the United Kingdom (UK). Instruments were assessed for data quality and scaling assumptions. Where appropriate, dimensionality was assessed using principle component analysis (PCA). Internal consistency reliability was tested using Cronbach's alpha. Test-retest reliability was assessed in those patients reporting no change in AS-specific health at 2 weeks. The convergent validity of the instruments was assessed and scores were correlated with responses to the health transition questions. Responsiveness was assessed for patients reporting change in health at 6 months. RESULTS: The BASDAI and Body Chart have low self-completion rates. Item responses for the RLDQ were skewed towards higher levels of functional ability. PCA supported instrument unidimensionality. Cronbach's alpha ranged from 0.87 (BASDAI) to 0.93 (RLDQ). Test-retest reliability estimates support the use of the ASQoL and RLDQ in individual evaluation (>0.90). Correlations between instruments were in the hypothesized direction; the largest was between the ASQoL and BASDAI (0.79). The BASDAI had the strongest linear relationship, with responses to both specific and general health transition questions (P<0.01). With the exception of the Body Chart, instruments had a stronger relationship with general health transition. The BASDAI was the most responsive instrument. The Body Chart and RLDQ had low levels of responsiveness. CONCLUSION: The instruments have undergone a comprehensive comparative evaluation to assess the measurement properties required for patient-assessed measures of health outcome. Adequate levels of reliability and validity were found for all instruments. The BASDAI and the ASQoL were the most responsive to self-perceived change in health, but the BASDAI had low levels of self-completion.