Kirstie L. Haywood

Patient and Public Involvement in Patient-Reported Outcome Measures

This paper considers the potential for collaborative patient and public involvement in the development, application, evaluation, and interpretation of patient-reported outcome measures (PROMs). The development of PROMs has followed a well trodden methodological path, with patients contributing as research subjects to the content of many PROMs. This paper argues that the development of PROMs should embrace more collaborative forms of patient and public involvement with patients as research partners in the research process, not just as those individuals who are consulted or as subjects, from whom data are sourced, to ensure the acceptability, relevance, and quality of research. We consider the potential for patients to be involved in a much wider range of methodological activities in PROM development working in partnership with researchers, which we hope will promote paradigmal evolution rather than revolution.

Developing the evidence base of patient and public involvement in health and social care research: the case for measuring impact

While patient and public involvement (PPI) in health and social care research has progressed successfully in the last decade, a range of difficulties with the evidence base exist, including poor understanding of the concept of impact, limited theorization and an absence of quantitative impact measurement. In this paper, we argue that a paradigm change towards robust measurement of the impact of involvement in research is needed to complement qualitative explorations. We argue that service users should be collaboratively involved in the conceptualization, theorization and development of instruments to measure PPI impact. We consider the key advantages measurement would bring in strengthening the PPI evidence base through a greater understanding of what works, for whom, in what circumstances and why.

Quality and acceptability of patient-reported outcome measures used in chronic fatigue syndrome/myalgic encephalomyelitis (CFS/ME): a systematic review.

PurposeTo review the quality and acceptability of condition-specific, domain-specific and generic multi-item patient-reported outcome measures (PROMs) used in the assessment of adults with chronic fatigue syndrome/myalgic encephalomyelitis (CFS/ME).MethodsSystematic literature searches were made to identify PROMs. Quality and acceptability was assessed against an appraisal framework, which captured evidence of both the thoroughness and results of evaluations: evidence of measurement (reliability, validity, responsiveness, interpretability, data quality/precision) and practical properties (feasibility, patient acceptability), and the extent of active patient involvement was sought.ResultsA total of 11 CFS/ME-specific, 55 domain-specific and 11 generic measures were reviewed. With the exception of the generic SF-36, all measures had mostly limited evidence of measurement and/or practical properties. Patient involvement was poorly reported and often cursory.ConclusionsThe quality and acceptability of reviewed PROMs is limited, and recommendations for patient-reported assessment are difficult. Significant methodological and quality issues in PROM development/evaluation were identified by the appraisal framework, which must be addressed in future research. Clear discrepancies exist between what is measured in research and how patients define their experience of CFS/ME. Future PROM development/evaluation must seek to involve patients more collaboratively to measure outcomes of importance using relevant and credible methods of assessment.

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.

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.

Ethik der Reanimation und Entscheidungen am Lebensende

Der unerwartete Kreislaufstillstand stellt ein Ereignis dar, das haufig verheerende Folgen fur die betroffene Person, Fami-lie und Freunde mit sich bringt. Wahrend einige Wiederbelebungsversuche erfolg-reich mit einem guten Langzeit-Outcome einhergehen, trifft dies fur die Mehrzahl der Falle trotz groser Anstrengungen und einiger Verbesserungen in der Versorgung von Herz-Kreislauf-Patienten wahrend des letzten Jahrzehnts nicht zu.Professionelle Helfer sind verpflichtet, alles Notige zu tun, um Leben zu schut-zen und zu bewahren. Die Gesellschaft als Ganzes, besonders aber Rettungsdienst-personal, Krankenhauser und andere Ein-richtungen des Gesundheitswesens mus-sen fur den Fall des plotzlichen Kreislauf-stillstands geeignete Masnahmen planen, organisieren und anwenden. Damit sind meist der Einsatz vieler Ressourcen und hohe Kosten verbunden, v. a. in den wohl-habenderen Landern. Technische Neue-rungen, medizinische Evidenz und stei-gende Erwartungen der Offentlichkeit haben ethische Uberlegungen zu einem wichtigen Bestandteil jeglicher Interven-tion oder Entscheidung am Lebensende werden lassen. Dazu gehort, durch geeig-nete Allokation der verfugbaren Ressour-cen das bestmogliche Ergebnis fur den einzelnen Patienten, die Angehorigen und die gesamte Gesellschaft zu erzielen.Um sicherzustellen, dass Entschei-dungen, mit Wiederbelebungsversuchen zu beginnen oder nicht, angemessen sind und dass Patienten mit Wurde behandelt werden, bedarf es einiger Erwagungen. Derartige Entscheidungen sind komplex und konnen durch individuelle, internati-onal und lokal kulturelle, rechtliche, traditi-onelle, religiose, soziale und okonomische Faktoren beeinflusst werden [1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11]. Zuweilen konnen die Ent-schlusse im Voraus getroffen werden, aber haufig mussen diese schwierigen Entschei-dungen zum Zeitpunkt des Notfalls in-nerhalb von Sekunden oder Minuten fal-len, insbesondere im auserklinischen Be-reich aufgrund von begrenzten Informati-onen. Daher ist es wichtig, dass professio-nelle Helfer sich mit den zugrunde liegen-den Prinzipien vertraut machen, bevor sie in eine Situation geraten, in der eine Re-animationsentscheidung getroffen wer-den muss. Entscheidungen zum Lebens-ende und ethische Erwagungen sollen von professionellen Helfern also im Vorhinein und im jeweiligen gesellschaftlichen Kon-text erfolgen. Obwohl es nur wenige wis-senschaftliche Grundlagen gibt, die Anlei-tungen fur Entscheidungen am Lebensen-de bieten konnten, handelt es sich um ein wichtiges Thema. Daher wurden Informa-tionen fur professionelle Helfer in diese Re-animationsleitlinien aufgenommen.Diese Sektion der Leitlinien beschaf-tigt sich mit einigen wiederkehrenden ethischen Aspekten und Entscheidungen am Lebensende:F Ethische Grundprinzipien,F plotzlicher Kreislaufstillstand aus glo-baler Sicht,F Outcome und Prognosestellung,F wann Wiederbelebungsversuche be-ginnen und wann beenden,F Patientenverfugungen und Anwei-sungen zum „do not attempt resusci-tation“ (DNAR, d. h. kein Reanimati-onsversuch),F Anwesenheit von Familienangehori-gen wahrend der Reanimation,F Organbeschaffung,F Reanimationsforschung und infor-mierte Einwilligung sowieF Forschung und Ausbildung an gerade Verstorbenen.

EuroQol EQ-5D and condition-specific measures of health outcome in women with urinary incontinence: reliability, validity and responsiveness

ObjectivesTo evaluate the measurement properties of the EuroQoL EQ-5D and two condition-specific patient-reported outcome measures—the Symptom Severity Index (SSI) and the Urinary Incontinence-Specific Quality of Life instrument (I-QoL)—in women with urinary incontinence.MethodsA questionnaire comprising all instruments was completed by women taking part in a clinical trial of physiotherapy for urinary incontinence. Follow-up questionnaires were at 6 weeks and 5 months. Data quality, internal consistency reliability, validity and responsiveness were assessed.ResultsOne hundred and seventy-four patients taking part in the clinical trial completed the questionnaire. Instruments had low levels of missing data. The EQ-5D had a large ceiling effect and poor responsiveness. The SSI had poor validity and responsiveness. The I-QoL had levels of reliability that supported application in group assessment, and in some cases, individual assessment, and good evidence of validity. The I-QoL was the most responsive instrument at both 6 weeks and 5 months.ConclusionThe I-QoL was the best performing instrument and is recommended as a continence-specific measure of quality of life in a clinical trial setting. The SSI and EQ-5D are not recommended. Alternative generic instruments, which support economic evaluation, require further evaluation in trials of female urinary incontinence.

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

A range of terms (including quality of life, health-related quality of life and health status) describe a broad set of aspects of self-reported health that can be captured by questionnaires and related instruments. Health in older people particularly requires attention to broader aspects of the impact of health on physical, social and psychological function. A structured review was performed to assess how well such issues are measured by available instruments. A total of 45 instruments were identified that have received some degree of evaluation to establish their use in assessing the health of older individuals. Two types of instruments were included in the review: generic and older-people-specific instruments. The SF-36 has by far the largest evidence base to support its use in older people. The OMFAQ has the best evidence base amongst older-people-specific instruments. Many instruments have only minimal evidence of measurement properties. Concepts such as quality of life are still defined and used in a variety of ways, and major questions must be addressed before uniform practice emerges in this important area of health policy.

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:

Emerging Guidelines for Patient Engagement in Research

There is growing recognition that involving patients in the development of new patient-reported outcome measures helps ensure that the outcomes that matter most to people living with health conditions are captured. Here, we describe and discuss different experiences of integrating patients as full patient research partners (PRPs) in outcomes research from multiple perspectives (e.g., researcher, patient, and funder), drawing from three real-world examples. These diverse experiences highlight the strengths, challenges, and impact of partnering with patients to conceptualize, design, and conduct research and disseminate findings. On the basis of our experiences, we suggest basic guidelines for outcomes researchers on establishing research partnerships with patients, including: 1) establishing supportive organizational/institutional policies; 2) cultivating supportive attitudes of researchers and PRPs with recognition that partnerships evolve over time, are grounded in strong communication, and have shared goals; 3) adhering to principles of respect, trust, reciprocity, and co-learning; 4) addressing training needs of all team members to ensure communications and that PRPs are conversant in and familiar with the language and process of research; 5) identifying the resources and advanced planning required for successful patient engagement; and 6) recognizing the value of partnerships across all stages of research. The three experiences presented explore different approaches to partnering; demonstrate how this can fundamentally change the way research work is conceptualized, conducted, and disseminated; and can serve as exemplars for other forms of patient-centered outcomes research. Further work is needed to identify the skills, qualities, and approaches that best support effective patient-researcher partnerships.