Stakeholder endorsement advancing the implementation of a patient-reported domain for harms in rheumatology clinical trials: Outcome of the OMERACT Safety Working Group.

OBJECTIVES: To develop an understanding of the concept of safety/harms experienced by patients involved in clinical trials for their rheumatic and musculoskeletal diseases (RMDs) and to seek input from the OMERACT community before moving forward to developing or selecting an outcome measurement instrument. METHODS: OMERACT 2023 presented and discussed interview results from 34 patients indicating that up to 171 items might be important for patients' harm-reporting. RESULTS: Domain was defined in detail and supported by qualitative work. Participants in the Special-Interest-Group endorsed (96 %) that enough qualitative data are available to start Delphi survey(s). CONCLUSION: We present a definition of safety/harms that represents the patient voice (i.e., patients' perception of safety) evaluating the symptomatic treatment-related adverse events for people with RMDs enrolled in clinical trials.

Nursing sensitive outcomes in patients with rheumatoid arthritis: A systematic literature review.

BACKGROUND: Although rheumatology nursing has been shown to be effective in managing patients with rheumatoid arthritis, patient outcomes sensitive to nursing interventions (nursing sensitive outcomes) have not been systematically studied. OBJECTIVES: The objective of this study was to identify and delineate relevant patient outcomes measured in studies that reported nursing interventions in patients with rheumatoid arthritis. DESIGN: A systematic search was conducted from 1990 to 2016. Inclusion criteria were (i) patients with rheumatoid arthritis, (ii) adult population age ≥16years, (iii) nurse as part of the care team or intervention delivery, (iv) primary research only, (v) English language, and (vi) quantitative studies with nursing sensitive outcomes. DATA SOURCES: Medline, CINAHL, Ovid nursing, Cochrane library and PsycINFO databases were searched for relevant studies. REVIEW METHODS: Using the predetermined inclusion/exclusion criteria, nine reviewers working in pairs assessed the eligibility of the identified studies based on titles and abstracts. Papers meeting the inclusion criteria were retrieved and full texts were further assessed. Critical Appraisal Skills Programme tools were used to assess the quality of the included studies. Data on nursing sensitive outcomes were extracted independently by two reviewers. The Outcome Measures in Rheumatology comprehensive conceptual framework for health was used to contextualise and present findings. RESULTS: Of the 820 articles retrieved, 7 randomised controlled trials and 3 observational studies met the inclusion criteria. Seventeen nursing sensitive outcomes were identified (disease activity, clinical effects, pain, early morning stiffness duration, fatigue, patient safety issues, function, knowledge, patient satisfaction, confidence in care received, mental health status, self-efficacy, patient attitude/perception of ability to control arthritis, quality of life, health utility, health care resources, death). These fitted into 10 health intervention domains in keeping with the pre-specified conceptual framework for health: disease status, effectiveness, safety, function, knowledge, satisfaction, psychological status, quality of life, cost, death. A total of 59 measurement instruments were identified comprising patient reported outcome measures (n=31), and biologic measures and reports (n=28). CONCLUSIONS: This review is notable in that it is the first to have identified, and reported, a set of multidimensional outcome measures that are sensitive to nursing interventions in rheumatology specifically. Further research is required to determine a core set of outcomes to be used in all rheumatology nursing intervention studies.

A Patient-reported Outcome Measure for Effect of Glucocorticoid Therapy in Adults with Inflammatory Diseases Is Needed: Report from the OMERACT 2016 Special Interest Group.

OBJECTIVE: The need for a standardized instrument to measure the effect of glucocorticoid (GC) therapy has been well documented in the literature. The aim of the first GC Special Interest Group was to define a research agenda around the development of a patient-reported outcome measure (PROM) in this area. METHODS: The results of a background literature search and the preliminary results of a pilot survey and 2 qualitative studies were presented to facilitate the development of a research agenda. RESULTS: It was agreed that there was a need for a data-driven PROM that identified both positive and negative effects of GC therapy to be used across all inflammatory indications for systemic GC use in adults. A research agenda was developed, consisting of further qualitative work to assess the effect of GC across different groups including various indications for GC use, different age groups, different dosages, and duration of treatment. CONCLUSION: There was agreement on the need for a PROM in this area and a research agenda was set.

Treating rheumatoid arthritis to target: 2014 update of the recommendations of an international task force.

BACKGROUND: Reaching the therapeutic target of remission or low-disease activity has improved outcomes in patients with rheumatoid arthritis (RA) significantly. The treat-to-target recommendations, formulated in 2010, have provided a basis for implementation of a strategic approach towards this therapeutic goal in routine clinical practice, but these recommendations need to be re-evaluated for appropriateness and practicability in the light of new insights. OBJECTIVE: To update the 2010 treat-to-target recommendations based on systematic literature reviews (SLR) and expert opinion. METHODS: A task force of rheumatologists, patients and a nurse specialist assessed the SLR results and evaluated the individual items of the 2010 recommendations accordingly, reformulating many of the items. These were subsequently discussed, amended and voted upon by >40 experts, including 5 patients, from various regions of the world. Levels of evidence, strengths of recommendations and levels of agreement were derived. RESULTS: The update resulted in 4 overarching principles and 10 recommendations. The previous recommendations were partly adapted and their order changed as deemed appropriate in terms of importance in the view of the experts. The SLR had now provided also data for the effectiveness of targeting low-disease activity or remission in established rather than only early disease. The role of comorbidities, including their potential to preclude treatment intensification, was highlighted more strongly than before. The treatment aim was again defined as remission with low-disease activity being an alternative goal especially in patients with long-standing disease. Regular follow-up (every 1-3 months during active disease) with according therapeutic adaptations to reach the desired state was recommended. Follow-up examinations ought to employ composite measures of disease activity that include joint counts. Additional items provide further details for particular aspects of the disease, especially comorbidity and shared decision-making with the patient. Levels of evidence had increased for many items compared with the 2010 recommendations, and levels of agreement were very high for most of the individual recommendations (≥9/10). CONCLUSIONS: The 4 overarching principles and 10 recommendations are based on stronger evidence than before and are supposed to inform patients, rheumatologists and other stakeholders about strategies to reach optimal outcomes of RA.

Updating the OMERACT filter: implications for patient-reported outcomes.

OBJECTIVE: At a previous Outcome Measures in Rheumatology (OMERACT) meeting, participants reflected on the underlying methods of patient-reported outcome (PRO) instrument development. The participants requested proposals for more explicit instrument development protocols that would contribute to an enhanced version of the "Truth" statement in the OMERACT Filter, a widely used guide for outcome validation. In the present OMERACT session, we explored to what extent these new Filter 2.0 proposals were practicable, feasible, and already being applied. METHODS: Following overview presentations, discussion groups critically reviewed the extent to which case studies of current OMERACT Working Groups complied with or negated the proposed PRO development framework, whether these observations had a more general application, and what issues remained to be resolved. RESULTS: Several aspects of PRO development were recognized as particularly important, and the need to directly involve patients at every stage of an iterative PRO development program was endorsed. This included recognition that patients contribute as partners in the research and not merely as subjects. Correct communication of concepts with the words used in questionnaires was central to their performance as measuring instruments, and ensuring this understanding crossed cultural and linguistic boundaries was important in international studies or comparisons. CONCLUSION: Participants recognized, endorsed, and were generally already putting into practice the principles of PRO development presented in the plenary session. Further work is needed on some existing instruments and on establishing widespread good practice for working in close collaboration with patients.

It's like a juggling act: rheumatoid arthritis patient perspectives on daily life and flare while on current treatment regimes.

OBJECTIVE: The objective of this study was to explore patients' experiences of RA daily life while on modern treatments. METHODS: The methods of this study comprised semi-structured interviews with 15 RA patients, analysed using inductive thematic analysis. RESULTS: Four themes suggest patients experience life with RA along a continuum from RA in the background to the foreground of their lives, underpinned by constant actions to maintain balance. Living with RA in the background shows patients experience continuous, daily symptoms, which they mediate through micromanagement (mediating the impact of RA on daily life), while learning to incorporate RA into their identity (redefining me). RA moving into the foreground shows patients experience fluctuating symptoms (unwelcome reminders) that may or may not lead to a flare (trying to make sense of fluctuation). Dealing with RA in the foreground shows how patients attempt to manage RA flares (trying to regain control) and decide to seek medical help only after feeling they are losing control. Patients employ a stepped approach to self-management (mediation ladder) as symptoms increase, with seeking medical help often seen as the last resort. Patients seek to find a balance between managing their fluctuating RA and living their daily lives. CONCLUSION: Patients move back and forth along a continuum of RA in the background vs the foreground by balancing self-management of symptoms and everyday life. Clinicians need to appreciate that daily micromanagement is needed, even on current treatment regimes. Further research is needed to quantify the level and impact of daily symptoms and identify barriers and facilitators to seeking help.

Utilizing qualitative data from nominal groups: exploring the influences on treatment outcome prioritization with rheumatoid arthritis patients.

The nominal group technique generates quantitative data through a process of experts ranking items of interest. This article focuses on the additional collection of qualitative data from nominal groups with rheumatoid arthritis (RA) patients, used to explore the influences on prioritizing treatment outcomes. Across all groups, the top five outcomes with the highest importance scores were identified as: pain; joint damage; fatigue; activities of daily living; and mobility. Qualitative findings showed that the personal impact of RA influenced decisions on how to rank specific outcomes through four domains: disease impact; adaptation to illness; external resources and stressors; and social expectations.

Patient perspective workshop: moving towards OMERACT guidelines for choosing or developing instruments to measure patient-reported outcomes.

The workshop Choosing or Developing Instruments held at the Outcome Measures in Rheumatology (OMERACT) 10 meeting was designed to help participants think about the underlying methods of instrument development. Conference pre-reading material and 3 brief introductory presentations elaborated the issues, and participants broke into discussion groups before reconvening to share insights, engage in a more general discussion of the issues, and vote on recommendations. Tradeoffs between using current imperfect measures and the long and complex process of developing new instruments were considered, together with the need for rigor in patient-reported outcome (PRO) instrument development. The main considerations for PRO instrument development were listed and a research agenda for action produced. As part of the agenda for action, it is recommended that researchers and patient partners work together to tackle these issues, and that OMERACT bring forward proposals for acceptable instrument development protocols that would meet an enhanced "Truth" statement in the OMERACT Filter.

Shifting normalities: interactions of changing conceptions of a normal life and the normalisation of symptoms in rheumatoid arthritis.

Biographical disruption and related concepts have been widely applied in chronic illness. Different conceptualisations of normalisation have also been proposed in order to explain individuals' diverse responses to illness on their biography, but as yet, not clearly related to changing bodily experience or normalisation of symptoms. This article aims to examine the relevance of these concepts in rheumatoid arthritis (RA), an unpredictable autoinflammatory disease characterised by painful and swollen joints, disability, fatigue and joint damage. Interviews were conducted with 23 people living with RA, and analysed using Framework, to enable people's whole narratives and context to be considered. Six typologies of normality emerged from the data: disrupted; struggling to maintain; fluctuating; resetting; returning; and continuing normality. Multiple normalities were often present in individuals' narratives, with one normality typology usually dominating at the time of the interview. The typologies connect to several biographical concepts, and instances of 'biographical reinstatement' were also found, where participants described returning to normal life, through perceived effective medication rather than reconceptualisation of health. The concept of 'shifting normalities' is proposed, providing a dynamic explanatory model of chronic illness that captures the interaction of changing conceptions of a normal life and the normalisation of symptoms.

The impact of patient-perceived restricted access to anti-TNF therapy for rheumatoid arthritis: a qualitative study.

OBJECTIVE: To explore rheumatoid arthritis (RA) patients' experience of access to anti-tumour necrosis factor (anti-TNF) therapy in the UK, and of switching therapies after an initial failure. METHODS: Patients were asked about their experience of accessing, receiving and discontinuing anti-TNF therapy in face-to-face indepth interviews, within the context of the larger study about treatment outcomes. Seventeen individuals with a diagnosis of RA and experience of receiving anti-TNF therapy were interviewed in one hospital trust in England. RESULTS: Different emotions (Theme 1) surrounded the process of accessing anti-TNF therapy: hope, desperation, apprehension, anxiety and frustration. Experience of receiving anti-TNF therapy (Theme 2) included not only positive transformation, but also fear of failure and discontinuation. The subsequent value that patients placed on anti-TNF therapy (Theme 3) included having a right to receive therapy and being lucky. These three themes were underpinned by the core category of 'willing to try anything'. Those switching therapies reported increased caution over the possibility of recurring side effects, but some suggestion of benefit. There was a perception that access to anti-TNF therapy was restricted by cost, rather than being recommended for those in clinical need. CONCLUSIONS: Anti-TNF therapies may have a sudden and dramatic impact on RA patients' lives that contrast with other available medications. However, the stress of the patient's journey through the need to 'qualify' for anti-TNF therapy, and the fear of failing or discontinuation of therapy, should not be underestimated by clinicians.

Sensitivity to change of the Rheumatoid Arthritis Self-Efficacy scale (RASE) and predictors of change in self-efficacy.

OBJECTIVES: Patient education in rheumatoid arthritis (RA) aims to improve health outcomes by prompting people to adopt self-management behaviours. One precursor for initiating behaviour change is self-efficacy (SE), a belief that you can do a task. This study tested the sensitivity to change of a new scale to measure SE for self-management in people with RA, the Rheumatoid Arthritis Self-Efficacy scale (RASE). Exploratory analysis examined potential predictors of change in SE. METHODS: People with RA at 11 rheumatology centres, who had accepted an education programme as part of clinical care, completed questionnaires at baseline, and two and eight weeks after their programme end. Programmes were not standardized, as this was a pragmatic study in clinical practice. RESULTS: A total of 128 patients participated. After controlling for baseline scores, the RASE showed small but significant improvements in SE from baseline (RASE 107.57, CI 105.42-109.72) to two weeks after programme end (RASE 110.80, CI 108.60-112.99), and eight weeks (RASE 110.62, CI 108.40-112.85, p<0.001). Standardized response means, calculated both by absolute and percentage change, were 0.339 and 0.371 at two weeks after programme end, and 0.321 and 0.352 at eight weeks. Changes in the RASE were associated with behaviour initiation at two and eight weeks (r=0.419, r=0.342, p<0.001). No substantial predictors of change in SE could be identified. CONCLUSIONS: The RASE is sensitive to change in a cohort of people with RA in the UK receiving education programmes as routine clinical care. Exploratory analysis did not identify clinical or psychological factors that predict change in SE, suggesting that programmes should not be restricted to particular patients.

Self-efficacy as an appraisal that moderates the coping-emotion relationship: associations among people with rheumatoid arthritis.

The way a coping strategy is expressed might depend on the nature of underlying efficacy expectations. For example, a cognitive coping strategy may have different content depending on efficacy beliefs underpinning the strategy's formulation and application. As such, self-efficacy (SE), as an appraisal, may moderate relationships between coping and outcomes: coping effects may differ depending on SE. This process was examined in 127 rheumatoid arthritis patients attending routine patient education/self-management programmes. Participants completed questionnaire measures of SE, coping, anxiety and depression at baseline and at 8 weeks follow-up. Regression analyses focused on coping and SE change variables, and their concurrent association with measures of change in anxiety and depression. Results highlighted contributions to these emotional variables from interactions between coping and SE. The nature of associations between coping and emotional outcomes was found to differ according to efficacy appraisals. This may have implications for clinical practice in that the adaptive significance of adjustment efforts may differ according to underlying SE.

Patient perspective: fatigue as a recommended patient centered outcome measure in rheumatoid arthritis.

The Patient Perspective Workshop at OMERACT 8 considered evidence for the importance of fatigue to patients with rheumatoid arthritis (RA) and whether measurement of fatigue meets the requirements of the OMERACT filter. The workshop participants included 20 patients from 10 countries and 60 other OMERACT participants. Introductory papers and detailed notes for discussion group members set out the evidence from the literature and from recent analyses of clinical study data available to several participants. The workshop concluded that fatigue is a symptom that is important to patients, is commonly reported by patients, is often severe, can be measured by several current instruments that pass the OMERACT filter, is responsive to some interventions, and provides information additional to that commonly obtained from currently used outcomes. The final OMERACT plenary session endorsed by a very large majority (89%) the proposal that, in addition to the "core set" of outcome measures currently in widespread use, fatigue should be measured in future studies of RA whenever possible.

Progress since OMERACT 6 on including patient perspective in rheumatoid arthritis outcome assessment.

The first OMERACT Patient Perspective Workshop took place at OMERACT 6 in 2002. Through a series of meetings and discussion sessions a research agenda emerged and this report outlines progress made on this agenda. Work on identifying novel outcomes, instruments, and methods has shown similarities across European countries in the importance patients with rheumatoid arthritis (RA) attach to specific outcomes, in particular fatigue. Validation of an appropriate instrument to measure fatigue in patients with RA is currently being investigated. Frequent or repeated real-time assessment of symptoms such as pain and fatigue is becoming possible using electronic systems. An OMERACT Patient Panel has been established, and has produced a glossary for patients involved in supporting clinical research. In some centers, efforts are being made to provide Patient Research Partners with knowledge and skills that will enhance their contribution, and some of these approaches will be incorporated into OMERACT 7. The research agenda that was developed during the first Patient Perspective Workshop has stimulated new work in several areas. In addition, international attention has been drawn to the need to make sure that the patient's perspective is not lost among the technical expertise of rheumatology.

Outcomes generated by patients with rheumatoid arthritis: how important are they?

BACKGROUND: It has been shown previously that patients with rheumatoid arthritis (RA) can generate a wide range of outcomes that they consider important in treatment. It is not known if these outcomes are generally important in the wider RA patient community. OBJECTIVES: (1) To examine whether recent patient-generated outcomes are generalizable within a wider RA population; (2) to assess the relative importance of each outcome; and(3) to explore whether any important outcomes have been omitted. METHODS: A questionnaire, listing 23 outcomes previously generated by RA patients, was distributed through three rheumatology centres in the UK. Patients gave an importance score to each outcome (0-3), selected their top three most important outcomes, and then listed any outcomes of personal importance that were missing. RESULTS: 323 questionnaires were returned (65%). All outcomes were deemed important. Independence, pain, and mobility were most frequently selected by patients in their top three outcomes but were not chosen by 61-66% of patients. The next most commonly chosen outcomes related to feeling well and fatigue. Factor analysis revealed six reasonably distinct groupings: general well-being (11.9% explained variance), day-to-day functioning(10.6%), emotional and psychological well-being (10.6%), social role and confidence (10%), physical symptoms (9.5%) and medication issues (7.9%). CONCLUSION: Outcomes generated by patients as important in RA, are generalizable and inclusive. The most important (independence, pain and mobility) are routinely treated and measured. The next most important (feeling well, fatigue) are infrequently addressed and deserve urgent consideration for measurement, treatment and research.