Tessa Sanderson

Identifying core domains to assess flare in rheumatoid arthritis: an OMERACT international patient and provider combined Delphi consensus

Objective For rheumatoid arthritis (RA), there is no consensus on how to define and assess flare. Variability in flare definitions impairs understanding of findings across studies and limits ability to pool results. The OMERACT RA Flare Group sought to identify domains to define RA flares from patient and healthcare professional (HCP) perspectives. Methods Flare was described as a worsening of disease activity of sufficient intensity and duration to consider a change in therapy. International patients and HCPs participated in separate and combined rounds of Delphi exercises to rate candidate flare domains previously generated in patient focus groups. Core domains were defined as those with ≥70% ratings of being ‘essential’ according to the third/final Delphi exercise. Results The final Delphi included 125 RA patients from 10 countries and 108 HCPs from 23 countries who rated 14 domains. Patients had a mean (±SD) age of 56±12 years and disease duration of 18±12 years. HCPs included physicians from clinical practice/research and industry, allied health providers and researchers with 17±11 years experience. Core domains comprised: pain (93%), function (89%), swollen joints (84%), tender joints (81%), participation (81%), stiffness (79%), patient global assessment (76%) and self-management (75%). Fatigue (68%), which did not reach group consensus, will receive additional consideration. Conclusions As part of the process to develop a measure for RA flare, patients and HCPs agreed on eight core domains. Next steps include identifying items to assess domains and conducting studies to validate and refine a new measure.

The patient perspective on remission in rheumatoid arthritis: ‘You've got limits, but you're back to being you again’

OBJECTIVES The aim of rheumatoid arthritis (RA) treatment is remission. As treatment should be targeted at outcomes relevant to patients, it is important to understand how patients perceive remission, and to assess whether the current definition of remission adequately reflects these perceptions. The objective of this study is to explore the patient perspective on remission in RA. METHODS Nine focus-group discussions in Austria, The Netherlands and UK were conducted, including patients in American College of Rheumatology (ACR)/ European League of Rheumatology (EULAR) remission, self-declared remission and in moderate/high disease activity. Moderators employed a prespecified interview guide helped to engage patients in a discussion on their experience with remission. Inductive thematic analysis was performed within each country, and identified themes were discussed across countries. RESULTS 47 RA patients (66% women, disease duration 9 years) participated. Three major themes of patient-perceived remission emerged: (1) symptoms would either be absent or strongly reduced, (2) impact of the disease on daily life would diminish by increased independence, ability to do valued activities, improved mood and ability to cope; (3) leading to a return to normality, including work, family role and perception of others. Patients felt the concept of remission was influenced by ageing, side effects of medication, comorbidities, accrued damage to joints and disease duration. Opinions on duration of state, the role of medication and measurement instruments varied widely. CONCLUSIONS Patients characterise remission by the absence or reduction of symptoms, but more directly by decreased daily impact of their condition and the feeling of a return to normality. The next step is to study whether an additional patient-perceived measure of remission may add value to the ACR/EULAR definition of remission.

Developing a construct to evaluate flares in rheumatoid arthritis: a conceptual report of the OMERACT RA Flare Definition Working Group.

Rheumatoid arthritis (RA) patients and healthcare professionals (HCP) recognize that episodic worsening disease activity, often described as a “flare,” is a common feature of RA that can contribute to impaired function and disability. However, there is no standard definition to enable measurement of its intensity and impact. The conceptual framework of the Outcome Measures in Rheumatology Clinical Trials (OMERACT) RA Flare Definition Working Group includes an anchoring statement, developed at OMERACT 9 in 2008: “flare in RA” is defined as worsening of signs and symptoms of sufficient intensity and duration to lead to change in therapy. Subsequently, domains characterizing flare have been identified by comprehensive literature review, patient focus groups, and patient/HCP Delphi exercises. This led to a consensus regarding preliminary domains and a research agenda at OMERACT 10 in May 2010. The conceptual framework of flare takes into account validated approaches to measurement in RA: (1) various disease activity indices (e.g., Disease Activity Score, Clinical Disease Activity Index, Simplified Disease Activity Index); (2) use of patient-reported outcomes (PRO); and (3) characterization of minimally clinically detectable and important differences (MCDD, MCID). The measurement of RA flare is composed of data collection assessing a range of unique domains describing key features of RA worsening at the time of patient self-report of flare, and then periodically for the duration of the flare. The components envisioned are: (1) Patient self-report using a “patient global question” with well characterized and validated anchors; (2) Patient assessment using a flare questionnaire and PRO available at the time of each self-report; (3) Physician/HCP assessment of disease activity status; and (4) Physician’s determination whether to change treatment. In randomized controlled trials and observational studies, such a conceptual approach is intended to lead to a valid measure of this outcome/response, thus expanding an understanding of the true impact of a therapy to limit disease activity. Clinically, this approach is intended to enhance patient-HCP communication. This article describes the conceptual framework being used by the OMERACT RA Flare Definition Working Group in developing a standardized method for description and measurement of “flare in RA” to guide individual patient treatment.

Identifying preliminary domains to detect and measure Rheumatoid Arthritis Flares: Report of the OMERACT 10 RA Flare Workshop

Background. While disease flares in rheumatoid arthritis (RA) are a recognized aspect of the disease process, there is limited formative research to describe them. Methods. The Outcome Measures in Rheumatology Clinical Trials (OMERACT) RA Flare Definition Working Group is conducting an international research project to understand the specific characteristics and impact of episodic disease worsening, or “flare,” so that outcome measures can be developed or modified to reflect this uncommonly measured, but very real and sometimes disabling RA disease feature. Patient research partners provided critical insights into the multidimensional nature of flare. The perspectives of patients and healthcare and research professionals are being integrated to ensure that any outcome measurement to detect flares fulfills the first OMERACT criteria of Truth. Through an iterative data-driven Delphi process, a preliminary list of key domains has been identified to evaluate flare. Results. At OMERACT 10, consensus was achieved identifying features of flare in addition to the existing core set for RA, including fatigue, stiffness, symptom persistence, systemic features, and participation. Patient self-report of flare was identified as a component of the research agenda needed to establish criterion validity for a flare definition; this can be used in prospective studies to further evaluate the Discrimination and Feasibility components of the OMERACT filter for a flare outcome measure. Conclusion. Our work to date has provided better understanding of key aspects of the RA disease process as episodic, potentially disabling disease worsening even when a patient is in low disease activity. It also highlights the importance of developing ways to enhance communication between patients and clinicians and improve the ability to achieve “tight control” of disease.

Progress on Incorporating the Patient Perspective in Outcome Assessment in Rheumatology and the Emergence of Life Impact Measures at OMERACT 9

The Patient Perspective Workshop included over 100 researchers and 18 patient participants from 8 countries. Following preconference reading and short plenary presentations, breakout groups considered work undertaken on measurement of sleep, assessing interventions to develop the effective consumer, and assessing psychological and educational interventions. The workshop explored the best way to identify other outcome domains (and instruments) that should be measured in observational or interventional studies with broader intentions than simply altering outcomes captured in the traditional “core set” plus fatigue. Four sleep questionnaires showed promise and will be the subject of further study. The Effective Consumer scale (EC-17) was reviewed and the concept Effective Consumer was well received. Participants thought it worthwhile to measure the skills and attributes of an effective consumer and develop an intervention that would include education in all of the scale’s categories. Assessment of educational and psychological interventions requires a wider set of instruments than is currently used; these should relate to the purpose of the intervention. This principle was extended to include wider measures of the impact of disease on life, as indicated in the International Classification of Functioning, Disability and Health. Life impact measure sets covering domains appropriate to different rheumatic conditions and focused on different interventions might be defined by future OMERACT consensus. Measurement instruments within these domains that are valid for use in rheumatic conditions can then be identified and, in the case of psychological and educational interventions, chosen to fit with the purpose of the intervention.

Patient-reported outcomes for arthritis: time to focus on personal life impact measures?

Only patients themselves can report their perspective on the outcomes of illness and its treatment. For some treatment effects, the patient may be the only source of information. Therefore, patient-reported outcomes (PROs) are standard measures of pain and have become standards for functional disability and global health/disease activity due to their inclusion in American College of Rheumatology (ACR) criteria and other disease activity measures (1,2). They have been shown to be as effective as the traditional physicianor laboratory-reported outcomes in reflecting changes in disease activity over time and predicting longterm morbidity and mortality (3). In addition, there are a range of health-related quality of life instruments to measure the broader impact of conditions (4). However, these currently used PROs are not necessarily synonymous with the patient perspective. The US Food and Drug Administration, recognizing the need to incorporate this wider patient perspective in drug evaluation programs, has called for (and issued draft guidance on) standardization of methods for developing PROs that incorporate the patient perspective (5). We believe that the development of measures across rheumatic conditions and a wide range of interventions will be beneficial. Examining the patient’s experience, as reported in new data from the BeST study in this issue of Arthritis Care & Research (including patient-reported functional difficulties) (6), may be particularly crucial in the context of chronic conditions that have a major impact on patients but are only partially treatable, which is a common situation in rheumatology. The multidisciplinary team intervenes not only to change the disease process itself, but also to ameliorate the shortand long-term consequences of the condition. This can be achieved through other therapeutic measures (such as compensating for loss of function by increasing general fitness) or through changes in personal or social circumstances (such as education about appropriate domestic arrangements or altering the living environment). PROs have increasingly been used in rheumatology to test treatment strategies in clinical trials where interventions may be close to the disease pathology, but PROs have a much wider use in health-related domains such as informing individual patient care in a routine practice setting, in health policy and resource allocation, in the measurement of health care quality (7), and capturing participation in society (8). The patient’s perspective of the impact of rheumatic conditions is broad (9), whereas the clinician tends to focus on pathology and functional disability. The growing amount of literature on the patient perspective has revealed 2 important issues: clinicians and patients have different perspectives on outcomes (10), and patients prioritize treatment outcomes that are not routinely measured, such as well-being, normality, and sleep (11–14). Differences between what clinicians and patients believe is important are sometimes due to prioritizing treatment of disease over its consequences, and/or a lack of research about particular aspects of the disease. For example, the significance or multidimensionality of fatigue in rheumatoid arthritis (RA) was not recognized by health professionals (15), nor was dyscognition in fibromyalgia (“fibro-fog”) (16). Mapping the experience of symptoms in detail, the impact of conditions on different domains of life, and patients’ important treatment outcomes may enhance rheumatology research at many levels, from the underlying mechanisms of the disease to the broad social impact. First, understanding the experience of symptoms in detail may provide new insights into the mechanisms of the disease and/or provide evidence for the cohesion (or otherwise) of symptoms caused by different pathologic processes. For example, morning stiffness was improved by time-released glucocorticoids when other symptoms remained unchanged (17). Second, discrepancies between the patient’s experience and apparent disease control may be identified. Treatment may result in a clinically significant reduction in the Disease Activity Score (DAS), but not change in a patient’s important outcomes, such as being able to climb stairs. Third, patient-clinician communication may be improved by establishing the different ways in which common terms, such as flare, are used (18), or the Tessa Sanderson, MSc: University of West England, Bristol, UK; John Kirwan, MD: University of Bristol, Bristol, UK. Address correspondence to John Kirwan, MD, Division of Medicine, Bristol Royal Infirmary, Marlborough Street, Bristol, Avon, UK BS2 8HW. E-mail: john.kirwan@bristol. ac.uk. Submitted for publication October 6, 2008; accepted in revised form October 20, 2008. Arthritis & Rheumatism (Arthritis Care & Research) Vol. 61, No. 1, January 15, 2009, pp 1–3 DOI 10.1002/art.24270

The Impact Triad (Severity, Importance, Self-management) as a Method of Enhancing Measurement of Personal Life Impact of Rheumatic Diseases

Clinical trials in rheumatoid arthritis (RA) currently focus on measuring severity of functional disability, patient global assessment, pain, and morning stiffness1. However, the inclusion of patient-reported outcome measures (PROM) to assess the impact of illness has been recognized as an important development, and collaboration with patients in developing PROM is becoming mandatory2. While it is recognized that patients and professionals may have different values and priorities3, discussion continues about how to incorporate outcomes that are important to patients. Recent collaborations with patients in the conception of outcome measures have revealed 3 aspects of impact that require assessment. These are the severity of an outcome, its importance to the patient, and patient ability to self-manage (Figure 1). We provide 3 examples of how this impact triad emerged in recent RA research and discuss how the concept may influence the development of future instruments. Figure 1. The impact triad. The first example comes from extensive focus group data exploring a patient definition of flare in RA4. The emergent definition included 3 components: as symptom intensity increased (i.e., severity), symptoms were sufficiently different from normal background variations (i.e., became important), and the actions patients took to deal with them failed (i.e., self-management). This resulted in a tipping point being reached where medical help was sought for uncontrolled flare. The patients’ complex model of flare goes beyond a simple increase in the number of swollen joints or the intensity of pain, to include these additional considerations. Therefore, the future development of a flare instrument will require not only items on the severity of flare-related symptoms, but also items on how these are different from patients’ perceptions of normal background symptoms and items on the controllability of symptoms. The second example comes from the development of a set of … Address correspondence to Prof. S.E. Hewlett, Academic Rheumatology, Bristol Royal Infirmary, Bristol BS2 8HW. E-mail: Sarah.Hewlett{at}uwe.ac.uk

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.

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.

‘It’s this whole picture, this well-being’: patients’ understanding of ‘feeling well’ with rheumatoid arthritis

Objectives: The aim of this study was to explore the meaning of ‘feeling well’ for people with rheumatoid arthritis (RA). Methods: In-depth interviews were conducted with 23 RA patients, purposively sampled for medication type, disease duration, disease activity, age and gender. Data were analysed using Framework, emphasizing participants’ personal contexts. Results: Well-being was viewed as a broad concept, with ‘feeling well’ being the result of an on-going process to actively engage with a changing body, self and life. Four dimensions emerged: ‘Living in the body’, ‘Being in the mind’, ‘Adapting to illness’ and ‘Being in the world’. The physical impact of RA underpinned the global perception of well-being and was clearly described as linking to the experience of psychological well-being. Physical and psychological wellness was often affected by the individual’s adaptation to RA and personal context (e.g. home environment, broader social attitudes). Discussion: Well-being is a multidimensional concept that is meaningful to RA patients regardless of medication type and disease severity. Patients commonly illustrated a process of actively engaging in cognitive and behavioural adjustments to move towards wellness. Clinical practice and research must take account of the complexity of well-being in long-term conditions, in order to fulfil patients’ expectations.