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Aim: We developed the Patient-Engaged Health Technology Assessment strategy for survey-based goal collection from patients to yield patient-important outcomes suitable for use in multi-criteria decision analysis. Methods: Rheumatoid arthritis patients were recruited from online patient networks for proof-of-concept testing of goal collection and prioritization using a survey. A Project Steering Committee and Expert Panel rated the feasibility of scaling to larger samples. Results: Survey respondents (n = 47) completed the goal collection exercise. Finding effective treatments was rated by respondents as the most important goal, and reducing stiffness was rated as the least important. Feedback from our steering committee and expert panel support the approach's feasibility for goal identification and ranking. Conclusion: Goals relevant for treatment evaluation can be identified and rated for importance by patients to permit wide input from patients with lived experience of disease.

Explore trial participants’ and research team members’ perceptions of the impact of the videoconference-based, supportive care program (SPIN-CHAT Program) during early COVID-19 for individuals with systemic sclerosis (SSc). Data were collected cross-sectionally. A social constructivist paradigm was adopted, and one-on-one videoconference-based, semi-structured interviews were conducted with SPIN-CHAT Trial participants and research team members. A hybrid inductive-deductive approach and reflexive thematic analysis were used. Of the 40 SPIN-CHAT Trial participants and 28 research team members approached, 30 trial participants (Mean age = 54.9; SD = 13.0 years) and 22 research team members agreed to participate. Those who took part in interviews had similar characteristics to those who declined. Five themes were identified: (1) The SPIN-CHAT Program conferred a range of positive psychological health outcomes, (2) People who don’t have SSc don’t get it: The importance of SSc-specific programming, (3) The group-based format of the SPIN-CHAT Program created a safe space to connect and meet similar others, (4) The structure and schedule of the SPIN-CHAT Program reduced feelings of boredom and contributed to enhanced psychological health, (5) The necessity of knowledge, skills, and tools to self-manage SSc and navigate COVID-19. Participants’ and research team members’ perspectives elucidated SPIN-CHAT Program benefits and how these benefits may have been realized. Results underscore the importance of social support from similar others, structure, and self-management to enhance psychological health during COVID-19. clinicaltrials.gov (NCT04335279)IMPLICATIONS FOR REHABILITATIONThe videoconference-based, supportive care SPIN-CHAT Program enhanced psychological health amongst individuals affected by systemic sclerosis.SPIN-CHAT Program participants and research team members shared that being around similar others, program structure, and self-management support were important and may have contributed to enhanced psychological health.Further efforts are required to explore experiences within supportive care programs to better understand if and how psychological health is impacted. The videoconference-based, supportive care SPIN-CHAT Program enhanced psychological health amongst individuals affected by systemic sclerosis. SPIN-CHAT Program participants and research team members shared that being around similar others, program structure, and self-management support were important and may have contributed to enhanced psychological health. Further efforts are required to explore experiences within supportive care programs to better understand if and how psychological health is impacted.

ObjectiveTo generate initial data on the frequency and effect of symptomatic adverse events (AEs) associated with rheumatoid arthritis (RA) drug therapy from the patient perspective.MethodsWe conducted an exploratory online survey asking patients with RA to indicate whether they currently or had ever experienced the 80 different symptomatic AEs included in the Patient-Reported Outcomes of the Common Terminology Criteria for Adverse Events (PRO-CTCAE). Results were summarized to report their frequency, and regression models were used to estimate their associations with RA medication use and overall bother.ResultsThe 560 patients who completed the survey and reported taking ≥ 1 RA medication (disease-modifying antirheumatic drugs [DMARDs], steroids, nonsteroidal antiinflammatory drugs [NSAIDs]), had a mean disease duration of 8 years, and were on a wide range of DMARDs. The number of symptomatic AEs experienced in the past 7 days was none (6%), 1–10 (28%), 11–20 (28%), and > 20 (38%). Overall, most participants reported that side effects bothered them somewhat (28%), quite a bit (24%), or very much (15%). In multivariable regression analyses, current prednisone and NSAID use were associated with the greatest number of current side effects (26 and 22, respectively). Many of the strongest associations between current symptomatic AEs and medication use aligned with known side effect profiles.ConclusionIn this exploratory online survey, patients with RA reported frequent symptomatic AEs with their medications that are bothersome. Further work is needed to develop and validate a measure for use in patients with rheumatic disease.

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Contains fulltext : 231157.pdf (Publisher’s version ) (Closed access) Objective: To support physical activity among people with systemic sclerosis (SSc; scleroderma), we sought to determine the (1) prevalence and importance of barriers and (2) likelihood of using possible facilitators. Methods: We invited 1,707 participants from an international SSc cohort to rate the (1) importance of 20 barriers (14 medical; 4 social or personal; 1 lifestyle; 1 environmental); and (2) likelihood of using 91 corresponding barrier-specific and 12 general facilitators. Results: Among 721 respondents, 13 barriers were experienced by ≥25% of participants, including 2 (fatigue, Raynaud's) rated 'important' or 'very important' by ≥50% of participants, 7 (joint stiffness and contractures, shortness of breath, gastrointestinal problems, difficulty grasping, pain, muscle weakness and mobility limitations, low motivation) by 26-50%, and 4 by 50% as 'likely' or 'very likely'. Conclusion: Medical-related physical activity barriers were common and considered important. Facilitators considered as most likely to be used involved adapting exercise, taking care of one’s body, keeping warm, and protecting skin. 11 p.

Background Systemic sclerosis (scleroderma; SSc) is a rare autoimmune connective tissue disease. We completed an initial feasibility trial of an online self-administered version of the Scleroderma Patient-centered Intervention Network Self-Management (SPIN-SELF) Program using the cohort multiple randomized controlled trial (RCT) design. Due to low intervention offer uptake, we will conduct a new feasibility trial with progression to full-scale trial, using a two-arm parallel, partially nested RCT design. The SPIN-SELF Program has also been revised to include facilitator-led videoconference group sessions in addition to online material. We will test the group-based intervention delivery format, then evaluate the effect of the SPIN-SELF Program on disease management self-efficacy (primary) and patient activation, social appearance anxiety, and functional health outcomes (secondary). Methods This study is a feasibility trial with progression to full-scale RCT, pending meeting pre-defined criteria, of the SPIN-SELF Program. Participants will be recruited from the ongoing SPIN Cohort (http://www.spinsclero.com/en/cohort) and via social media and partner patient organizations. Eligible participants must have SSc and low to moderate disease management self-efficacy (Self-Efficacy for Managing Chronic Disease (SEMCD) Scale score ≤ 7.0). Participants will be randomized (1:1 allocation) to the group-based SPIN-SELF Program or usual care for 3 months. The primary outcome in the full-scale trial will be disease management self-efficacy based on SEMCD Scale scores at 3 months post-randomization. Secondary outcomes include SEMCD scores 6 months post-randomization plus patient activation, social appearance anxiety, and functional health outcomes at 3 and 6 months post-randomization. We will include 40 participants to assess feasibility. At the end of the feasibility portion, stoppage criteria will be used to determine if the trial procedures or SPIN-SELF Program need important modifications, thereby requiring a re-set for the full-scale trial. Otherwise, the full-scale RCT will proceed, and outcome data from the feasibility portion will be utilized in the full-scale trial. In the full-scale RCT, 524 participants will be recruited. Discussion The SPIN-SELF Program may improve disease management self-efficacy, patient activation, social appearance anxiety, and functional health outcomes in people with SSc. SPIN works with partner patient organizations around the world to disseminate its programs free-of-charge. Trial registration ClinicalTrials.govNCT04246528. Registered on 27 January 2020

Objectives This study aimed to implement a patient-centred and evidence-based approach to develop a novel patient-reported outcome (PRO) instrument to measure fatigue in patients with SLE. Methods A three-step mixed methods psychometric (MMP) approach was followed. Steps comprised first draft item generation and review using interview data; evaluation and refinement of second draft items using mixed methods data, including interview and quantitative data from a phase 2 clinical study in SLE analysed using Rasch Measurement Theory (RMT) analysis; and evaluation of the final FATIGUE-PRO items using RMT and complementary Classical Test Theory (CTT) analyses. Guided by MMP criteria, a team of clinicians and outcome-measurement experts assessed evidence to inform instrument development. Results Step 1 culminated in 55 items (n = 39 patients interviewed). Their refinement in step 2 using mixed methods evidence led to the final FATIGUE-PRO instrument comprising 31 items across three scales of fatigue: physical fatigue (9 items), mental and cognitive fatigue (11 items) and susceptibility to fatigue (11 items). Qualitative (n = 43 patients) and quantitative (n = 106 patients) evidence strongly supported the scales’ content comprehensiveness and targeting, item quality and fit, conceptual uniqueness and appropriateness of the response scale. The FATIGUE-PRO further benefited from excellent reliability (RMT: 0.92–0.94 and CTT: 0.95–0.96) and supportive evidence of construct validity from assessments against other PROs. Conclusion The conceptual advances, comprehensive coverage and strong psychometric properties of the FATIGUE-PRO will significantly advance the measurement and management of fatigue in SLE, both in clinical trials and routine practice. Trial registration ClinicalTrials.gov (https://clinicaltrials.gov), NCT02804763

The Rheumatoid Arthritis Flare Questionnaire (RA-FQ) is a patient-reported measure of disease activity in RA. We estimated minimal and meaningful change from the perspective of RA patients, physicians, and using a disease activity index.Data were from 3- to 6-month visits of adults with early RA enrolled in the Canadian Early Arthritis Cohort. Participants completed the RA-FQ, the Patient Global Assessment of RA, and the Patient Global Change Impression at consecutive visits. Rheumatologists recorded joint counts and MD Global. Clinical Disease Activity Index (CDAI) scores were computed. We compared mean RA-FQ change across categories using patients, physicians, and CDAI anchors.The 808 adults were mostly white (84%) women (71%) with a mean age of 55 and moderate-high disease activity (85%) at enrollment. At V2, 79% of patients classified their RA as changed; 59% were better and 20% were worse. Patients reporting they were a lot worse had a mean RA-FQ increase of 8.9 points, whereas those who were a lot better had a -6.0 decrease. Minimal worsening and improvement were associated with a mean 4.7 and - 1.8 change in RA-FQ, respectively, while patients rating their RA unchanged had stable scores. Physician and CDAI classified more patients as worse than patients, and minimal and meaningful RA-FQ thresholds differed by group.Thresholds to identify meaningful change vary by anchor used. These data offer new evidence demonstrating robust psychometric properties of the RA-FQ and offer guidance about improvement or worsening, supporting its use in RA care, research, and decision-making.

The Edmonton Symptom Assessment System-revised (ESASr) is widely used in clinical oncology to screen for physical and emotional symptoms. The performance of the anxiety and depression items (ESASr-A and ESASr-D, respectively) as screening tools have not been evaluated in patients treated with renal replacement therapy. Kidney transplant recipients and patients on dialysis were recruited in Toronto. Patients were classified as having moderate/severe depression and anxiety symptoms using the established cut-off score of ≥ 10 on the Patient Health Questionnaire-9 (PHQ-9) and the General Anxiety Disorder-7 (GAD-7) questionnaires. This study included 931 participants; 62% male, mean age (SD) 55(16), and 52% White. All participants completed ESASr, however only 748 participants completed PHQ-9 and 769 participants completed GAD-7. Correlation between ESASr item scores and legacy scores were moderately strong (ESASr-D/PHQ-9: 0.61; ESASr-A/GAD-7: 0.64). We found good discrimination for moderate/severe depression and anxiety [area under the receiver operating characteristics curve (95% CI) ESASr-D 0.82(0.78–0.86); ESASr-A 0.87 (0.82, 0.92)]. The cut-off ≥ 2 for ESASr-D [Sensitivity = 0.76; Specificity = 0.77; Likelihood Ratio (LR) + = 3.29; LR − = 0.31] and ≥ 4 for ESASr-A (Sensitivity = 0.75; Specificity = 0.87; LR + = 5.76; LR − = 0.29) had the best combination of measurement characteristics. The identified ESASr-D and ESASr-A cut-off scores may be used to rule out patients without emotional distress with few false negatives. However, the low sensitivity identified in our analysis suggests that neither ESASr-D or ESASr-A are acceptable as standalone screening tools.

Summary Background No trials have tested multifaceted mental health interventions recommended by public health organisations during COVID-19. The objective of this trial was to evaluate the effect of the Scleroderma Patient-centered Intervention Network COVID-19 Home-isolation Activities Together (SPIN-CHAT) Program on anxiety symptoms and other mental health outcomes among people vulnerable during COVID-19 owing to a pre-existing medical condition. Methods The SPIN-CHAT Trial was a pragmatic, two-arm, parallel, partially nested, randomised, controlled trial (1:1 allocation to intervention or waitlist). Eligible participants with systemic sclerosis were recruited from the international SPIN COVID-19 Cohort. SPIN COVID-19 Cohort participants were eligible for the trial if they completed baseline measures and had at least mild anxiety symptoms, had not tested positive for COVID-19, and were not currently receiving mental health counselling. SPIN-CHAT is a 4-week (3 sessions per week) videoconference-based group intervention that provided education and practice with mental health coping strategies, and provided social support to reduce isolation. Groups included 6–10 participants. The primary outcome analysed in the intention-to-treat population was anxiety symptoms (PROMIS Anxiety 4a version 1.0) immediately post-intervention. This trial is registered with ClinicalTrials.gov, NCT04335279 and is complete. Findings Of participants who completed baseline measures between April 9, 2020, and April 27, 2020, 560 participants were eligible and 172 participants were randomly assigned to intervention (n=86) or waitlist (n=86). Mean age was 55·0 years (SD 11·4 years), 162 (94%) were women, and 136 (79%) identified as White. In intention-to-treat analyses, the intervention did not significantly reduce anxiety symptoms post-intervention (−1·57 points, 95% CI −3·59 to 0·45; standardised mean difference [SMD] −0·22 points) but reduced symptoms 6 weeks later (−2·36 points, 95% CI −4·56 to −0·16; SMD −0·31). Depression symptoms were significantly lower 6 weeks post-intervention (−1·64 points, 95% CI −2·91 to −0·37; SMD −0·31); no other secondary outcomes were significant. No adverse events were reported. Interpretation The intervention did not significantly improve anxiety symptoms or other mental health outcomes post-intervention. However, anxiety and depression symptoms were significantly lower 6 weeks later, potentially capturing the time it took for new skills and social support between intervention participants to affect mental health. Multi-faceted interventions such as SPIN-CHAT have potential to address mental health needs in vulnerable groups during COVID-19, yet uncertainty remains about effectiveness. Funding Canadian Institutes of Health Research (CIHR; VR4-172745, MS1-173066); McGill Interdisciplinary Initiative in Infection and Immunity Emergency COVID-19 Research Fund; Scleroderma Canada, made possible by an educational grant for patient support programming from Boehringer Ingelheim; the Scleroderma Society of Ontario; Scleroderma Manitoba; Scleroderma Atlantic; Scleroderma Australia; Scleroderma New South Wales; Scleroderma Victoria; Scleroderma Queensland; Scleroderma SASK; the Scleroderma Association of BC; and Sclerodermie Quebec.

ObjectiveTo describe the perspectives of patients with inflammatory arthritis (IA) on outcome domains of trials evaluating medication adherence interventions.MethodsAdult patients (≥ 18 yrs) with IA taking disease-modifying antirheumatic drugs from centers across Australia, Canada, and the Netherlands participated in 6 focus groups to discuss outcome domains that they consider important when participating in medication adherence trials. We analyzed the transcripts using inductive thematic analysis.ResultsOf the 38 participants, 23 (61%) had rheumatoid arthritis and 21 (55%) were female. The mean age was 57.3 ± (SD 15.0) years. Improved outcome domains that patients wanted from participating in an adherence trial were categorized into 5 types: medication adherence, adherence-related factors (supporting adherence; e.g., medication knowledge), pathophysiology (e.g., physical functioning), life impact (e.g., ability to work), and economic impact (e.g., productivity loss). Three overarching themes reflecting why these outcome domains matter to patients were identified: how taking medications could improve patients’ emotional and physical fitness to maintain their social function; how improving knowledge and confidence in self-management increases patients’ trust and motivation to take medications as agreed with minimal risk of harms; and how respect and reassurance, reflecting health care that values patients’ opinions and is sensitive to patients’ individual goals, could improve medication-taking behavior.ConclusionPatients value various outcome domains related to their overall well-being, confidence in medication use, and patient–healthcare provider relationships to be evaluated in future adherence trials.

Objectives To understand the perspectives of patients and rheumatologists for tapering DMARDs in RA. Methods Using semi-structured interview guides, we conducted individual interviews and focus groups with RA patients and rheumatologists, which were audiotaped and transcribed. We conducted a pragmatic thematic analysis to identify major themes, comparing and contrasting different views on DMARD tapering between patients and rheumatologists. Results We recruited 28 adult patients with RA (64% women; disease duration 1–54 y) and 23 rheumatologists (52% women). Attitudes across both groups towards tapering DMARDs were ambivalent, ranging from wary to enthusiastic. Both groups expressed concerns, particularly the inability to ‘recapture’ the same level of disease control, while also acknowledging potential positive outcomes such as reduced drug harms. Patient tapering perspectives (whether to and when) changed over time and commonly included non-biologic DMARDs. Patient preferences were influenced by lived experiences, side effects, previous tapering experiences, disease trajectory, remission duration and current life roles. Rheumatologists’ perspectives varied on timing and patient profile to initiate tapering, and were informed by both data and clinical experience. Patients expressed interest in shared decision-making (SDM) and close monitoring during tapering, with ready access to their health-care team if problems arose. Rheumatologists were generally open to tapering (not stopping), though sometimes only when requested by their patients. Conclusion The perspectives of patients and rheumatologists on tapering DMARDs in RA vary and evolve over time. Rheumatologists should periodically discuss DMARD tapering with patients as part of SDM, and ensure monitoring and flare management plans are in place.

BACKGROUND: Clinical trial design requires value judgements and understanding patient preferences may help inform these judgements, for example when prioritizing treatment candidates, designing complex interventions, selecting appropriate outcomes, determining clinically important thresholds, or weighting composite outcomes. Preference elicitation methods are quantitative approaches that can estimate patients' preferences to quantify the absolute or relative importance of outcomes or other attributes relevant to the decision context. We aimed to explore stakeholder perceptions of using preference elicitation methods to inform judgements when designing clinical trials in rheumatology.METHODS: We conducted 1-on-1 semi-structured interviews with patients with rheumatic diseases and rheumatology clinicians/researchers, recruited using purposive and snowball sampling. Participants were provided pre-interview materials, including a video and a document, to introduce the topic of preference elicitation methods and case examples of potential applications to clinical trials. Interviews were conducted via Zoom and were audio-recorded and transcribed. We used thematic analysis to analyze our data.RESULTS: We interviewed 17 patients and 9 clinicians/researchers, until data and inductive thematic saturation were achieved within each group. Themes were grouped into overall perceptions, barriers, and facilitators. Patients and clinicians/researchers generally agreed that preference elicitation studies can improve clinical trial design, but that many considerations are required around preference heterogeneity and feasibility. A key barrier identified was the additional resources and expertise required to measure and incorporate preferences effectively in trial design. Key facilitators included developing guidance on how to use preference elicitation to inform trial design, as well as the role of external decision-makers in developing such guidance, and the need to leverage the movement towards patient engagement in research to encourage including patient preferences when designing trials.CONCLUSION: Our findings allowed us to consider the potential applications of patient preferences in trial design according to stakeholders within rheumatology who are involved in the trial process. Future research should be conducted to develop comprehensive guidance on how to meaningfully include patient preferences when designing clinical trials in rheumatology. Doing so may have important downstream effects for shared decision-making, especially given the chronic nature of rheumatic diseases.

OBJECTIVE The Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) is validated for measuring fatigue in rheumatoid arthritis (RA). However, 10 of 13 FACIT-F items are identified as relevant to patients with RA. The Patient-Reported Outcomes Measurement Information System (PROMIS) uses an item response theory-calibrated T score metric. The PROMIS Fatigue item bank includes the FACIT-F items, enabling score conversion. The performance of converted PROMIS Fatigue scores has not been evaluated in RA populations or clinical trials. Our objective was to assess the performance of converted PROMIS Fatigue scores in 2 RA clinical trials of baricitinib. METHODS Crosswalk tables and pattern-scoring methods converted FACIT-F scores to PROMIS Fatigue for both the 13-item FACIT-F and the 10-item RA-optimized FACIT-F instrument, in 2 RA clinical trials evaluating baricitinib, RA-BEAM, and RA-BEACON. RA-BEAM patients had an inadequate response to methotrexate. RA-BEACON patients had an inadequate response or intolerance to ≥1 tumor necrosis factor inhibitor. Baricitinib was compared to all treatment arms via analysis of covariance on PROMIS Fatigue score conversions. RESULTS Baseline FACIT-F-derived PROMIS Fatigue scores reflected severe fatigue across treatment groups and were similar using different scoring methods. At week 24 in both studies, baricitinib was associated with clinically meaningful improvements in PROMIS Fatigue scores. PROMIS Fatigue scores were consistent for conversion methods and for the 13-item or 10-item FACIT-F. CONCLUSION All 4 conversion methods showed differentiation of active treatment compared with placebo from week 12, supporting the use of the PROMIS Fatigue and converting the 10-item FACIT-F to assess fatigue and demonstrate treatment benefit in RA clinical trials on a standardized metric.

Objectives Using patient-reported outcomes to inform clinical decision-making depends on knowing how to interpret scores. Patient-Reported Outcome Measurement Information System® (PROMIS®) instruments are increasingly used in rheumatology research and care, but there is little information available to guide interpretation of scores. We sought to identify thresholds and meaningful change for PROMIS Pain Interference and Fatigue scores from the perspective of RA patients and clinicians. Methods We developed patient vignettes using the PROMIS item banks representing a continuum of Pain Interference and Fatigue levels. During a series of face-to-face ‘bookmarking’ sessions, patients and clinicians identified thresholds for mild, moderate and severe levels of symptoms and identified change deemed meaningful for making treatment decisions. Results In general, patients selected higher cut points to demarcate thresholds than clinicians. Patients and clinicians generally identified changes of 5–10 points as representing meaningful change. The thresholds and meaningful change scores of patients were grounded in their lived experiences having RA, approach to self-management, and the impacts on function, roles and social participation. Conclusion Results offer new information about how both patients and clinicians view RA symptoms and functional impacts. Results suggest that patients and providers may use different strategies to define and interpret RA symptoms, and select different thresholds when describing symptoms as mild, moderate or severe. The magnitude of symptom change selected by patients and clinicians as being clinically meaningful in interpreting treatment efficacy and loss of response may be greater than levels determined by external anchor and statistical methods.

OBJECTIVE Higher self-reported disability (high Health Assessment Questionnaire [HAQ] score) has been associated with hospitalizations and mortality in established rheumatoid arthritis (RA), but associations in early RA are unknown. METHODS Patients with early RA (symptom duration

Objective To estimate the contributions of health-related quality of life domains to the patient global assessment of disease activity (PtGA) in rheumatoid arthritis (RA). Methods Data are drawn from baseline visits of 2 observational RA cohorts. Participants completed forms for patient-reported outcome measures, including PtGA and measures from the Patient-Reported Outcomes Measurement Information System, and clinical data were collected. Factor analysis was used to identify latent variables, and multivariable linear regression was used to estimate determinants of the PtGA. Results Patients were mostly female (81%), white (78%), and had established disease (mean ± SD 12.3 ± 10.7 years), with 62% in remission or having low disease activity. In cohort 1 (n = 196), the following 2 factors emerged: 1) daily function (moderate-to-strong [i.e., >|0.65|] loadings of physical function, pain interference, social participation, and fatigue, and weak [>0.35] loadings of sleep disturbance); and 2) emotional distress (strong loadings of depression and anxiety). In crude analysis, daily function explained up to 53% and emotional distress up to 20% of the variance in PtGA. In both cohorts, in adjusted analyses, daily function and, to a much lesser extent, swollen joint count independently predicted PtGA; age was inversely related to PtGA in cohort 1 only. Conclusion These findings suggest that in patients with RA, PtGA ratings largely reflect the extent to which patients feel they can function in everyday roles and are not impacted by mood. This suggests that higher than expected PtGA scores may offer an opportunity to discuss patient expectations regarding roles and activities and the impact of their RA symptoms on daily function.

Objective: To estimate, among people with multiple sclerosis, the extent to which a personally tailored exercise programme (MSTEP©) resulted in greater improvements in exercise capacity and related outcomes over 12 months in comparison with general exercise guidelines. Design: Two-group randomized trial. Subjects: Ambulatory and sedentary. Interventions: MSTEP©, a personally adapted exercise regimen done on most days including two days of high intensity exercise; guidelines recommending 30 minutes of moderate intensity aerobic and strength training two times per week. Main measures: Primary outcome was peak oxygen consumption (VO2peak) at 12 months; secondary outcomes were composite measures of physical function, fatigue, and health-related quality of life. Results: In total, 137 people were randomized, 66 were lost over 12 months leaving 71 with outcome data, 34 in MSTEP© group, and 37 in the Guideline group. Exercise enjoyment and confidence and exercise-induced fatigue predicted retention. There were no differences between groups on the proportion making a 10% increase in VO2peak (27.1% MSTEP© vs 29.6% Guidelines; OR: 0.83; 95% CI: 0.23–3.08) by the 12 month assessment. The effect on fatigue was larger in the MSTEP© group than the Guideline groups (OR: 1.59; 95% CI: 0.93–2.74), the effect on physical function was more modest (OR: 1.35; 95% CI: 0.80–2.25), and null for health-related quality of life outcomes. Conclusions: The disappointing exercise retention suggests that people with multiple sclerosis may not consider exercise important to their brain health. Either type of exercise resulted in stable exercise capacity over 1 year in those sticking with the programme.

Objective To evaluate the impact of integrating patient-reported outcomes (PROs) into routine clinics, from the perspective of patients with RA, clinicians and other staff. Methods We conducted a prospective cohort study using a mixed methods sequential explanatory design at an academic arthritis clinic. RA patients completed selected Patient-Reported Outcomes Measurement Information System measures on tablets in the waiting room. Results were immediately available to discuss during the visit. Post-visit surveys with patients and physicians evaluated topics discussed and their impact on decision making; patients rated confidence in treatment. Focus groups or interviews with patients, treating rheumatologists and clinic staff were conducted to understand perspectives and experiences. Results Some 196 patients and 20 rheumatologists completed post-visit surveys at 816 and 806 visits, respectively. Focus groups were conducted with 24 patients, 10 rheumatologists and 4 research/clinic staff. PROs influenced medical decision-making and RA treatment changes (38 and 18% of visits, respectively). Patients reported very high satisfaction and treatment confidence. Impact on clinical workflow was minimal after a period of initial adjustment. PROs were valued by patients and physicians, and provided new insight into how patients felt and functioned over time. Reviewing results together improved communication, and facilitated patient-centred care, shared decision making, and the identification of new symptoms and contributing psychosocial/behavioural factors. Conclusion PRO use at RA visits was feasible, increased understanding of how disease affects how patients feel and function, facilitated shared decision-making, and was associated with high patient satisfaction and treatment confidence.

Objective.The Outcome Measures in Rheumatology (OMERACT) Shared Decision Making (SDM) Working Group aims to determine the core outcome domain set for measuring the effectiveness of SDM interventions in rheumatology trials.Methods.A white paper was developed to clarify the draft core domain set. It was then used to prepare for interviews to investigate reasons for lack of consensus on it and to suggest further improvements.Results.OMERACT scientists/clinicians (n = 13) and patients (n = 10) suggested limiting the core domain set to outcome domains, removing process domains, and clarifying remaining domains.Conclusion.A revised core domain set will undergo further consensus-building.

To examine the validity and reliability of the PROMIS Sleep Disturbance Item Bank Computer Adaptive Test (PROMIS-SD CAT) in patients on renal replacement therapy, using the Insomnia Severity Index as the primary legacy instrument.Cross-sectional sample of adults on renal replacement therapy completed PROMIS-SD CAT, Insomnia Severity Index, General Anxiety Disorder-7, Edmonton Symptom Assessment System-revised Renal, SF-12 questionnaires. Construct validity was confirmed by associations with legacy instrument scores and measures of emotional wellbeing. Test-retest and scale reliability were assessed using intraclass correlation coefficient and standard errors of measurement, respectively. Discrimination was assessed using receiver-operating characteristic curve analysis.Among 217 participants (133 kidney transplant recipients, 84 on dialysis), 15% had moderate/severe sleep disturbance (Insomnia Severity Index ≥15). PROMIS-SD CAT exhibited good test-retest (intraclass correlation coefficient = 0.89, 95% confidence interval (0.83-0.93)) and scale (0.9 for T-scores 34-73) reliability. PROMIS-SD CAT T-scores were strongly correlated with Insomnia Severity Index (rho = 0.85, 95% confidence interval (0.81-0.88)), Edmonton Symptom Assessment System-revised Renal Sleep Item (rho = 0.75, 95% confidence interval (0.69-0.81)) and moderately with General Anxiety Disorder-7 scores and SF-12 Mental Component Summary scores (p 0.001 for all). PROMIS-SD CAT demonstrated outstanding discrimination for moderate/severe insomnia (defined as Insomnia Severity Index ≥15) (AUROC 0.93, 95% confidence interval (0.88, 0.98)). PROMIS-SD CAT and Insomnia Severity Index measurement characteristics were qualitatively similar between patients on dialysis and kidney transplant recipients.These results support PROMIS-SD CAT's validity and reliability for research and as potential screening tool among patients on renal replacement therapy.

Background Research on adherence interventions in rheumatology is limited by methodological issues, particularly heterogeneous outcomes. We aimed to describe researchers’ experiences with conducting interventional studies targeting medication adherence in rheumatology and their perspectives on establishing core outcomes. Methods Semi-structured interviews using audio conference were conducted with researchers who had conducted an adherence study of any design in the past 10 years. Data collection and thematic analysis were performed iteratively, until saturation. Results We interviewed 13 researchers, most of whom worked in academia and specialized in epidemiology and/or health services research. We identified three themes: 1) improving measurement of adherence (considering all phases of adherence, using appropriate and relevant measures, and establishing clinically meaningful thresholds); 2) challenges in designing and appraising adherence intervention studies (considering the confusion over a plethora of outcomes, difficulties with powering studies to demonstrate meaningful changes, and suboptimal descriptions of adherence interventions in published studies); and 3) advancing outcome assessment in adherence intervention studies (capturing rationale for developing a core domain set as well as recommendations and anticipated challenges by participants). Conclusions Uniquely gathering perspectives from international adherence researchers, our findings led to researcher-informed recommendations for improving adherence research including specifying the targeted adherence phase in designing interventions and studies and providing a glossary of terms to promote consistency in reporting. We also identified recommendations for developing a core domain set for interventional studies targeting medication adherence including involvement of patients, clinicians, and other stakeholders and methodological and practical considerations to establish rigor and support uptake.

Contains fulltext : 237267.pdf (Publisher’s version ) (Closed access) Background: Pain is one of the most common symptoms affecting patients with systemic sclerosis; however, little is known about the relationship between self-efficacy and pain and changes in pain over time. Objectives The purpose of this study was to describe the relationships between self-efficacy and pain in patients with systemic sclerosis, as well as determine whether changes in self-efficacy mediate changes in pain. Methods: A prospective longitudinal study was conducted using data from the Scleroderma Patient-Centered Intervention Network Cohort. The baseline sample included 1,903 adults, with a trajectory subsample of 427 who completed 3-month assessments across 3 years. Hierarchical (sequential) forward multivariable regression, covarying for participant characteristics, was conducted to determine the association between self-efficacy and patient characteristics on pain outcomes. Trajectory models, covarying for participant characteristics, were used to examine changes in self-efficacy and pain outcomes across time and whether self-efficacy mediated the pain trajectories. Results: Mean time since diagnosis was 9.5 years, with 39.2% diagnosed with diffuse cutaneous systemic sclerosis. Greater self-efficacy was associated with less pain interference and intensity. Increasing age, female gender, finger ulcers, and small joint contractures were related to greater pain interference and intensity. Esophageal gastrointestinal symptoms were associated with more pain interference. Self-efficacy and pain trajectories remained stable across time, and self-efficacy did not mediate the pain trajectories. Discussion: This study identified self-efficacy, age, gender, finger ulcers, small joint contractures, and esophageal gastrointestinal symptoms as important correlates associated with pain in patients with systemic sclerosis. In addition, this study found that self-efficacy and pain outcomes remained stable over time, providing important insights into the longitudinal pain experiences of patients with systemic sclerosis. 10 p.

Objective: To gain consensus on the Outcome Measures in Rheumatology (OMERACT) core domain set for rheumatology trials of shared decision making (SDM) interventions.Methods: The process followed the OMERACT Filter 2.1 methodology, and used consensus-building methods, with patients involved since the inception. After developing the draft core domain set in previous research, we conducted five steps: (i) improving the draft core domain set; (ii) developing and disseminating whiteboard videos to promote its understanding; (iii) conducting an electronic survey to gather feedback on the draft core domain set; (iv) finalizing the core domain set and developing summaries, a plenary session video and discussion boards to promote its understanding; and (v) conducting virtual workshops with voting to endorse the core domain set.Results: A total of 167 participants from 28 countries answered the survey (62% were patients/caregivers). Most participants rated domains as relevant (81%-95%) and clear (82%-93%). A total of 149 participants (n = 48 patients/caregivers, 101 clinicians/researchers) participated in virtual workshops and voted on the proposed core domain set which received endorsement by 95%. Endorsed domains are: 1-Knowledge of options, their potential benefits and harms; 2-Chosen option aligned with each patient's values and preferences; 3-Confidence in the chosen option; 4-Satisfaction with the decision-making process; 5-Adherence to the chosen option and 6-Potential negative consequences of the SDM intervention.Conclusion: We achieved consensus among an international group of stakeholders on the OMERACT core domain set for rheumatology trials of SDM interventions. Future research will develop the Core Outcome Measurement Set.Clinical significance: Prior to this study, there had been no consensus on the OMERACT core domain set for SDM interventions. The current study shows that the OMERACT core domain set achieved a high level of endorsement by key stakeholders, including patients/caregivers, clinicians and researchers.(c) 2021 Elsevier Inc. All rights reserved.

Background Fatigue, a common symptom of rheumatoid arthritis (RA), is detrimental to health-related quality of life (HRQoL). We evaluated the impact of tofacitinib on fatigue, sleep, and HRQoL and explored associations between fatigue, related patient-reported outcomes (PROs), and disease activity in RA patients. Methods This post hoc analysis pooled data from three Phase 3 studies of tofacitinib (ORAL Scan; ORAL Standard; ORAL Sync) in RA patients. Patients received tofacitinib 5 or 10 mg twice daily, placebo, or adalimumab (active control; ORAL Standard only, not powered for superiority) with conventional synthetic disease-modifying antirheumatic drugs. Assessed through Month (M)12 were changes from baseline in disease activity, Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F), Medical Outcomes Study Sleep scale (MOS-SS), and Short Form-36 Health Survey (SF-36) composite/domain scores, and proportions of patients reporting improvements from baseline in FACIT-F total and SF-36 domain scores ≥ minimum clinically important differences (MCIDs) or ≥ population normative values. Pearson correlations examined associations among PROs at M6. Treatment comparisons were exploratory, with p < 0.05 considered nominally significant. Results Generally, active treatment led to significant improvements from baseline in FACIT-F total, and MOS-SS and SF-36 composite/domain scores vs placebo, observed by M1 and maintained through M6 (last placebo-controlled time point). Through M6, more patients achieved improvements from baseline ≥ MCID and achieved scores ≥ population normative values in FACIT-F total and SF-36 domain scores with tofacitinib vs placebo. Through M12, some nominally significant improvements with tofacitinib vs adalimumab were observed. With active treatment at M6, FACIT-F scores were moderately (0.40–0.59) to highly (≥ 0.60) correlated with SF-36 composite/domain scores (particularly vitality), moderately correlated with most MOS-SS domain scores, and highly correlated with MOS-SS Sleep Problems Index I scores. Disease activity correlations were moderate with FACIT-F scores and low (0.20–0.39) to moderate with SF-36 general health domain/composite scores. Conclusion Tofacitinib and adalimumab generally conferred significant, clinically meaningful improvements in fatigue, sleep, and HRQoL (including vitality) vs placebo through M6, with improvements maintained to M12. M6 correlations between FACIT-F, PROs of sleep, HRQoL, and disease activity underscore the interrelatedness of multiple PROs and disease activity in RA. Trial registration ClinicalTrials.govNCT00847613 (registered: February 19, 2009); NCT00853385 (registered: March 2, 2009); NCT00856544 (registered: March 5, 2009).

OBJECTIVE Meditation is a stress-reduction and contemplative technique that can improve emotional distress in people with chronic disease and may be especially beneficial for patients with rheumatic diseases. However, patient access to in-person programs is challenging. The goal of this pilot study was to evaluate the feasibility/acceptability associated with physician-directed use of a widely available smartphone application (app), Calm©. METHODS In this single-arm, pre-post intervention study with recruitment over a 10-month period, adults with rheumatic disease were asked to use the app for ≥5 min/day for 30 days. Participants completed sociodemographic surveys and validated health related quality of life (HRQL) questionnaires from the Patient Reported Outcomes Information System (PROMIS) and NIH Toolbox at baseline and 30-days. RESULTS Thirty-five participants who were mostly well-educated (66% ≥college degree) females (91%) with a mean age of 50 (SD 13) completed baseline questionnaires; 18 participants completed post-study questionnaires ("full completers"). Full completers had higher baseline stress, anxiety, pain, and patient global assessment scores (p's

OBJECTIVE To describe patterns of glucocorticoid use in a large real-world cohort with early rheumatoid arthritis (RA) and assess the impact on disease activity and treatment. METHODS Data are from adults with new RA (≤1 year) recruited to the Canadian Early Arthritis Cohort (CATCH) and are stratified on the basis of whether a person was prescribed oral glucocorticoids within 3 months of study entry. Disease activity was compared over 24 months. Mixed-effects logistic regression was used for adjusted odds ratios (aORs) of escalation to biologics separately for 12 and 24 months, with random effects terms to account for prescribing patterns clustering by study site. RESULTS Among 1891 persons, 30% received oral steroids. Users were older, were less often employed, and had shorter disease duration and higher disease activity. Disease activity improved over time, with early glucocorticoid users starting at higher levels of disease activity. Participants with early oral glucocorticoids were more likely to be on a biologic at 12 months (aOR = 2.4; 95% confidence interval [CI], 1.5-3.7) and 24 months (aOR = 1.9; 95% CI, 1.3-3.0). Despite Canadian clinical practice guidelines to limit corticosteroid use to short-term or 'bridge' therapy, 30% of patients who used oral glucocorticoids still used them 2 years later. CONCLUSION Early steroids were prescribed sparingly in CATCH and were often indicative of more active baseline disease as well as the need for progression to biologics.

We appreciate the interest in our manuscript concerning the Health Assessment Questionnaire disability index (HAQ) in an early rheumatoid arthritis incident cohort (the CATCH cohort) which predicted all-cause mortality (1). We will clarify queries raised in letters to the editor (2-4).

Objective.The OMERACT-Adherence meeting was convened to discuss the conceptual and methodological challenges in developing a core domain set (Adherence-CDS) for trials of interventions for medication adherence in rheumatology.Methods.Forty participants from nine countries participated.Results.Four ideas emerged: for adherence trials, the Adherence-CDS could include adherence and the condition-specific CDS; many factors affect adherence and are intervention targets, contextual factors, or outcome domains; adherence is a critical factor in drug trials; and standardized adherence measures are needed.Conclusion.Despite the challenges, the meeting clarified an approach to developing an Adherence-CDS that complements existing OMERACT work and methodology.