"I couldn't carry on taking a drug like that": A qualitative study of patient perspectives on side effects from rheumatology drugs.

OBJECTIVES: There is growing interest in collecting outcome information directly from patients in clinical trials. This study evaluates what patients with rheumatic and musculoskeletal diseases (RMDs) consider important to know about symptomatic side effects they may experience from a new prescription drug. METHODS: Patients with inflammatory arthritis, who had one or more prescribed drugs for their disease for at least 12 months, participated in focus groups and individual interviews. Discussions were analysed using reflexive thematic analysis. RESULTS: We conducted seven focus groups with 34 participants across three continents. We found four overarching and two underpinning themes. The 'impact on life' was connected to participants 'daily life', 'family life', 'work life', and 'social life'. In 'psychological and physical aspects' participants described 'limitation to physical function', 'emotional dysregulation' and 'an overall mental state'. Extra tests, hospital visits and payment for medication were considered a 'time, energy and financial burden' of side effects. Participants explained important measurement issues to be 'severity', 'frequency', and 'duration'. Underpinning these issues, participants evaluated the 'benefit-harm-balance' which includes 'the cumulative burden' of having several side effects and the persistence of side effects over time. CONCLUSIONS: In treatment for RMDs, there seems to be an urgent need for feasible measures of patient-reported bother (impact on life and cumulative burden) from side effects and the benefit-harm-balance. These findings contribute new evidence in support of a target domain-an outcome that represents the patient voice evaluating the symptomatic treatment-related side effects for people with RMDs enrolled in clinical trials.

Factors associated with physical function among people with systemic sclerosis: a SPIN cohort cross-sectional study.

OBJECTIVES: To compare physical function in systemic sclerosis (SSc, scleroderma) to general population normative data and identify associated factors. METHODS: Scleroderma Patient-centered Intervention Network Cohort participants completed the Physical Function domain of the Patient-Reported Outcomes Measurement Information System Version 2 upon enrolment. Multivariable linear regression was used to assess associations of sociodemographic, lifestyle, and disease-related variables. RESULTS: Among 2,385 participants, mean physical function T-score (43.7, SD = 8.9) was ∼2/3 of a standard deviation (SD) below the US general population (mean = 50, SD = 10). Factors associated in multivariable analysis included older age (-0.74 points per SD years, 95% CI -0.78 to -1.08), female sex (-1.35, -2.37 to -0.34), fewer years of education (-0.41 points per SD in years, -0.75 to -0.07), being single, divorced, or widowed (-0.76, -1.48 to -0.03), smoking (-3.14, -4.42 to -1.85), alcohol consumption (0.79 points per SD drinks per week, 0.45-1.14), BMI (-1.41 points per SD, -1.75 to -1.07), diffuse subtype (-1.43, -2.23 to -0.62), gastrointestinal involvement (-2.58, -3.53 to -1.62), digital ulcers (-1.96, -2.94 to -0.98), moderate (-1.94, -2.94 to -0.93) and severe (-1.76, -3.24 to -0.28) small joint contractures, moderate (-2.10, -3.44 to -0.76) and severe (-2.54, -4.64 to -0.44) large joint contractures, interstitial lung disease (-1.52, -2.27 to -0.77), pulmonary arterial hypertension (-3.72, -4.91 to -2.52), rheumatoid arthritis (-2.10, -3.64 to -0.56) and idiopathic inflammatory myositis (-2.10, -3.63 to -0.56). CONCLUSION: Physical function is impaired for many individuals with SSc and associated with multiple disease factors.

Translation and linguistic validation of 24 PROMIS item banks into French.

PURPOSE: The Patient-Reported Outcome Measurement Information System (PROMIS®) was developed to provide reliable, valid, and normed item banks to measure health. The item banks provide standardized scores on a common metric allowing for individualized, brief assessment (computerized adaptive tests), short forms (e.g. heart failure specific), or profile assessments (e.g. PROMIS-29). The objective of this study was to translate and linguistically validate 24 PROMIS adult item banks into French and highlight cultural nuances arising during the translation process. METHODS: We used the FACIT translation methodology. Forward translation into French by two native French-speaking translators was followed by reconciliation by a third native French-speaking translator. A native English-speaking translator fluent in French then completed a back translation of the reconciled version from French into English. Three independent reviews by bilingual translators were completed to assess the clarity and consistency of terminology and equivalency across the English source and French translations. Reconciled versions were evaluated in cognitive interviews for conceptual and linguistic equivalence. RESULTS: Twenty-four adult item banks were translated: 12 mental health, 10 physical health, and two social health. Interview data revealed that 577 items of the 590 items translated required no revisions. Conceptual and linguistic differences were evident for 11 items that required iterations to improve conceptual equivalence and two items were revised to accurately reflect the English source. CONCLUSION: French translations of 24 item banks were created for routine clinical use and research. Initial translation supported conceptual equivalence and comprehensibility. Next steps will include validation of the item banks.

Moderators of Loneliness Trajectories in People with Systemic Sclerosis During the COVID-19 Pandemic: A SPIN COVID-19 Cohort Longitudinal Study.

BACKGROUND: Many individuals with systemic sclerosis (SSc) are at heightened risk for COVID-19 related morbidity and isolation due to interstitial lung disease, frailty, and immunosuppressant use. Minimal research has explored loneliness predictors in individuals with chronic illnesses during COVID-19. This study evaluated moderators of loneliness trajectories in individuals with SSc during COVID-19. METHODS: Longitudinal data were analyzed across 30 timepoints from April 2020 to May 2022 from 775 adults in the Scleroderma Patient-centered Intervention Network (SPIN) COVID-19 Cohort. Hierarchical linear modeling evaluated cross-level moderators of loneliness trajectories, including marital status, baseline number of household members, number of virtual or telephone one-on-one or virtual group conversations, number of hours spent enjoying in-person household conversations or activities, and satisfaction with quality of in-person household conversations (all in the past week). Level-1 moderation analyses assessed effects of conversation, activity, and satisfaction means and slopes over time. RESULTS: Baseline values were not statistically significant moderators of loneliness trajectories. Higher mean (averaged over time) virtual or telephone one-on-one and in-person household conversations, in-person household activity, and in-person household conversation satisfaction were associated with lower loneliness trajectories (ps < .05). The relationship between in-person household conversation satisfaction and loneliness trajectory was statistically significantly but minimally attenuated over time (p < .001). CONCLUSIONS: For people with SSc, higher mean conversation, activity, and satisfaction variables were associated with lower levels of loneliness during the pandemic, but changes in these social variables were generally not predictive of changes in loneliness.

Having More Tender Than Swollen Joints Is Associated With Worse Patient-Reported Outcomes in Patients With Early RA.

BACKGROUND/OBJECTIVE: In patients with rheumatoid arthritis (RA), high tender-swollen joint differences (TSJDs) have been associated with worse outcomes. A better understanding of the phenotype and impact of high TSJD on patient-reported outcomes (PROs) in early RA may lead to earlier personalized treatment targeting domains that are important to patients today. Our objectives were to evaluate the impact of TSJD on updated PROs in patients with early RA over 1 year and to determine differences in associations by joint size. METHODS: This longitudinal cohort study followed patients with active, early RA enrolled in the Canadian Early Arthritis Cohort between 2016 and 2022, who completed clinical assessments and PROMIS-29 measures over 1 year. Twenty-eight joint counts were performed and TSJDs calculated. Adjusted associations between TSJD and PROMIS-29 scores were estimated using separate linear-mixed models. Separate analyses of large versus small-joint TJSDs were performed. RESULTS: Patients with early RA (n = 547; 70% female; mean [SD] age, 56 [15] years; mean [SD] symptom duration, 5.3 [2.9] months) were evaluated. A 1-point increase in TSJD was significantly associated with worse PROMIS T-scores in all domains: physical function (adjusted regression coefficient, -0.27; 95% confidence interval [CI], -0.39, -0.15), social participation (adjusted regression coefficient, -0.34; 95% CI, -0.50, -0.19), pain interference (adjusted regression coefficient, 0.49; 95% CI, 0.35, 0.64), sleep problems (adjusted regression coefficient, 0.29; 95% CI, 0.16, 0.43), fatigue (adjusted regression coefficient, 0.34; 95% CI, 0.18, 0.50), anxiety (adjusted regression coefficient, 0.23; 95% CI, 0.08, 0.38), and depression (adjusted regression coefficient, 0.20; 95% CI, 0.06, 0.35). Large-joint TSJD was associated with markedly worse PROs compared with small-joint TSJD. CONCLUSIONS: Elevated TSJD is associated with worse PROs particularly pain interference, social participation, and fatigue. Patients with more tender than swollen joints, especially large joints, may benefit from earlier, targeted therapeutic interventions.

Assessing Fatigue in Patients Receiving Kidney Replacement Therapy Using PROMIS Computer Adaptive Testing.

RATIONALE & OBJECTIVE: Fatigue is a debilitating symptom for many patients receiving kidney replacement therapy (KRT). Patient-reported outcome measures can help clinicians identify and manage fatigue efficiently. We assessed the measurement characteristics of the Patient Reported Outcome Measurement Information System (PROMIS)-Fatigue Computer Adaptive Test (PROMIS-F CAT) in patients receiving KRT using the previously validated Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) questionnaire. STUDY DESIGN: Cross-sectional study. SETTING & PARTICIPANTS: 198 adults treated with dialysis or recipients of a kidney transplant in Toronto, Canada. PREDICTORS: Demographic data, FACIT-F scores, KRT type. OUTCOME: Measurement properties of PROMIS-F CAT T scores. ANALYTICAL APPROACH: Reliability and test-retest reliability were assessed using standard errors of measurement and intraclass correlation coefficient (ICC), respectively. Construct validity was assessed using correlation and comparisons across predefined groups expected to have different levels of fatigue. Receiver operating characteristic (ROC) curves were used to assess the discrimination of PROMIS-F CAT, with clinically relevant fatigue defined by a FACIT-F score of≤30. RESULTS: Of the 198 participants, 57% were male, the mean±SD age was 57±14 years; 65% had received a kidney transplant. Based on the FACIT-F score, 47 patients (24%) had clinically relevant fatigue. PROMIS-F CAT and FACIT-F were strongly correlated (ρ =-0.80, P<0.001). PROMIS-F CAT had excellent reliability (>0.90 for 98% of sample), and good test-retest reliability (ICC=0.85). The ROC analysis demonstrated outstanding discrimination (area under ROC=0.93 [95%, CI 0.89-0.97]). A PROMIS-F CAT cutoff score of≥59 accurately identified most patients with clinically relevant fatigue (sensitivity=0.83; specificity=0.91). LIMITATIONS: A convenience sample of clinically stable patients. FACIT-F items are a part of the PROMIS-F item bank, although there was minimal overlap with only 4 FACIT-F items completed in PROMIS-F CAT. CONCLUSIONS: PROMIS-F CAT has robust measurement properties with low question burden to assess fatigue among patients with KRT.

Mental health symptoms in scleroderma during COVID-19: a Scleroderma Patient-centred Intervention Network (SPIN) cohort longitudinal study.

OBJECTIVES: People with systemic sclerosis (SSc) are vulnerable in COVID-19 and face challenges related to shifting COVID-19 risk and protective restrictions. We evaluated mental health symptom trajectories in people with SSc through March 2022. METHODS: The longitudinal Scleroderma Patient-centred Intervention Network (SPIN) COVID-19 cohort was launched in April 2020 and included participants from the ongoing SPIN Cohort and external enrolees. Analyses included estimated means with 95% CIs for anxiety and depression symptoms pre-COVID-19 for ongoing SPIN Cohort participants and anxiety, depression, loneliness, and fear of COVID-19 for all participants across 28 COVID-19 assessments up to March 2022. We conducted sensitivity analyse including estimating trajectories using only responses from participants who completed >90% of items for ≥21 of 28 possible assessments ("completers") and stratified analyses for all outcomes by sex, age, country, and SSc subtype. RESULTS: Anxiety symptoms increased in early 2020 but returned to pre-COVID-19 levels by mid-2020 and remained stable through March 2022. Depression symptoms did not initially change but were slightly lower by mid-2020 compared to pre-COVID-19 and were stable through March 2022. COVID-19 fear started high and decreased. Loneliness did not change across the pandemic. Results were similar for completers and for all subgroups. CONCLUSIONS: People with SSc continue to face COVID-19 challenges related to ongoing risk, the opening of societies, and removal of protective restrictions. People with SSc, in aggregate, appear to be weathering the pandemic well, but health care providers should be mindful that some individuals may benefit from mental health support.

"From Where I Stand": using multiple anchors yields different benchmarks for meaningful improvement and worsening in the rheumatoid arthritis flare questionnaire (RA-FQ).

PURPOSE: 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. METHODS: 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. RESULTS: 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. CONCLUSION: 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 FATIGUE-PRO: a new patient-reported outcome instrument to quantify fatigue in patients affected by systemic lupus erythematosus.

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.

Patient and rheumatologist perspectives on tapering DMARDs in rheumatoid arthritis: a qualitative study.

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.

Patients and clinicians define symptom levels and meaningful change for PROMIS pain interference and fatigue in RA using bookmarking.

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.

Pain and Self-Efficacy Among Patients With Systemic Sclerosis: A Scleroderma Patient-Centered Intervention Network Cohort Study.

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.

Patient-reported outcomes in RA care improve patient communication, decision-making, satisfaction and confidence: qualitative results.

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.

Anxiety impacts rheumatoid arthritis symptoms and health-related quality of life even at low levels.

OBJECTIVES: We explored the burden of symptoms of anxiety and depression on health-related quality of life (HRQL) in patients with rheumatoid arthritis (RA). METHODS: Adults with RA participating in an observational cohort completed PROMIS tests of depression, anxiety, fatigue, physical function (PF), pain interference (PI), sleep disturbance, and participation in social roles and activities at the baseline visit. Clinical measures of disease status were also obtained. We used ANOVA and partial correlation adjusting for the swollen joint count (SJC) to examine associations of anxiety and depression with other aspects of HRQL. Mild and moderate-severe anxiety were defined as T-scores ≥55.4 and ≥ 62.3 and mild and moderate-severe depression was defined as ≥52.5 and ≥58.6 based on previous validated clinical thresholds. Multivariable linear regression (MVR) was used to identify predictors of emotional distress with a subset analysis of those in remission/low disease activity. RESULTS: Of 196 RA participants, 18% had mild anxiety, 9% had moderate-severe anxiety, 18% had mild depression, and 14% had moderate-severe depression symptoms. Anxiety and depression scores were associated with significantly worse mean scores across HRQL domains (p <0.05). In MVR, depression (ß=0.75, p<0.001), PF (ß=0.14, p=0.024) and fatigue (ß=0.15, p=0.015) predicted higher anxiety levels, whereas only anxiety predicted higher depression levels (ß=0.70, p=<0.001). In subset analysis, PF no longer predicted higher anxiety levels. CONCLUSIONS: Emotional distress is common in RA, even when disease is well controlled, with considerable impacts on other aspects of HRQL even at mild levels.

Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS)-57 and -29 item short forms among kidney transplant recipients.

OBJECTIVE: The Patient-Reported Outcomes Measurement Information System (PROMIS) aims to address the lack of generalizable and universal measure of patient-reported outcomes to assess health-related quality of life. It has not been validated for patients with chronic kidney disease. We aim to validate the PROMIS-57 and PROMIS-29 questionnaires among kidney transplant recipients. METHODS: A cross-sectional sample of stable kidney transplant recipients was recruited. Each participant completed PROMIS-57, a 57-question instrument covering seven domains-physical function, anxiety, depression, fatigue, pain, sleep disturbance, and social functioning-alongside validated legacy questionnaires [Patient Health Questionnaire (PHQ9), General Anxiety Disorder (GAD7), Edmonton Symptom Assessment Scale revised (ESASr), and Kidney Disease Quality of Life (KDQoL-36)]. PROMIS-29, a 29-question instrument, is nested within PROMIS-57 and measures the same domains. Structural validity of PROMIS was assessed with confirmatory factor analysis, reported using the Comparative Fit Index (CFI). Construct validity was assessed with known-groups comparisons. Internal consistency was evaluated with Cronbach's α and convergent validity was assessed with Spearman's Rho. Test-retest reliability was assessed through the intraclass correlation coefficient (ICC). RESULTS: Mean (± SD) age of the 177 participants was 50 (± 17), 57% were male and 55% Caucasian. Internal consistency of each domain was high (Cronbach's α > 0.88). Confirmatory factor analysis showed good structural validity for most domains (CFI > 0.95, RMSEA < 0.05). Test-retest reliability indicated good agreement (ICC > 0.6). Known-groups comparisons by clinical and socio-demographic differences were found as hypothesized. CONCLUSIONS: Our results provide evidence that PROMIS-57 and PROMIS-29 are highly reliable and valid instruments among kidney transplant recipients. We propose it as a valuable tool to assess important domains of the illness experience.

The Role of Weight Management in the Treatment of Adult Obstructive Sleep Apnea. An Official American Thoracic Society Clinical Practice Guideline.

BACKGROUND: Overweight/obesity is a common, reversible risk factor for obstructive sleep apnea severity (OSA). The purpose of this guideline is to provide evidence-based recommendations for the management of overweight/obesity in patients with OSA. METHODS: The Grading of Recommendations, Assessment, Development and Evaluation approach was used to evaluate the literature. Clinical recommendations were formulated by a panel of pulmonary, sleep medicine, weight management, and behavioral science specialists. RESULTS: Behavioral, pharmacological, and surgical treatments promote weight loss and can reduce OSA severity, reverse common comorbidities, and improve quality of life, although published studies have methodological limitations. After considering the quality of evidence, feasibility, and acceptability of these interventions, the panel made a strong recommendation that patients with OSA who are overweight or obese be treated with comprehensive lifestyle intervention consisting of 1) a reduced-calorie diet, 2) exercise or increased physical activity, and 3) behavioral guidance. Conditional recommendations were made regarding reduced-calorie diet and exercise/increased physical activity as separate management tools. Pharmacological therapy and bariatric surgery are appropriate for selected patients who require further assistance with weight loss. CONCLUSIONS: Weight-loss interventions, especially comprehensive lifestyle interventions, are associated with improvements in OSA severity, cardiometabolic comorbidities, and quality of life. The American Thoracic Society recommends that clinicians regularly assess weight and incorporate weight management strategies that are tailored to individual patient preferences into the routine treatment of adult patients with OSA who are overweight or obese.

The Scleroderma Patient-Centered Intervention Network Cohort: baseline clinical features and comparison with other large scleroderma cohorts.

Objectives: The Scleroderma Patient-centered Intervention Network (SPIN) Cohort is a web-based cohort designed to collect patient-reported outcomes at regular intervals as a framework for conducting trials of psychosocial, educational, self-management and rehabilitation interventions for patients with SSc. The aim of this study was to present baseline demographic, medical and patient-reported outcome data of the SPIN Cohort and to compare it with other large SSc cohorts. Methods: Descriptive statistics were used to summarize SPIN Cohort characteristics; these were compared with published data of the European Scleroderma Trials and Research (EUSTAR) and Canadian Scleroderma Research Group (CSRG) cohorts. Results: Demographic, organ involvement and antibody profile data for SPIN (N = 1125) were generally comparable with that of the EUSTAR (N = 7319) and CSRG (N = 1390) cohorts. There was a high proportion of women and White patients in all cohorts, though relative proportions differed. Scl70 antibody frequency was highest in EUSTAR, somewhat lower in SPIN, and lowest in CSRG, consistent with the higher proportion of interstitial lung disease among dcSSc patients in SPIN compared with in CSRG (48.5 vs 40.3%). RNA polymerase III antibody frequency was highest in SPIN and remarkably lower in EUSTAR (21.1 vs 2.4%), in line with the higher prevalence of SSc renal crisis (4.5 vs 2.1%) in SPIN. Conclusion: Although there are some differences, the SPIN Cohort is broadly comparable with other large prevalent SSc cohorts, increasing confidence that insights gained from the SPIN Cohort should be generalizable, although it should be noted that all three cohorts include primarily White participants.

Performance of the Patient-Reported Outcomes Measurement Information System-29 in scleroderma: a Scleroderma Patient-centered Intervention Network Cohort Study.

Objective: The Patient-Reported Outcomes Measurement Information System (PROMIS)-29 assesses seven health-related quality of life domains plus pain intensity. The objective was to examine PROMIS-29v2 validity and explore clinical associations in patients with SSc. Methods: English-speaking SSc patients in the Scleroderma Patient-centered Intervention Network Cohort from 26 sites in Canada, the USA and the UK completed the PROMIS-29v2 between July 2014 and November 2015. Enrolling physicians provided medical data. To examine convergent validity, hypotheses on the direction and magnitude of correlations with legacy measures were tested. For clinical associations, t -tests were conducted for dichotomous variables and PROMIS-29v2 domain scores. Effect sizes (ESs) were labelled as small (<0.25), small to moderate (0.25-0.45), moderate (0.46-0.55), moderate to large (0.56-0.75) and large (>0.75). Results: There were 696 patients (87% female), mean ( s . d .) disease duration 11.6 (8.7) years, 57% with limited cutaneous subtype. Validity indices were consistent with seven of nine hypotheses (| r | =0.51-0.87, P < 0.001), with minor divergence for two hypotheses. Gastrointestinal involvement was associated with significantly worse outcomes for all eight PROMIS-29v2 domains (moderate or moderate to large ES in six of eight). Presence of joint contractures was associated with significant decrements in seven domains (small or small to moderate ESs). Skin thickening, diffuse cutaneous subtype and presence of overlap syndromes were significantly associated (small or small to moderate ESs) with five or six domains. Conclusion: This study further establishes the validity of the PROMIS-29v2 in SSc and underlines the importance of gastrointestinal symptoms and joint contractures in reduced health-related quality of life.

Emerging Guidelines for Patient Engagement in Research.

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