Patient-centered research should assess outcomes important to patients and include patient-reported outcome measures (PROMs) to assess health-related quality of life (HRQOL) domains. Using a well-known HRQOL framework (World Health Organization QOL, or WHOQOL), we reviewed established PROMs used with adults with different types of arthritis to evaluate their HRQOL domain coverage and psychometric evidence to help PROM users select measures and determine whether other measures should be validated and/or developed. Nineteen PROMs and 92 corresponding articles were reviewed to determine which HRQOL domains were assessed. To support a streamlined but rigorous review, we used a rating system based on criteria established in part through existing rubrics (e.g., OMERACT COSMIN). Psychometric properties were rated on a scale from 1 to 18, where 18 was strongest. We examined the intersection between level of domain coverage and extent of psychometric support. Measures most commonly assessed physical health and level of independence, while fewer assessed social relations, environment, and psychological health. No measures assessed spirituality and religion, which may be relevant depending on intended use. PROMs with higher psychometric evidence tended to assess a broader range of HRQOL domains. Rubric scores ranged from 3 to 16, with an average of 9.3. Prominent and psychometrically sound PROMs are available that cover many of the WHOQOL domains. While gaps exist in the domain of spirituality, future work should focus on refining optimal use of existing PROMs relevant for arthritis versus developing new measures. We provide guidance on selecting PROMs, to that end.

Objective This study aimed to identify differences in patient empowerment based on biopsychosocial patient-reported measures, the magnitude of those differences, and which measures best explain differences in patient empowerment. Methods This was a cross-sectional observational study of 6918 adults with arthritis in the US. Data were collected from March 2019 to March 2020 through the Arthritis Foundation Live Yes! INSIGHTS program. Patient empowerment, measured by the Health Care Empowerment Questionnaire, included 2 scales: Patient Information Seeking and Healthcare Interaction Results. Patient-reported outcomes were measured using the Patient Reported Outcomes Measurement Information System (PROMIS)-29 and PROMIS emotional support scale. ANOVA assessed differences between groups, and Spearman rank correlation assessed correlations between variables. Hierarchical regression analysis determined the contributions of sociodemographic characteristics, arthritis type, and patient-reported health measures in explaining patient empowerment (α = 0.05). Results Empowerment was lower among those who were male, older, less educated, or who had lower income, osteoarthritis, less emotional support, or better physical function, although the effect was small-to-negligible for most of these variables in the final regression models. Empowerment did not differ by race/ethnicity in unadjusted or adjusted analysis. In final regression models, emotional support contributed the most to explaining patient empowerment. Conclusion Emotional support is important for patient empowerment. This suggests that programs that seek to improve patient empowerment should target and measure effects on emotional support.

The translation of research findings into clinical practice is challenging, especially fields like in pediatric rheumatology, where the evidence base is limited, there are few clinical trials, and the conditions are rare and heterogeneous. Implementation science methodologies have been shown to reduce the research- to- practice gap in other clinical settings may have similar utility in pediatric rheumatology. This paper describes the key discussion points from the inaugural Childhood Arthritis and Rheumatology Research Alliance Implementation Science retreat held in February 2020. The aim of this report is to synthesize those findings into an Implementation Science Roadmap for pediatric rheumatology research. This roadmap is based on three foundational principles: fostering curiosity and ensuring discovery, integration of research and quality improvement, and patient-centeredness. We include six key steps anchored in the principles of implementation science. Applying this roadmap will enable researchers to evaluate the full range of research activities, from the initial clinical design and evidence acquisition to the application of those findings in pediatric rheumatology clinics and direct patient care.

OBJECTIVE The development of new treatment approaches for degenerative lumbar spondylolisthesis (DLS) has introduced many questions about comparative effectiveness and long-term outcomes. Patient registries collect robust, longitudinal data that could be combined or aggregated to form a national and potentially international research data infrastructure to address these and other research questions. However, linking data across registries is challenging because registries typically define and capture different outcome measures. Variation in outcome measures occurs in clinical practice and other types of research studies as well, limiting the utility of existing data sources for addressing new research questions. The purpose of this project was to develop a minimum set of patient- and clinician-relevant standardized outcome measures that are feasible for collection in DLS registries and clinical practice. METHODS Nineteen DLS registries, observational studies, and quality improvement efforts were invited to participate and submit outcome measures. A stakeholder panel was organized that included representatives from medical specialty societies, health systems, government agencies, payers, industries, health information technology organizations, and patient advocacy groups. The panel categorized the measures using the Agency for Healthcare Research and Quality’s Outcome Measures Framework (OMF), identified a minimum set of outcome measures, and developed standardized definitions through a consensus-based process. RESULTS The panel identified and harmonized 57 outcome measures into a minimum set of 10 core outcome measure areas and 6 supplemental outcome measure areas. The measures are organized into the OMF categories of survival, clinical response, events of interest, patient-reported outcomes, and resource utilization. CONCLUSIONS This effort identified a minimum set of standardized measures that are relevant to patients and clinicians and appropriate for use in DLS registries, other research efforts, and clinical practice. Collection of these measures across registries and clinical practice is an important step for building research data infrastructure, creating learning healthcare systems, and improving patient management and outcomes in DLS.

Abstract Background Patient empowerment can improve health‐related outcomes and is important in chronic conditions, such as arthritis. This study aimed to validate the Health Care Empowerment Questionnaire (HCEQ), a patient‐reported experience measure of empowerment, for use with patients with arthritis and other rheumatic diseases. Methods The HCEQ measures Patient Information Seeking (or Involvement in Decisions) and Healthcare Interaction Results (or Involvement in Interactions) and asks respondents to answer questions in two ways: whether they feel something happened and its importance to them. Face validity was assessed through qualitative data (n = 8, nominal group technique; n = 55, focus groups). Measure structure was assessed through confirmatory factor analysis (CFA); internal consistency was also assessed (n = 9226). Test‐retest reliability was assessed with sub‐sample of participants (n = 182). Results We found adequate face validity of the HCEQ for patients with arthritis. The CFA indicated good fit to the data for the two‐factor structure of the HCEQ (RMSEA = 0.075; CFI = 0.987; TLI = 0.978; SRMR = 0.026). Internal consistency was strong (α=0.94 for both subscales). Test‐retest reliability was moderate for Patient Information Seeking (ICC=0.67) and good for Healthcare Interaction Results (ICC=0.77). Conclusions The HCEQ, with modifications, demonstrated promising psychometric properties within this sample, laying the foundation for further assessment. This work supports the HCEQ as an appropriate instrument for examining experiences with and perceived importance of empowerment in individuals with arthritis and other rheumatic conditions. Patient Contribution Patients contributed to the assessment of face validity. As a measure of patient empowerment, the HCEQ’s use can enable further participation of patients in health care.

Purpose: Knee osteoarthritis (KOA) is a prevalent form of chronic joint disease associated with functional restrictions, morphological changes and pain. Pain and disability from KOA negatively impact social connectedness and psychological well-being, reducing the quality of life (QoL) of patients. Healthcare providers are increasingly realizing the importance of ensuring the implementation of psychosocial support to improve the health and overall well-being of KOA patients. Assessing QoL is an imperative first step in evaluating well-being, disease progression, and intervention efficacy. The purpose of this review is to provide an international resource summarizing available studies, which have reported individual factors affecting QoL in KOA patients. Our Results aim to prompt incorporation of psychosocial assessment in management strategies. Patient organization representatives designed and executed this summary to prompt routine evaluation of such. Methods: We conducted a systematic review examining the literature up to JAN/2017 available at MEDLINE, EMBASE, Cochrane, and PsycINFO using KOA and QOL related keywords. All articles were reviewed for inclusion by 3 independent reviewers. QoL domains and items relevant to patients with KOA were extracted. Only original articles were included when containing information on QoL of patients with KOA. The quality of included studies was assessed using a quality appraisal tool. Inclusion criteria were QOL compared to at least one demographic factor (e.g., age, gender), lifestyle factor (e.g., functional independence), or comorbidity factor (e.g., diabetes, obesity) and a control group. Analytical methods were not considered as part of the original design. Results: A total of 610 articles were reviewed, of which 62 met inclusion criteria. Instruments used to measure QoL included: SF-36, EQ-5D, KOOS, WHOQOL, HAS, AIMS, NHP, and JKOM. All studies reported worse QoL in KOA patients when compared to a control group. When females were compared to males, females reported worse QOL. Obesity, as well as, lower levels of physical activity were reported with lower QoL scores. Knee self-management programs delivered by healthcare professionals improved QoL in patients with KOA. Educational level and higher total mindfulness were reported to improve QoL whereas poverty, psychological distress, depression and lacking familial relationships reduce it. Surgical KOA intervention outcomes depended on patients’ individual factors. Conclusions: KOA studies routinely include pain and function scores yet haven’t routinely included psychosocial variables assessing QoL, which also influences how patients feel, function, and survive. KOA has a substantial impact on QoL. In KOA patients, QoL is also influenced by specific individual factors including gender, body weight, physical activity, mental health, and education. Importantly, education and management programs designed to support KOA patients report improved QoL. QoL data is a valuable tool providing health care professionals with a better comprehension of KOA disease to aid the implementation of the most effective management plan. Ensuring a standard QoL assessment is implemented, as routine care globally is imperative for healthcare professionals to gain a better understanding of OA disease whilst ensuring the most optimal management. This study was coordinated by patient organizations Future KOA QoL research should combine efforts globally and focus on consistent quantitative and qualitative measures for more meaningful impact and interpretation.

Background Globally, osteoarthritis (OA) is the third condition associated with disability. There is still poor treatment in OA but science holds the key to finding better treatments and a cure. It is essential to learn what’s important to patients from them to implement the most effective OA management. The OA Patients Task Force, conducted the Global OA Patient Perception Survey (GOAPPS)-the first global survey made by patients to analize the quality of life (QoL) & patient perceptions of care. The goal was to collect data on OA patients’ perception of OA to understand patients’ needs and expectations to improve OA management. Methods Observational, cross-sectional study by online survey data collection from six countries, translated into three languages. The questionnaire was comprised of 3 sections: patient demographics and clinical symptomology characteristics; relationship with physicians: perception of attention, treatment, and information provided; and OA impact on daily activity and QoL. The results of the survey were evaluated using the Limited Data Set. The survey results were analyzed using descriptive statistics to characterize the patients’ answers. Additionally, Cronbach’s alpha was calculated to determine internal consistency validity. Results A total of 1512 surveys were completed in 6 countries. 84.2% of respondents reported pain/tenderness and 91.1% experienced limitations to physical activities. 42.3% of patients were not satisfied with their current OA treatment. 86% had comorbidities, especially hypertension, and obesity. 51.3 and 78% would like access to additional drug or additional non-drug/non-surgical treatments respectively. 48.2% of patients perceived their QoL to be affected by OA. The Cronbach’s alpha was 0.61. Conclusions OA has a significant impact on patients’ daily activities and their desire to play an active role in managing this disease. Patients are seeking additional treatments, especially no pharmacological/no surgical treatments stressing the need for investing in clinical research, implementing OA preventive measures, and managing interventions to improve the healthcare value chain in OA.

The Biomarkers Consortium aims to facilitate drug development with biomarkers across a range of diseases. Here, we briefly highlight its accomplishments so far and its recent expansion in scope to include related tools along the lines of the Biomarkers, EndpointS and other Tools (BEST) resource, such as patient-reported outcomes and clinical outcome assessments.

Background Treatment decision-making regarding immunosuppressive therapy is challenging for individuals with lupus. We assessed the effectiveness of a decision aid for immunosuppressive therapy in lupus nephritis. Methods and findings In a United States multicenter, open-label, randomized controlled trial (RCT), adult women with lupus nephritis, mostly from racial/ethnic minority backgrounds with low socioeconomic status (SES), seen in in- or outpatient settings, were randomized to an individualized, culturally tailored, computerized decision aid versus American College of Rheumatology (ACR) lupus pamphlet (1:1 ratio), using computer-generated randomization. We hypothesized that the co-primary outcomes of decisional conflict and informed choice regarding immunosuppressive medications would improve more in the decision aid group. Of 301 randomized women, 298 were analyzed; 47% were African-American, 26% Hispanic, and 15% white. Mean age (standard deviation [SD]) was 37 (12) years, 57% had annual income of <$40,000, and 36% had a high school education or less. Compared with the provision of the ACR lupus pamphlet (n = 147), participants randomized to the decision aid (n = 151) had (1) a clinically meaningful and statistically significant reduction in decisional conflict, 21.8 (standard error [SE], 2.5) versus 12.7 (SE, 2.0; p = 0.005) and (2) no difference in informed choice in the main analysis, 41% versus 31% (p = 0.08), but clinically meaningful and statistically significant difference in sensitivity analysis (net values for immunosuppressives positive [in favor] versus negative [against]), 50% versus 35% (p = 0.006). Unresolved decisional conflict was lower in the decision aid versus pamphlet groups, 22% versus 44% (p < 0.001). Significantly more patients in the decision aid versus pamphlet group rated information to be excellent for understanding lupus nephritis (49% versus 33%), risk factors (43% versus 27%), medication options (50% versus 33%; p ≤ 0.003 for all); and the ease of use of materials was higher in the decision aid versus pamphlet groups (51% versus 38%; p = 0.006). Key study limitations were the exclusion of men, short follow-up, and the lack of clinical outcomes, including medication adherence. Conclusions An individualized decision aid was more effective than usual care in reducing decisional conflict for choice of immunosuppressive medications in women with lupus nephritis. Trial registration Clinicaltrials.gov, NCT02319525.

Background Knee OA (KOA) is the most common form of chronic joint disease and bears more responsibility than any other disease for disability1. It associates with remarkable functional restrictions due to pain. The limitations in activity caused by KOA seriously affect social relationships, emotional well-being, reducing the quality of life (QoL) of patients. The identification of therapies and factors that affects and improve QoL in KOA patients may mitigate the clinical, economic, and social burden of this disease2. Thus, the assessment of QoL in KOA is becoming increasingly common in both research and clinical practice3. Still a general recompilation of the factors of interest as demographic features, lifestyle characteristics and comorbidity are missing. Objectives Our aim was to recapitulate the existing information on QoL in KOA patients as an international tool to raise awareness on their condition and guide future actions for patient's management Methods We conducted a systematic review examining the breadth of the literature regarding the QoL in patients with KOA (up to 2017). We identify articles using MEDLINE, EMBASE, Cochrane, and PsycINFO using relevant keywords as KOA, QoL and well-being and their short forms. All articles were reviewed for inclusion by 3 independent reviewers. QoL domains and items relevant to patients with KOA were extracted. Only original articles were included when containing information on QoL of patients with KOA. Inclusion criteria were QoL compared to one or more demographic factors (e.g., age, gender), lifestyle factor (e.g., functional independence), or comorbidity factor (e.g., diabetes, obesity) or a control group. The quality of included studies was assessed using a quality appraisal tool. Results We retrieved 610 articles, of which 62 articles fulfilled inclusion criteria for review. Most of the studies were carried out in Europe, American Continent and Asia. The mean of participants in these 62 studies was 561 patients and the majority of them were female, the mean age was 63 years. All the studies described a worse QoL in KOA patients when compared to a control group having women a worst QoL perception than men. A higher BMI, a lower level of physical activity and higher energy expenditure were one of the main factors that correlated with worse QoL. Educational level and higher total mindfulness were shown to improve QoL while poverty, physiological distress, depression and having severely dysfunctional families reduce it. The delivery of a knee self-management program by health care professionals was proven to improve QoL. Finally, surgical KOA interventions generally resulted in good outcomes these results were influenced by individual factors as age, weight, and depression Conclusion This is the first review pertaining to QoL in KOA patients. KOA has a strong impact on QoL. Individual factors (sex, weight, exercise, mental health, education) can influence QoL. These factors affect treatment outcomes and should be considered for a better patient's management. These data are a valuable tool for health professionals, to better understand the disease and to implement more adequate standard of care. Disclosure of Interests: Josep Verges: None declared, Marianna Vitaloni: None declared, Marco Bibas: None declared, Rosa Sciortino: None declared, Maritza Quintero: None declared, Jordi Monfort Speakers bureau: Bioibérica Procare Health, Francisco de Abajo: None declared, Patrick du Souich: None declared, Ingrid Möller: None declared, Elizabeth Oswald: None declared, Marco Matucci-Cerinic Grant/research support from: Actelion, MSD, Pfizer, BMS, Chemomab, Sanipedia, Speakers bureau: Actelion, BMS; MSD, Janssen, Guy Eakin: None declared, Angie Botto-van Bemden: None declared

Background Knee osteoarthritis (KOA) is a prevalent form of chronic joint disease associated with functional restrictions and pain. Activity limitations negatively impact social connectedness and psychological well-being, reducing the quality of life (QoL) of patients. The purpose of this review is to summarize the existing information on QoL in KOA patients and share the reported individual factors, which may influence it. Methods We conducted a systematic review examining the literature up to JAN/2017 available at MEDLINE, EMBASE, Cochrane, and PsycINFO using KOA and QOL related keywords. Inclusion criteria were QOL compared to at least one demographic factor (e.g., age, gender), lifestyle factor (e.g., functional independence), or comorbidity factor (e.g., diabetes, obesity) and a control group. Analytical methods were not considered as part of the original design. Results A total of 610 articles were reviewed, of which 62 met inclusion criteria. Instruments used to measure QoL included: SF-36, EQ-5D, KOOS, WHOQOL, HAS, AIMS, NHP and JKOM. All studies reported worse QoL in KOA patients when compared to a control group. When females were compared to males, females reported worse QOL. Obesity as well as lower level of physical activity were reported with lower QoL scores. Knee self-management programs delivered by healthcare professionals improved QoL in patients with KOA. Educational level and higher total mindfulness were reported to improve QoL whereas poverty, psychological distress, depression and lacking familial relationships reduce it. Surgical KOA interventions resulted in good to excellent outcomes generally; although, results varied by age, weight, and depression. Conclusion KOA has a substantial impact on QoL. In KOA patients, QoL is also influenced by specific individual factors including gender, body weight, physical activity, mental health, and education. Importantly, education and management programs designed to support KOA patients report improved QoL. QoL data is a valuable tool providing health care professionals with a better comprehension of KOA disease to aid implementation of the most effective management plan.

A meeting was organized to bring together multiple stakeholders involved in the testing and authorization of new medications for juvenile idiopathic arthritis (JIA) to discuss current issues surrounding clinical trials and access to new medications for children and adolescents with JIA. The Childhood Arthritis and Rheumatology Research Alliance invited representatives of regulatory agencies (Food and Drug Administration and European Medicines Agency), and major pharmaceutical companies with JIA-approved products or products in development, patient and parent representatives, representatives of an advocacy organization (Arthritis Foundation), and pediatric rheumatology clinicians/investigators to a 1-day meeting in April 2018. The participants engaged in discussion regarding issues in clinical trials. As the pharmacologic options to treat inflammatory arthritis rapidly expand, registration trial designs to test medications in JIA patients must adapt. Many methodologies successfully used in the recent past are no longer feasible. The pool of patients meeting entry criteria who are willing to participate is shrinking while the number of medications to be tested is growing. Suggested solutions included proposing innovative clinical trial methods to regulatory agencies, as well as open discussions among stakeholders. Ensuring that new medications are authorized in a timely manner to meet the needs of JIA patients worldwide is critical. Approaches should include open dialog between regulatory agencies, pharmaceutical companies, and other stakeholders to develop and implement novel study designs, including patient and clinician perspectives to define meaningful trial outcomes, and changing existing study plans.

Purpose: Globally, osteoarthritis (OA) is the third most rapidly rising condition associated with disability. Science holds the key to finding better treatments and one day a cure. Still it is essential we learn what's important to patients from the patients themselves in order to optimize implementation of better healthcare options for most effective global management of OA. Integrated people-centred health services, as defined by the World Health Organization, implies putting the comprehensive needs of people and communities, not only diseases, at the centre of health systems, and empowering people to have a more active role in their own health. Based on this definition, the International Osteoarthritis Task Force, an initiative of the Osteoarthritis Foundation International (Barcelona, Spain) and the Arthritis Foundation (Atlanta, United States) with participation from member organizations in additional countries created the Global Osteoarthritis (OA) Patient Perception Survey GOAPPS)-the first global survey of its kind to directly compare quality of life & patient perceptions of care in the same OA patient survey across languages and cultures. The goal of the survey was to help all stakeholders in OA healthcare develop a better understanding of patients' perceptions and how they may differ between cultures by collecting data on adult OA patient perceptions regarding their OA care. The survey will also collect data on patient demographics, OA symptomology, and impact of OA on daily functioning and quality of life. Understanding patients' needs and perceptions of care is the first step in optimizing global OA management. Methods: Observational, cross-sectional study involving data collection through an online survey. Survey administration in each country was managed by a local Survey Coordinator who collaborated with local organizations to promote access to the registration webpage through social media promotions and by distributing brochures and other promotional materials. The original version of the survey was composed in English; the local Survey Coordinator oversaw translation into the predominant local language. Using the Limited Data Set, we used descriptive statistics to characterize responses to questions on patient perceptions regarding their OA care. The Task Force used data collected on patient demographics, OA symptomology, and impact of OA on daily functioning and quality of life to (1) segment patient perception data and (2) conduct preliminary investigation into the relationship between patient perceptions of their OA care, OA symptoms and impacts, and osteoarthritis-related QoL. Responses were collected in each participating country. The final questionnaire was divided into 4 sections. The first includes questions to measure clinical characteristics of the patient and the relationship with physicians and treatment. This was followed by a section on personal perception of attention, treatment and information received. In the last session patients were asked to evaluate their quality of life. Inclusion criteria included resident age 18 or older with an OA diagnosis Results: A total of 1485 surveys were completed by OA patients from 7 different countries. 1264 surveys were answered in English, 218 in Spanish and 3 in Italian; thus, analysis of cultural differences was unable to be carried out in this pilot. Preliminary data show that the majority of the respondents were female (90%) and more than 55 years of age (82%). The majority (73%) of patients had knee OA, followed by hand (57%) and spine (54%). Comorbidities included hypertension (50%) and obesity (43%). Nearly all patients reported limitations related to physical activities (97%), followed by work activities (49%), social interaction (43%) and sex life (23%). Notably, 37% of patients experience emotional, physiological or a mental health issue. Of those surveyed, 58% say their doctor adequately explained their OA diagnosis and 55% understand their OA treatment options and the associated risks. Importantly, 41% of respondents are not satisfied with their current OA treatment plan and 79% would like access to additional non-drug/non-surgical treatments for their OA. While 52% of respondents rate their quality of life as good or very good, 95% would rate it as good or very good if their OA were eliminated. Conclusions: The results emphasize the significant impact of OA disease on patients' daily activities and their desire to play an active role in managing their disease. Notably the majority asks for access to additional options for non-drug/non-surgical treatments stressing the need for an OA management improvement. This is a pilot survey and the results do not permit analysis of cultural differences. Continued survey distribution & analysis will determine differences in patient perception of QoL and care between OA patients living in different countries to further optimize global OA management.

Arthritis is well known in all cultures, with medical descriptions of inflammatory arthritis dating as early as the writings of Hippocrates,1 and paleontological evidence dating back 150 million years.2 Today, the data regarding the prevalence of these diseases remains scant, but suggest that close to 300 million people around the world live with arthritis.3 In terms of years lived with disability (YLD), arthritis and allied disorders account for 21.2%, second only to mental health issues.4 Though often associated with aging, in reality, arthritis can manifest at any age, even in childhood. In the US, approximately 300,000 children are affected by pediatric forms of arthritis.5