Defining domains: developing consensus-based definitions for foundational domains in OMERACT core outcome sets.

OBJECTIVE: To develop a set of detailed definitions for foundational domains commonly used in OMERACT (Outcome Measures in Rheumatology) core domain sets. METHODS: We identified candidate domain definitions from prior OMERACT publications and websites and publications of major organizations involved in outcomes research for six domains commonly used in OMERACT Core Domain Sets: pain intensity, pain interference, physical function, fatigue, patient global assessment, and health-related quality of life. We conducted a two-round survey of OMERACT working groups, patient research partners, and then the OMERACT Technical Advisory Group to establish their preferred domain definitions. Results were presented at the OMERACT 2023 Methodology Workshop, where participants discussed their relevant lived experience and identified potential sources of variability giving the needed detail in our domain definitions. RESULTS: One-hundred four people responded to both rounds of the survey, and a preferred definition was established for each of the domains except for patient global assessment for which no agreement was reached. Seventy-five participants at the OMERACT 2023 Methodology Workshop provided lived experience examples, which were used to contextualise domain definition reports for each of the five domains. CONCLUSION: Using a consensus-based approach, we have created a detailed definition for five of the foundational domains in OMERACT core domain sets; patient global assessment requires further research. These definitions, although not mandatory for working groups to use, may facilitate the initial domain-match assessment step of instrument selection, and reduce the time and resources required by future OMERACT groups when developing core outcome sets.

Development of a research capacity and culture tool for people with lived experience of mental health challenges.

OBJECTIVE: This study investigated research capacity and culture in people with lived experience of mental health challenges by developing a quantitative tool that measures capacity to engage in research. METHOD: A cross-sectional, correlational methodology was employed, which comprised of three phases: lived experience consultations for item development (n = 15), item refinement (n = 20) and tool piloting. Items were adapted from, and extended, an existing research capacity and culture tool for healthcare workers. People (N = 112) with lived experience as mental health consumers, carers, peer workers and/or advocates aged 18-75 years took part in the tool piloting survey. RESULTS: Overall, participants rated their individual research capacity and culture skills as moderate (mean = 5.41, standard deviation = 2.04). The most commonly reported barriers to research engagement related to lack of knowledge, familiarity or experience with the research process. The most commonly reported enablers were altruistic, such as using their experiences to improve services and help others. Research capacity and culture significantly correlated with current research activities (rs = 0.25-0.41; ps < 0.05), but not with being a research participant (r = 0.09; p > 0.05), suggesting that building research capacity of people with lived experience requires them to be active members of research teams. CONCLUSION: The Lived Experience research capacity and culture tool developed in this study revealed that people with lived experience of mental health challenges are intrinsically motivated to engage in research to improve consumer outcomes. The tool may be useful to assess self, research team and organisational preparedness to conduct genuinely co-designed research, and to assess changes in lived experience research capacities and culture over time.

Australian recommendations on tapering of biologic and targeted synthetic disease-modifying anti-rheumatic drugs in inflammatory arthritis.

Biological and targeted synthetic disease-modifying antirheumatic drugs (b/tsDMARD) have been an important advance in the management of inflammatory arthritis, but are expensive medications, carry a risk of infection and other adverse effects, and are often perceived as a burden by patients. We used GRADE methodology to develop recommendations for dose reduction and discontinuation of b/tsDMARD in people with rheumatoid arthritis (RA), axial spondyloarthritis (AxSpA) and psoriatic arthritis (PsA) who have achieved a low disease activity state or remission. The recommendations form part of the Australian Living Guideline for the Pharmacological Management of Inflammatory Arthritis, an NHMRC-endorsed 'living' guideline, in which recommendations are updated in near real-time as new evidence emerges. Conditional recommendations were made in favour of dose reduction in RA and AxSpA but not in PsA. Abrupt discontinuation of b/tsDMARD is not recommended in any of the three diseases.

Involving People With Lived Experience as Partners in Musculoskeletal Research: Lessons From a Survey of Aotearoa/New Zealand Musculoskeletal Researchers.

SYNOPSIS: Involving patients as partners in research enables their concerns, perspectives, lived experiences, and priorities to be integrated into research. Involving patient partners improves research processes, outcomes, and translating findings into practice. Although musculoskeletal researchers consider that it is important to involve patient partners, few projects involve them. Researchers who involve patient partners report that the contributions of patient partners are very valuable, and researchers perceive the process to be less challenging than expected. Musculoskeletal research is staring at a significant unrealized opportunity to enhance the quality and impact of research and reduce research waste-think what the field could achieve if researchers and patients worked better together. A culture change is needed so that involving patient partners in musculoskeletal research becomes standard practice, expected and supported by funders, journals, research institutions, and researchers alike. J Orthop Sports Phys Ther 2022;52(6):307-311. doi:10.2519/jospt.2022.10986.

"Listen to me, learn from me": a priority setting partnership for shaping interdisciplinary pain training to strengthen chronic pain care.

ABSTRACT: What are the care-seeking priorities of people living with chronic pain and carers and how can these shape interdisciplinary workforce training to improve high-value pain care? Phase 1: Australian people living with chronic pain (n = 206; 90% female) and carers (n = 10; 40% female) described their pain care priorities (eDelphi, round 1). A coding framework was inductively derived from 842 pain care priorities (9 categories, 52 priorities), including validation; communication; multidisciplinary approaches; holistic care; partnerships; practitioner knowledge; self-management; medicines; and diagnosis. Phase 2: In eDelphi round 2, panellists (n = 170; valid responses) rated the importance (1 = less important; 9 = more important) of the represented framework. In parallel, cross-discipline health professionals (n = 267; 75% female) rated the importance of these same priorities. Applying the RAND-UCLA method (panel medians: 1-3: "not important," 4-6: "equivocal," or 7-9: "important"), "important" items were retained where the panel median score was >7 with panel agreement ≥70%, with 44 items (84.6%) retained. Specific workforce training targets included the following: empathic validation; effective, respectful, safe communication; and ensuring genuine partnerships in coplanning personalised care. Panellists and health professionals agreed or strongly agreed (95.7% and 95.2%, respectively) that this framework meaningfully reflected the importance in care seeking for pain. More than 74% of health professionals were fairly or extremely confident in their ability to support care priorities for 6 of 9 categories (66.7%). Phase 3: An interdisciplinary panel (n = 5) mapped an existing foundation-level workforce training program against the framework, identifying gaps and training targets. Recommendations were determined for framework adoption to genuinely shape, from a partnership perspective, Australian interdisciplinary pain training.

Efficacy of probucol on cognitive function in Alzheimer's disease: study protocol for a double-blind, placebo-controlled, randomised phase II trial (PIA study).

INTRODUCTION: Preclinical, clinical and epidemiological studies support the hypothesis that aberrant systemic metabolism of amyloid beta (Aß) in the peripheral circulation is causally related to the development of Alzheimer's disease (AD). Specifically, recent studies suggest that increased plasma concentrations of lipoprotein-Aß compromise the brain microvasculature, resulting in extravasation and retention of the lipoprotein-Aß moiety. The latter results in an inflammatory response and neurodegeneration ensues. Probucol, a historic cholesterol-lowering drug, has been shown in murine models to suppress lipoprotein-Aß secretion, concomitant with maintaining blood-brain-barrier function, suppressing neurovascular inflammation and supporting cognitive function. This protocol details the probucol in Alzheimer's study, a drug intervention trial investigating if probucol has potential to attenuate cognitive decline, delay brain atrophy and reduce cerebral amyloid burden in patients with mild-to-moderate AD. METHODS AND ANALYSIS: The study is a phase II, randomised, placebo-controlled, double-blind single-site clinical trial held in Perth, Australia. The target sample is 314 participants with mild-to-moderate AD. Participants will be recruited and randomised (1:1) to a 104-week intervention consisting of placebo induction for 2 weeks followed by 102 weeks of probucol (Lorelco) or placebo. The primary outcome is changed in cognitive performance determined via the Alzheimer's Disease Assessment Scales-Cognitive Subscale test between baseline and 104 weeks. Secondary outcomes measures will be the change in brain structure and function, cerebral amyloid load, quality of life, and the safety and tolerability of Lorelco, after a 104week intervention. ETHICS AND DISSEMINATION: The study has been approved by the Bellberry Limited Human Research Ethics Committee (approval number: HREC2019-11-1063; Version 4, 6 October 2021). Informed consent will be obtained from participants prior to any study procedures being performed. The investigator group will disseminate study findings through peer-reviewed publications, key conferences and local stakeholder events. TRIAL REGISTRATION NUMBER: Australian New Zealand Clinical Trials Registry (ACTRN12621000726853).

Health systems strengthening to arrest the global disability burden: empirical development of prioritised components for a global strategy for improving musculoskeletal health.

INTRODUCTION: Despite the profound burden of disease, a strategic global response to optimise musculoskeletal (MSK) health and guide national-level health systems strengthening priorities remains absent. Auspiced by the Global Alliance for Musculoskeletal Health (G-MUSC), we aimed to empirically derive requisite priorities and components of a strategic response to guide global and national-level action on MSK health. METHODS: Design: mixed-methods, three-phase design.Phase 1: qualitative study with international key informants (KIs), including patient representatives and people with lived experience. KIs characterised the contemporary landscape for MSK health and priorities for a global strategic response.Phase 2: scoping review of national health policies to identify contemporary MSK policy trends and foci.Phase 3: informed by phases 1-2, was a global eDelphi where multisectoral panellists rated and iterated a framework of priorities and detailed components/actions. RESULTS: Phase 1: 31 KIs representing 25 organisations were sampled from 20 countries (40% low and middle income (LMIC)). Inductively derived themes were used to construct a logic model to underpin latter phases, consisting of five guiding principles, eight strategic priority areas and seven accelerators for action.Phase 2: of the 165 documents identified, 41 (24.8%) from 22 countries (88% high-income countries) and 2 regions met the inclusion criteria. Eight overarching policy themes, supported by 47 subthemes, were derived, aligning closely with the logic model.Phase 3: 674 panellists from 72 countries (46% LMICs) participated in round 1 and 439 (65%) in round 2 of the eDelphi. Fifty-nine components were retained with 10 (17%) identified as essential for health systems. 97.6% and 94.8% agreed or strongly agreed the framework was valuable and credible, respectively, for health systems strengthening. CONCLUSION: An empirically derived framework, co-designed and strongly supported by multisectoral stakeholders, can now be used as a blueprint for global and country-level responses to improve MSK health and prioritise system strengthening initiatives.

ANZICS guiding principles for complex decision making during the COVID-19 pandemic.

The global 2019 coronavirus disease (COVID-19) pandemic has led to major challenges in clinical decision making when the demand for intensive care exceeds local capacity. In order to promote consistent, transparent, objective and ethical decision making, the Australian and New Zealand Intensive Care Society (ANZICS) formed a committee to urgently develop guidelines outlining key principles that should be utilised during the pandemic. This guidance is intended to support the practice of intensive care specialists during the COVID-19 pandemic and to promote the development of local admission policies that should be endorsed by health care organisations and relevant local authorities.

Pain Measurement in Rheumatic and Musculoskeletal Diseases: Where To Go from Here? Report from a Special Interest Group at OMERACT 2018.

OBJECTIVE: Establishing a research agenda on standardizing pain measurement in clinical trials in rheumatic and musculoskeletal diseases (RMD). METHODS: Discussion during a meeting at the Outcome Measures in Rheumatology (OMERACT) 2018, prepared by a systematic review of existing core outcome sets and a patient online survey. RESULTS: Several key questions were debated: Is pain a symptom or a disease? Are pain core (sub)domains consistent across RMD? How to account for pain mechanistic descriptors (e.g., central sensitization) in pain measurement? CONCLUSION: Characterizing and assessing the spectrum of pain experience across RMD in a standardized fashion is the objective of the OMERACT Pain Working Group.

Establishing an Updated Core Domain Set for Studies in Juvenile Idiopathic Arthritis: A Report from the OMERACT 2018 JIA Workshop.

OBJECTIVE: The current Juvenile Idiopathic Arthritis (JIA) Core Set used in randomized controlled trials (RCT) and longitudinal observational studies (LOS) was developed without the input of patients/parents. At the Outcome Measures in Rheumatology (OMERACT) 2016, a special interest group voted to reconsider the core set, incorporating broader input. We describe subsequent work culminating in an OMERACT 2018 plenary and consensus voting. METHODS: Candidate domains were identified through literature review, qualitative surveys, and online discussion boards (ODB) held with patients with JIA and parents in Australia, Italy, and the United States. A Delphi process with parents, patients, healthcare providers, researchers, and regulators served to edit the domain list and prioritize candidate domains. After the presentation of results, OMERACT workshop participants voted, with consensus set at > 70%. RESULTS: Participants in ODB were 53 patients with JIA (ages 15-24 yrs) and 55 parents. Three rounds of Delphi considering 27 domains were completed by 190 (response rate 85%), 201 (84%), and 182 (77%) people, respectively, from 50 countries. There was discordance noted between domains prioritized by patients/parents compared to others. OMERACT conference voting approved domains for JIA RCT and LOS with 83% endorsement. Mandatory domains are pain, joint inflammatory signs, activity limitation/physical function, patient's perception of disease activity (overall well-being), and adverse events. Mandatory in specific circumstances: inflammation/other features relevant to specific JIA categories. CONCLUSION: Following the OMERACT methodology, we developed an updated JIA Core Domain Set. Next steps are to identify and systematically evaluate best outcome measures for these domains.

Health literacy: what is it and why is it important to measure?

This report summarizes the proceedings of the first Outcome Measures in Rheumatology Clinical Trials (OMERACT) Health Literacy Special Interest Group workshop at the OMERACT 10 conference. Health literacy refers to an individual's capacity to seek, understand, and use health information. Discussion centered on the relevance of health literacy to the rheumatology field; whether measures of health literacy were important in the context of clinical trials and routine care; and, if so, whether disease-specific measures were required. A nominal group process involving 27 workshop participants, comprising a patient group (n = 12) and a healthcare professional and researcher group (n = 15), confirmed that health literacy encompasses a broad range of concepts and skills that existing scales do not measure. It identified the importance and relevance of patient abilities and characteristics, but also health professional factors and broader contextual factors. Sixteen themes were identified: access to information; cognitive capacity; disease; expression/communication; finances; health professionals; health system; information; literacy/numeracy; management skills; medication; patient approach; dealing with problems; psychological characteristics; social supports; and time. Each of these was divided further into subthemes of one or more of the following: knowledge, attitude, attribute, relationship, skill, action, or context. There were virtually no musculoskeletal-specific statements, suggesting that a generic health literacy tool in rheumatology is justified. The detailed concepts across themes provided new and systematic insight into what needs to be done to improve health literacy and consequently reduce health inequalities. These data will be used to derive a more comprehensive measure of health literacy.