EULAR recommendations for the involvement of patient research partners in rheumatology research: 2023 update.

BACKGROUND: Since the publication of the 2011 European Alliance of Associations for Rheumatology (EULAR) recommendations for patient research partner (PRP) involvement in rheumatology research, the role of PRPs has evolved considerably. Therefore, an update of the 2011 recommendations was deemed necessary. METHODS: In accordance with the EULAR Standardised Operational Procedures, a task force comprising 13 researchers, 2 health professionals and 10 PRPs was convened. The process included an online task force meeting, a systematic literature review and an in-person second task force meeting to formulate overarching principles (OAPs) and recommendations. The level of agreement of task force members was assessed anonymously (0-10 scale). RESULTS: The task force developed five new OAPs, updated seven existing recommendations and formulated three new recommendations. The OAPs address the definition of a PRP, the contribution of PRPs, the role of informal caregivers, the added value of PRPs and the importance of trust and communication in collaborative research efforts. The recommendations address the research type and phases of PRP involvement, the recommended number of PRPs per project, the support necessary for PRPs, training of PRPs and acknowledgement of PRP contributions. New recommendations concern the benefits of support and guidance for researchers, the need for regular evaluation of the patient-researcher collaboration and the role of a designated coordinator to facilitate collaboration. Agreements within the task force were high and ranged between 9.16 and 9.96. CONCLUSION: The updated EULAR recommendations for PRP involvement are more substantially based on evidence. Together with added OAPs, they should serve as a guide for researchers and PRPs and will ultimately strengthen the involvement of PRPs in rheumatology research.

Decision Aid-Led Tapering of Biologic and Targeted Synthetic DMARDs in Rheumatoid Arthritis: A Qualitative Study.

OBJECTIVE: To explore the experiences and perspectives of patients and rheumatologists on decision aid-led tapering of advanced therapy in rheumatoid arthritis (RA). METHODS: Semi-structured interviews were completed with patients and rheumatologists, embedded within a pilot study of decision aid-led tapering (i.e. dose reduction) of biologic and targeted synthetic (b/ts) DMARDs in RA. All patients were in sustained (>= 6 months) remission and had chosen to reduce their therapy after a decision aid-led shared decision with their rheumatologist. The rheumatologists included those participating in the pilot (n=4), and those who were not. Reflexive thematic analysis of audiotaped and transcribed interviews identified themes in the group experiences. RESULTS: Patients (n=10; 6 female) unanimously found the decision aid easy to understand and felt confident in shared decision-making about treatment tapering and managing flares. Rheumatologists' (n=12; 5 female) perspectives on tapering b/ts DMARDs varied widely, from very supportive to completely opposed, and influenced their views on the decision aid. Rheumatologists expressed concerns about patient comprehension, destabilizing a stable situation, risks of flare, and extending appointment times. Despite their initial reservations about sending the decision aid to all eligible patients ahead of appointments, three of four participating rheumatologists adopted this approach during the pilot, which had the benefit of facilitating patient-led conversations. CONCLUSION: A decision aid-led strategy for tapering advanced therapy in RA was acceptable to patients and feasible in practice. Sending patients a decision aid ahead of their appointment facilitated patient-led conversations about tapering.

Canadian Rheumatology Association Living Guidelines for Rheumatoid Arthritis: Update #2.

We have updated the Canadian Rheumatology Association (CRA) guidelines for rheumatoid arthritis (RA) with 3 recommendations for the use of glucocorticoids (GCs). The recommendations address the use of short-term GCs for RA flares or as bridging therapy when disease-modifying antirheumatic drugs (DMARDs) are initiated or changed, and the use of long-term GCs as adjuncts to DMARDs.

When Should I Get My Next COVID-19 Vaccine? Data From the Surveillance of Responses to COVID-19 Vaccines in Systemic Immune-Mediated Inflammatory Diseases (SUCCEED) Study.

OBJECTIVE: To determine how serologic responses to coronavirus disease 2019 (COVID-19) vaccination and infection in immune-mediated inflammatory disease (IMID) are affected by time since last vaccination and other factors. METHODS: Post-COVID-19 vaccination, data, and dried blood spots or sera were collected from adults with rheumatoid arthritis, inflammatory bowel disease, systemic lupus erythematosus, ankylosing spondylitis and spondylarthritis, and psoriasis and psoriatic arthritis. The first sample was collected at enrollment, then at 2 to 4 weeks and 3, 6, and 12 months after the latest vaccine dose. Multivariate generalized estimating equation regressions (including medications, demographics, and vaccination history) evaluated serologic response, based on log-transformed anti-receptor-binding domain (RBD) IgG titers; we also measured antinucleocapsid (anti-N) IgG. RESULTS: Positive associations for log-transformed anti-RBD titers were seen with female sex, number of doses, and self-reported COVID-19 infections in 2021 to 2023. Negative associations were seen with prednisone, anti-tumor necrosis factor agents, and rituximab. Over the 2021-2023 period, most (94%) of anti-N positivity was associated with a self-reported infection in the 3 months prior to testing. From March 2021 to February 2022, anti-N positivity was present in 5% to 15% of samples and was highest in the post-Omicron era, with antinucleocapsid positivity trending to 30% to 35% or higher as of March 2023. Anti-N positivity in IMID remained lower than Canada's general population seroprevalence (> 50% in 2022 and > 75% in 2023). Time since last vaccination was negatively associated with log-transformed anti-RBD titers, particularly after 210 days. CONCLUSION: Ours is the first pan-Canadian IMID assessment of how vaccine history and other factors affect serologic COVID-19 vaccine responses. These findings may help individuals personalize vaccination decisions, including consideration of additional vaccination when > 6 months has elapsed since last COVID-19 vaccination/infection.

Development of consensus-driven SPIRIT and CONSORT extensions for early phase dose-finding trials: the DEFINE study.

BACKGROUND: Early phase dose-finding (EPDF) trials are crucial for the development of a new intervention and influence whether it should be investigated in further trials. Guidance exists for clinical trial protocols and completed trial reports in the SPIRIT and CONSORT guidelines, respectively. However, both guidelines and their extensions do not adequately address the characteristics of EPDF trials. Building on the SPIRIT and CONSORT checklists, the DEFINE study aims to develop international consensus-driven guidelines for EPDF trial protocols (SPIRIT-DEFINE) and reports (CONSORT-DEFINE). METHODS: The initial generation of candidate items was informed by reviewing published EPDF trial reports. The early draft items were refined further through a review of the published and grey literature, analysis of real-world examples, citation and reference searches, and expert recommendations, followed by a two-round modified Delphi process. Patient and public involvement and engagement (PPIE) was pursued concurrently with the quantitative and thematic analysis of Delphi participants' feedback. RESULTS: The Delphi survey included 79 new or modified SPIRIT-DEFINE (n = 36) and CONSORT-DEFINE (n = 43) extension candidate items. In Round One, 206 interdisciplinary stakeholders from 24 countries voted and 151 stakeholders voted in Round Two. Following Round One feedback, one item for CONSORT-DEFINE was added in Round Two. Of the 80 items, 60 met the threshold for inclusion (≥ 70% of respondents voted critical: 26 SPIRIT-DEFINE, 34 CONSORT-DEFINE), with the remaining 20 items to be further discussed at the consensus meeting. The parallel PPIE work resulted in the development of an EPDF lay summary toolkit consisting of a template with guidance notes and an exemplar. CONCLUSIONS: By detailing the development journey of the DEFINE study and the decisions undertaken, we envision that this will enhance understanding and help researchers in the development of future guidelines. The SPIRIT-DEFINE and CONSORT-DEFINE guidelines will allow investigators to effectively address essential items that should be present in EPDF trial protocols and reports, thereby promoting transparency, comprehensiveness, and reproducibility. TRIAL REGISTRATION: SPIRIT-DEFINE and CONSORT-DEFINE are registered with the EQUATOR Network ( https://www.equator-network.org/ ).

Matching researchers' needs and patients' contributions: practical tips for meaningful patient engagement from the field of rheumatology.

There is an increasing recognition of the importance of patient engagement and involvement in health research, specifically within the field of rheumatology. In general, researchers in this specialty appreciate the value of patients as partners in research. In practice, however, the majority of researchers does not involve patients on their research teams. Many researchers find it difficult to match their needs for patient engagement and the potential contributions from individuals living with rheumatic disease. In this Viewpoint, we provide researchers and patients practical tips for matching 'supply and demand,' based on our own experiences as patient engagement consultants and trainers in rheumatology research. All authors started as a 'naïve' patient or caregiver, an identity that evolved through a process of 'adversarial growth': positive changes that are experienced as a result of the struggle with highly challenging life circumstances. Here, we introduce four stages of adversarial growth in the context of research. We submit that all types of patients have their own experiences, qualities and skills, and can add specific input to research. The recommendations for engagement are not strict directives. They are meant as starting points for discussion or interview. Regardless of individual qualities and knowledge, we believe that all patients engaged in research have a single goal in common: to contribute to research that ultimately will change the lives of many other patients.

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.

Oxford consensus on primary cam morphology and femoroacetabular impingement syndrome: part 1-definitions, terminology, taxonomy and imaging outcomes.

INTRODUCTION: Primary cam morphology is a mostly benign bony prominence that develops at the femoral head-neck junction of the hip, but it is highly prevalent in many athlete populations. In the small proportion of athletes for whom it is not benign, the resulting hip osteoarthritis can be debilitating. Clinicians, athletes, patients and researchers do not yet agree on important primary cam morphology elements. We aimed to ascertain and improve the level of agreement on primary cam morphology definitions, terminology, taxonomy and imaging outcome measures. METHODS: To collect and aggregate informed opinions, an expert panel-the Young Athlete's Hip Research Collaborative-rated primary cam morphology definition, terminology, taxonomy and imaging outcome statements through an online Delphi exercise followed by an online meeting to explore areas of tension and dissent. Reporting followed Conducting and REporting DElphi Studies. RESULTS: A diverse and inclusive Delphi panel (n=65 for rounds 1 and 2, representing 18 countries; 6 stakeholder groups; 40% women) agreed on 35 of 47 statements in 4 domains, while surfacing areas of tension and dissent. This Delphi panel agreed on four key issues essential to moving research and clinical care forward around primary cam morphology. They agreed on: (1) definition, confirming its conceptual attributes (tissue type, size, location, shape and ownership); (2) terminology-use 'morphology' and not terms with a negative connotation like 'lesion', 'abnormality' or 'deformity'; (3) taxonomy, distinguishing between primary and secondary cam morphology, and (4) imaging outcomes, a continuous bone/cartilage alpha angle on radial femoral head-neck MRI for primary cam morphology aetiology research. CONCLUSION: This consensus provides athletes, patients, clinicians and researchers with a strong foundation to guide more precise communication, better clinical decision-making and higher value research about primary cam morphology and its natural history.

Oxford consensus on primary cam morphology and femoroacetabular impingement syndrome: part 2-research priorities on conditions affecting the young person's hip.

INTRODUCTION: Primary cam morphology is highly prevalent in many athlete populations, causing debilitating hip osteoarthritis in some. Existing research is mired in confusion partly because stakeholders have not agreed on key primary cam morphology elements or a prioritised research agenda. We aimed to inform a more rigorous, inclusive and evidence-based approach to research on primary cam morphology and its natural history by working towards agreement on a set of research priorities for conditions affecting the young person's hip. METHODS: An international expert panel-the Young Athlete's Hip Research (YAHiR) Collaborative-rated research priority statements through an online two-round Delphi exercise and met online to explore areas of tension and dissent. Panellists ranked the prioritised research statements according to the Essential National Health Research (ENHR) ranking strategy. Reporting of results followed REPRISE (REporting guideline for PRIority SEtting of health). RESULTS: A diverse Delphi panel (n=65, Delphi rounds 1 and 2; three ENHR strategy surveys: n=49; n=44; n=42) from 18 countries representing six stakeholder groups, prioritised and ranked 18 of 38 research priority statements. The prioritised statements outlined seven research domains: (1) best practice physiotherapy, (2) rehabilitation progression and return to sport, (3) exercise intervention and load management, (4) primary cam morphology prognosis and aetiology, (5) femoroacetabular impingement syndrome prognosis and aetiology, (6) diagnostic criteria, and (7) screening. The panel recommended areas of tension and dissent for the research community to focus on immediately. CONCLUSION: While informing more rigorous, inclusive and evidence-based research, this consensus is a roadmap for researchers, policy-makers and funders to implement research dedicated to reducing the cost and burden of hip disease related to primary cam morphology.

Building Capacity for Patient-Oriented Research: Utilizing Decision Aids to Translate Evidence into Practice, Policy and Outcomes.

BACKGROUND: The aim of this project was to engage with patient partners to translate knowledge about the decision aids and develop a scaling-up strategy for wider effects and reach. METHOD: This project was guided by the World Health Organization and IDEAS (Integrate, Design, Assess and Share) frameworks for design thinking (e.g., ideating creative strategies), dissemination (e.g., sharing locally and widely) and scalability. RESULTS: We engaged 132 stakeholders in six webinars, had 321 total page views of the decision aids and conducted 16 interviews to determine revisions to the design of the decision aids before scalability. CONCLUSION: Patient-partner collaborations assisted with design thinking, dissemination and scalability.

Guidelines for Reporting Trial Protocols and Completed Trials Modified Due to the COVID-19 Pandemic and Other Extenuating Circumstances: The CONSERVE 2021 Statement.

Importance: Extenuating circumstances can trigger unplanned changes to randomized trials and introduce methodological, ethical, feasibility, and analytical challenges that can potentially compromise the validity of findings. Numerous randomized trials have required changes in response to the COVID-19 pandemic, but guidance for reporting such modifications is incomplete. Objective: As a joint extension for the CONSORT and SPIRIT reporting guidelines, CONSERVE (CONSORT and SPIRIT Extension for RCTs Revised in Extenuating Circumstances) aims to improve reporting of trial protocols and completed trials that undergo important modifications in response to extenuating circumstances. Evidence: A panel of 37 international trial investigators, patient representatives, methodologists and statisticians, ethicists, funders, regulators, and journal editors convened to develop the guideline. The panel developed CONSERVE following an accelerated, iterative process between June 2020 and February 2021 involving (1) a rapid literature review of multiple databases (OVID Medline, OVID EMBASE, and EBSCO CINAHL) and gray literature sources from 2003 to March 2021; (2) consensus-based panelist meetings using a modified Delphi process and surveys; and (3) a global survey of trial stakeholders. Findings: The rapid review yielded 41 673 citations, of which 38 titles were relevant, including emerging guidance from regulatory and funding agencies for managing the effects of the COVID-19 pandemic on trials. However, no generalizable guidance for all circumstances in which trials and trial protocols might face unanticipated modifications were identified. The CONSERVE panel used these findings to develop a consensus reporting guidelines following 4 rounds of meetings and surveys. Responses were received from 198 professionals from 34 countries, of whom 90% (n = 178) indicated that they understood the concept definitions and 85.4% (n = 169) indicated that they understood and could use the implementation tool. Feedback from survey respondents was used to finalize the guideline and confirm that the guideline's core concepts were applicable and had utility for the trial community. CONSERVE incorporates an implementation tool and checklists tailored to trial reports and trial protocols for which extenuating circumstances have resulted in important modifications to the intended study procedures. The checklists include 4 sections capturing extenuating circumstances, important modifications, responsible parties, and interim data analyses. Conclusions and Relevance: CONSERVE offers an extension to CONSORT and SPIRIT that could improve the transparency, quality, and completeness of reporting important modifications to trials in extenuating circumstances such as COVID-19.

Patient engagement in a Canadian health research funding institute: implementation and impact.

BACKGROUND: Patient engagement (PE) or involvement in research is when patient partners are integrated onto teams and initiatives (not participants in research). A number of health research funding organisations have PE frameworks or rubrics but we are unaware of them applying and reporting on their own internal PE efforts. We describe our work at the Canadian Institutes of Health Research's Institute of Musculoskeletal Health and Arthritis (CIHR IMHA) to implement, evaluate and understand the impact of its internal PE strategy. METHODS: A co-production model was used involving patient partners, a PE specialist and staff from IMHA. A logic model was co-developed to guide implementing and evaluating IMHA's PE strategy. Some of evaluating the PE strategy and understanding its impact was a collaboration between the Public and Patient Engagement Collaborative (McMaster University) and IMHA. RESULTS: IMHA convened a PE Research Ambassador (PERA) group which co-led this work with the support of a PE specialist. In doing so, PERA had a number of meetings since 2020, set its own priorities and co-produced a number of outputs (video, publications, webinars, blog and modules called the How-to Guide for PE in Research). This work to evaluate and measure impacts of IMHA's PE strategy revealed positive results, for example, on PERA members, Institute Advisory Board members and staff, as well as beyond the institute based on uptake and use of the modules. Areas for improvement are mainly related to increasing the diversity of PERA and to improving accessibility of the PE outputs (more languages and formats). CONCLUSIONS: Implementing a PE strategy within CIHR IMHA resulted in several PE activities and outputs with impacts within and beyond the institute. We provide templates and outputs related to this work that may inform the efforts of other health research funding organisations. We encourage health research funders to move beyond encouraging or requiring PE in funded projects to fully 'walk the talk' of PE by implementing and evaluating their own PE strategies.

Patient and Public Perceptions in Canada About Decentralized and Hybrid Clinical Trials: "It's About Time we Bring Trials to People".

BACKGROUND: Little is known about patient and the public perspectives on decentralized and hybrid clinical trials in Canada. METHODS: We conducted an online survey (English and French) promoted on social media to understand perspectives of people in Canada about decentralized and hybrid clinical trials. The survey had two sections. We co-produced this project entirely with patient, caregiver, and family partners. RESULTS: The survey had 284 (14 French) individuals who started or completed Section 1, and 180 (16 French) individuals who started or completed Section 2. People prefer to have options to participate in clinical trials where aspects are decentralized or hybridized. 79% of respondents preferred to have options related to study visits. There were concerns about handling adverse events or potential complications in decentralized trials, however, communication options such as a dedicated contact person for participants was deemed helpful. Most respondents were amenable to informed consent being done at a satellite site closer to home or via technology and were split on privacy concerns about this. Most preferred travel to a site within an hour, depending on what the trial was for or its impact on quality of life. Due to the response rate, we were unable to explore associations with gender, age, health status, geography, ethnicity, and prior clinical trial participation. CONCLUSION: Our findings indicate an openness in Canada to participating in trials that decentralize or hybridize some aspects. These trials are perceived to provide benefits to participants and ways to increase equity and accessibility for participants.

Co-creating and hosting PxP: a conference about patient engagement in research for and by patient partners.

Research projects, initiatives and conferences that include patients as partners rather than as participants are becoming more common. Including patients as partners (what we will call 'patient partners') is an approach called patient engagement or involvement in research, and we will call it patient engagement throughout this paper. Patient engagement moves traditional health research conferences and events to include a broader audience for their knowledge exchange and community building efforts, beyond academics and healthcare professionals. However, there are few examples of conferences where patients are given the opportunity to fully lead. Our conference went beyond patient engagement - it was patient-led. Patient partners conceived, planned, and decided on all aspects of a virtual conference.We present the work and processes we undertook throughout 2023 to create and produce a free conference called "PxP: For patients, by patients" or PxP for short, with a tagline of "Partnering to make research stronger." PxP was patient-led and about patient engagement in research rather than a specific disease or condition. PxP was supported by the Canadian Institutes of Health Research Institute of Musculoskeletal Health and Arthritis. The PxP website, known as the PxP Hub, now houses the conference recordings along with resources about patient engagement in research. These resources were recommended by the PxP Steering Committee members, speakers, and others who attended the 2023 conference. Here we lead you through how the idea for PxP was generated; how the international patient partner Steering Committee was convened and supported; how PxP was brought to life over nine months; the PxP 3-day event and feedback collected to improve future efforts; trade-offs, challenges and learnings; and resources required to support this type of event. We close with what the future holds for PxP in 2024 and beyond.It's time to elevate patients into leadership roles for conferences and events, and we encourage you to adopt the PxP ethos by using or adapting our approach and resources to support your opportunity.