Defining and evaluating novel procedures for involving patients in Core Outcome Set research: creating a meaningful long list of candidate outcome domains

Background Tinnitus is a complex audiological condition affecting many different domains of everyday life. Clinical trials of tinnitus interventions measure and report those outcome domains inconsistently and this hinders direct comparison between study findings. To address this problem, an ongoing project is developing a Core Outcome Set; an agreed list of outcome domains to be measured and reported in all future trials. Part of this project uses a consensus methodology (‘Delphi’ survey), whereby all relevant stakeholders identify important and critical outcome domains from a long list of candidates. This article addresses a gap in the patient involvement literature by describing and reflecting on our involvement of patients to create a meaningful long list of candidate outcome domains. Methods Two Public Research Partners with lived experience of tinnitus reviewed an initial list of 124 outcome domains over two face-to-face workshops. With the Study Management Team, they interpreted each candidate outcome domain and generated a plain language description. Following this, the domain names and descriptions underwent an additional lay review by 14 patients and 5 clinical experts, via an online survey platform. Results Insights gained from the workshops and survey feedback prompted substantial, unforeseen modifications to the long list. These included the reduction of the number of outcome domains (from 124 to 66) via the exclusion of broad concepts and consolidation of equivalent domains or domains outside the scope of the study. Reviewers also applied their lived experience of tinnitus to bring clarity and relevance to domain names and plain language descriptions. Four impacts on the Delphi survey were observed: recruitment exceeded the target by 171%, there were equivalent numbers of patient and professional participants (n=358 and n=312, respectively), feedback was mostly positive, and retention was high (87%). Conclusions Patient involvement was an integral and transformative step of the study design process. Patient involvement was impactful because the online Delphi survey was successful in recruiting and retaining participants, and there were many comments about a positive participatory experience. Seven general methodological features are highlighted which fit with general principles of good patient involvement. These can benefit other Core Outcome Set developers

Achieving minimal disease activity in psoriatic arthritis predicts meaningful improvements in patients’ health-related quality of life and productivity

Background Although psoriatic arthritis is complex and involves multiple domains, recent advances in treatments have made remission or near-remission of most symptoms a potentially achievable goal for many patients. We sought to evaluate whether achieving minimal disease activity (MDA) criteria represented meaningful improvement from the patient perspective. Methods Data were combined from two randomized, multinational, 24 week clinical studies of ixekizumab, a high-affinity monoclonal antibody selectively targeting interleukin-17A, in biological drug-naïve or experienced adults. MDA required 5 of 7 of: tender joint count ≤1; swollen joint count ≤1; Psoriasis Area and Severity Index total score ≤ 1 or body surface area ≤ 3%; patient’s assessment of pain visual analogue scale (VAS) ≤15; patient’s global assessment of disease activity VAS ≤20; Health Assessment Questionnaire Disability Index ≤0.5; and tender entheseal points ≤ 1. MDA responders and non-responders were compared for mean change from baseline on the 36-Item Short Form Health Survey (SF-36), European Quality of Life 5 Dimension 5 Level Health Questionnaire (EQ-5D-5 L); EQ-5D-5 L VAS; and Work Productivity and Activity Impairment–Specific Health Problem (WPAI-SHP) questionnaire. Results MDA responders had significantly greater improvements versus non-responders in each SF-36 domain and in the SF-36 physical summary score; improvements were also greater in the EQ-5D-5 L and EQ-5D-5 L VAS, and in 3 of the 4 WPAI-SHP domains. MDA responders were more likely to achieve minimal clinically important differences than non-responders. Conclusion These findings support MDA response as being strongly associated with achieving improved disease status based on measures of patient reported health-related quality of life and productivity. Trial registration SPIRIT-P1, NCT01695239, First Posted: September 27, 2012; and SPIRIT-P2, NCT02349295, First Posted: January 28, 2015

A systematic review of measurement properties of patient reported outcome measures in psoriatic arthritis: A GRAPPA-OMERACT initiative

Background: An updated psoriatic arthritis (PsA) core outcome set (COS) for randomized controlled trials (RCTs) was endorsed at the Outcome Measures in Rheumatology (OMERACT) meeting in 2016. Objectives: To synthesize the evidence on measurement properties of patient reported outcome measures (PROMs) for PsA and thereby contribute to development of a PsA core outcome measurement set (COMS) as described by the OMERACT Filter 2.0. Methods: A systematic literature search was performed in EMBASE, MEDLINE and PsycINFO on Jan 1, 2017 to identify full-text articles with an aim of assessing the measurement properties of PROMs in PsA. Two independent reviewers rated the quality of studies using the COnsensus based standards for the Selection of health Measurement INstruments (COSMIN) checklist, and performed a qualitative evidence synthesis. Results: Fifty-five studies were included in the systematic review. Forty-four instruments and a total of 89 scales were analyzed. PROMs measuring COS domains with at least fair quality evidence for good validity and reliability (and no evidence for poor properties) included the Stockerau Activity Score for PsA (German), Psoriasis Symptom Inventory, visual analogue scale for Patient Global, 36 Item Short Form Health Survey Physical Function subscale, Health Assessment Questionnaire Disability Index, Bath Ankylosing Spondylitis Functional Index, PsA Impact of Disease questionnaire, PsA Quality of Life questionnaire, VITACORA-19, Functional Assessment of Chronic Illness Therapy Fatigue scale and Social Role Participation Questionnaire. Conclusions: At least one PROM with some evidence for aspects of validity and reliability was available for six of the eight mandatory domains of the PsA COS

Measurement properties of radiographic outcome measures in Psoriatic Arthritis: A systematic review from the GRAPPA-OMERACT initiative

Background: Structural damage is as an important outcome in the Psoriatic Arthritis (PsA) Core Domain Set and its assessment is recommended at least once in the development of a new drug.Objectives: To conduct a systematic review (SR) to identify studies addressing the measurement properties of radiographic outcome instruments for structural damage in PsA and appraise the evidence through the Outcome Measures in Rheumatology (OMERACT) Filter 2.1 Framework Instrument Selection Algorithm (OFISA).Methods: A SR was conducted using search strategies in EMBASE and MEDLINE to identify full-text English studies which aimed to develop or assess the measurement properties of radiographic outcome instruments in PsA. Determination of eligibility and data extraction was performed independently by two reviewers with input from a third to achieve consensus. Two reviewers assessed the methodology and results of eligible studies and synthesized the evidence using OMERACT methodology. Results: Twelve articles evaluating radiographic instruments were included. The articles assessed nine peripheral (hands, wrists and/or feet) and six axial (spinal and/or sacroiliac joints) radiographic instruments. The peripheral radiographic instruments with some evidence for reliability, cross-sectional construct validity and longitudinal construct validity were the Ratingen and modified Sharp van der Heijde scores. No instruments had evidence for clinical trial discrimination or thresholds of meaning. There was limited evidence for the measurement properties of all identified axial instruments.Conclusion: There are significant knowledge gaps in the responsiveness of peripheral radiographic instruments. Axial radiographic instruments require further validation, and the need to generate novel instruments and utilise other imaging modalities should be considered.(c) 2021 Elsevier Inc. All rights reserved

Social Media Use for Health-Related Purposes By People with Rheumatic and Musculoskeletal Diseases - Results of a Global Survey

Background/Purpose: In Malaysia around one in ten older people are diagnosed with osteoarthritis (OA), with the knee being one of the most commonly affected areas. This can lead to functional limitations, impaired activities of daily living and reduced quality-of-life. Our systematic review of the literature concludes that a programme integrating exercise, education and active coping strategies, known as Enabling Self-management and Coping with Arthritic Pain using Exercise (ESCAPE-pain) provides the best evidence for implementation. Thus, this study aims to evaluate the feasibility of the ESCAPE-pain programme among patients with knee OA in the Malaysian healthcare context. Methods: A pragmatic, feasibility randomised controlled trial (RCT) was conducted recruiting patients with knee osteoarthritis from two hospitals in Malaysia. Participants were randomised to receive ESCAPE-pain intervention plus usual care (n=36) (intervention group) or usual care only (n=36) (control group). Outcomes were measured for physical function (TUG), knee injury and osteoarthritis outcome scores (KOOS), mental wellbeing (Short-WEMWBS), exercise health beliefs and self-efficacy (ExBeliefs) and fear of falling (Short-FES-I) at baseline, six-week and after 12-week of intervention. Results: There were no significant differences in baseline characteristics between the groups (p>0.05). Recruitment rate showed 90.5% (72/105) and retention rate at 12-week was 87.5% (63/72). Attendance to intervention programme at ≥10 of 12 sessions was high (82.4%). Using modified intention-to-treat analysis, repeated measures ANOVA showed no significant changes (p>0.05) for TUG or KOOS between intervention and control groups. However, better outcomes (p<0.05) were reported after 12 weeks for health beliefs, mental wellbeing, and fear of falling among patients in intervention group. Satisfaction survey among participants revealed that the ESCAPE-pain programme is easy to follow, straightforward and tolerable for future implementation. Conclusion: The findings of this study indicate that the ESCAPE-pain programme is feasible for patients with knee OA in Malaysia. As a feasibility study, this is not powered to detect significant differences on primary KOOS outcomes, nonetheless participants reported positive views towards exercise with significant improvements in belief in performing activities, mental wellbeing and reduced fear of falling

Measurement properties of radiographic outcome measures in Psoriatic Arthritis: A systematic review from the GRAPPA-OMERACT initiative

Background: Structural damage is as an important outcome in the Psoriatic Arthritis (PsA) Core Domain Set and its assessment is recommended at least once in the development of a new drug.Objectives: To conduct a systematic review (SR) to identify studies addressing the measurement properties of radiographic outcome instruments for structural damage in PsA and appraise the evidence through the Outcome Measures in Rheumatology (OMERACT) Filter 2.1 Framework Instrument Selection Algorithm (OFISA).Methods: A SR was conducted using search strategies in EMBASE and MEDLINE to identify full-text English studies which aimed to develop or assess the measurement properties of radiographic outcome instruments in PsA. Determination of eligibility and data extraction was performed independently by two reviewers with input from a third to achieve consensus. Two reviewers assessed the methodology and results of eligible studies and synthesized the evidence using OMERACT methodology. Results: Twelve articles evaluating radiographic instruments were included. The articles assessed nine peripheral (hands, wrists and/or feet) and six axial (spinal and/or sacroiliac joints) radiographic instruments. The peripheral radiographic instruments with some evidence for reliability, cross-sectional construct validity and longitudinal construct validity were the Ratingen and modified Sharp van der Heijde scores. No instruments had evidence for clinical trial discrimination or thresholds of meaning. There was limited evidence for the measurement properties of all identified axial instruments.Conclusion: There are significant knowledge gaps in the responsiveness of peripheral radiographic instruments. Axial radiographic instruments require further validation, and the need to generate novel instruments and utilise other imaging modalities should be considered.(c) 2021 Elsevier Inc. All rights reserved

Assessing Disease Activity in Psoriatic Arthritis: A Literature Review

Psoriatic arthritis (PsA) is a multifaceted disease, with a high impact on patients’ psychological and physical well-being. There is increasing recognition that assessment of both clinical aspects of disease and patient identified concerns, such as fatigue, work disability, and treatment satisfaction need to be addressed. Only then can we fully understand disease burden and make well-informed treatment decisions aimed at improving patients’ lives. In recent years, there has been much progress in the development of unidimensional and composite measures of disease activity, as well as questionnaires capturing the patient’s perspective in psoriatic disease. Despite these advances, there remains disagreement amongst clinicians as to which instruments should be used. As a consequence, they are yet to receive widespread implementation in routine clinical practice. This review aims to summarize currently available clinical and patient-derived assessment tools, which will provide clinicians with a practical and informative resource

Stiffness Is the Cardinal Symptom of Inflammatory Musculoskeletal Diseases, Yet Still Variably Measured: Report from the OMERACT 2016 Stiffness Special Interest Group

Objective: The objectives of the Outcome Measures in Rheumatology (OMERACT) Stiffness special interest group (SIG) are to characterize stiffness as an outcome in rheumatic disease and to identify and validate a stiffness patient-reported outcome (PRO) in rheumatology. Methods: At OMERACT 2016, international groups presented and discussed results of several concurrent research projects on stiffness: a literature review of rheumatoid arthritis (RA) stiffness PRO measures, a qualitative investigation into the RA and polymyalgia rheumatica patient perspective of stiffness, data-driven stiffness conceptual model development, development and testing of an RA stiffness PRO measure, and a quantitative work testing stiffness items in patients with RA and psoriatic arthritis. Results: The literature review identified 52 individual stiffness PRO measures assessing morning or early morning stiffness severity/intensity or duration. Items were heterogeneous, had little or inconsistent psychometric property evidence, and did not appear to have been developed according to the PRO development guidelines. A poor match between current stiffness PRO and the conceptual model identifying the RA patient experience of stiffness was identified, highlighting a major flaw in PRO selection according to the OMERACT filter 2.0. Conclusion: Discussions within the Stiffness SIG highlighted the importance of further research on stiffness and defined a research agenda

Anti-C1q antibodies in systemic lupus erythematosus

Anti-C1q has been associated with systemic lupus erythematosus (SLE) and lupus nephritis in previous studies. We studied anti-C1q specificity for SLE (vs. rheumatic disease controls) and the association with SLE manifestations in an international multi-center study