Toward a generalized framework of core measurement areas in clinical trials: a position paper for OMERACT 11.

Objective: The Outcome Measures in Rheumatology (OMERACT) international consensus initiative has successfully developed core sets of outcome measures for trials of many rheumatologic conditions, but its expanding scope called for clarification and updating of its underlying conceptual framework and working process. To develop a core set of what we propose to call outcome measurement instruments, consensus must be reached both on what to measure and how to measure. This article deals with the first part: a framework necessary to ensure comprehensiveness of the domains chosen for measurement.

Updating the OMERACT filter: implications for patient-reported outcomes.

Objective: At a previous Outcome Measures in Rheumatology (OMERACT) meeting, participants reflected on the underlying methods of patient-reported outcome (PRO) instrument development. The participants requested proposals for more explicit instrument development protocols that would contribute to an enhanced version of the "Truth" statement in the OMERACT Filter, a widely used guide for outcome validation. In the present OMERACT session, we explored to what extent these new Filter 2.

Can we decide which outcomes should be measured in every clinical trial? A scoping review of the existing conceptual frameworks and processes to develop core outcome sets.

Objective: The usefulness of randomized control trials to advance clinical care depends upon the outcomes reported, but disagreement on the choice of outcome measures has resulted in inconsistency and the potential for reporting bias. One solution to this problem is the development of a core outcome set: a minimum set of outcome measures deemed critical for clinical decision making. Within rheumatology the Outcome Measures in Rheumatology (OMERACT) initiative has pioneered the development of core outcome sets since 1992.

Updating the OMERACT filter: implications for imaging and soluble biomarkers.

Objective: The Outcome Measures in Rheumatology (OMERACT) Filter provides a framework for the validation of outcome measures for use in rheumatology clinical research. However, imaging and biochemical measures may face additional validation challenges because of their technical nature. The Imaging and Soluble Biomarker Session at OMERACT 11 aimed to provide a guide for the iterative development of an imaging or biochemical measurement instrument so it can be used in therapeutic assessment.

How to choose core outcome measurement sets for clinical trials: OMERACT 11 approves filter 2.0.

Objective: The Outcome Measures in Rheumatology (OMERACT) initiative works to develop core sets of outcome measures for trials and observational studies in rheumatology. At the OMERACT 11 meeting, substantial time was devoted to discussing a conceptual framework and a proposal for a more explicit working process to develop what we now propose to term core outcome measurement sets, collectively termed "OMERACT Filter 2.0.

Developing core outcome measurement sets for clinical trials: OMERACT filter 2.0.

Background: Lack of standardization of outcome measures limits the usefulness of clinical trial evidence to inform health care decisions. This can be addressed by agreeing on a minimum core set of outcome measures per health condition, containing measures relevant to patients and decision makers. Since 1992, the Outcome Measures in Rheumatology (OMERACT) consensus initiative has successfully developed core sets for many rheumatologic conditions, actively involving patients since 2002.

The patient perspective on remission in rheumatoid arthritis: 'You've got limits, but you're back to being you again'.

Objectives: The aim of rheumatoid arthritis (RA) treatment is remission. As treatment should be targeted at outcomes relevant to patients, it is important to understand how patients perceive remission, and to assess whether the current definition of remission adequately reflects these perceptions. The objective of this study is to explore the patient perspective on remission in RA.

Confounding by indication probably distorts the relationship between steroid use and cardiovascular disease in rheumatoid arthritis: results from a prospective cohort study.

Objective: To evaluate the risk of cardiovascular disease in patients with rheumatoid arthritis exposed to glucocorticoids.

Methods: Retrospective analysis of exposure to glucocorticoids in a prospective cohort of 353 patients with rheumatoid arthritis followed from June 2001 up to November 2011 for incident cardiovascular disease in a hospital-based outpatient cohort in the Netherlands. Hazard ratios with 95%-confidence intervals were calculated for the association between different types of exposure to glucocorticoids and incident cardiovascular disease.

Outcome measures used in clinical trials for Behçet syndrome: a systematic review.

Behçet syndrome (BS) is a multisystem vasculitis that is most active during young adulthood, causing serious disability and significant impairment in quality of life. Differences in the disease course, severity, and organ involvement between patients, depending on the age at presentation and sex, makes it impossible to determine a single management strategy. The diversity and variability in the outcome measures used in clinical trials in BS makes it difficult to compare the results or inform physicians about the best management strategy for individual patients.

Measurement of stiffness in patients with rheumatoid arthritis in low disease activity or remission: a systematic review.

Background: Recent qualitative research has shown that stiffness is an important symptom for patients to identify remission. However, it is unclear how to measure stiffness in low disease activity. This systematic review aims to summarise the existing literature on validity of patient reported outcomes to measure stiffness in RA low disease activity states, to aid the choice for a measurement instrument.

Current status of outcome measure development in vasculitis.

The conduct of randomized controlled trials for vasculitis, especially for the antineutrophil cytoplasmic antibody-associated vasculitides [AAV, granulomatosis with polyangiitis (Wegener's) and microscopic polyangiitis], has been greatly advanced by the development, use, and acceptance of validated outcome measures. Trials have subsequently provided the opportunity to validate and refine reliable, valid outcome measures for these multisystemic and relapsing rare diseases. The Outcome Measures in Rheumatology (OMERACT) Vasculitis Working Group was formed in 2004 to foster development of validated and widely accepted outcomes in vasculitis using data-driven analyses, a dedication to building consensus, and adherence to, and guidance by, the principles of the OMERACT approach.

Dutch-Flemish translation of 17 item banks from the patient-reported outcomes measurement information system (PROMIS).

Background: The Patient-Reported Outcomes Measurement Information System (PROMIS(®)) is a new, state-of-the-art assessment system for measuring patient-reported health and well-being of adults and children that has the potential to be more valid, reliable and responsive than existing PROMs. The PROMIS items can be administered in short forms or, more efficiently, through computerized adaptive testing. This paper describes the translation of 563 items from 17 PROMIS item banks (domains) for adults from the English source into Dutch-Flemish.

Gastrointestinal events in at-risk patients starting non-steroidal anti-inflammatory drugs (NSAIDs) for rheumatic diseases: the EVIDENCE study of European routine practice.

Objectives: Data concerning rates of gastrointestinal (GI) events in non-steroidal anti-inflammatory drug (NSAID) users derive mainly from clinical trials. The EVIDENCE study quantified the incidence of symptomatic uncomplicated and/or complicated GI events in at-risk European patients treated with NSAIDs in real-life practice.

Methods: This non-interventional study assessed 4144 adults with at least one GI risk factor who recently initiated NSAID therapy for osteoarthritis (85%), rheumatoid arthritis (11%), ankylosing spondylitis (3%) or a combination (1%).

OMERACT endorsement of measures of outcome for studies of acute gout.

Objective: To determine the extent to which participants at the Outcome Measures in Rheumatology (OMERACT) 11 meeting agree that instruments used in clinical trials to measure OMERACT core outcome domains in acute gout fulfill OMERACT filter requirements of truth, discrimination, and feasibility; and where future research efforts need to be directed.

Methods: Results of a systematic literature review and analysis of individual-level data from recent clinical studies of acute gout were presented to OMERACT participants. The information was discussed in breakout groups, and opinion was defined by subsequent voting in a plenary session.

EULAR recommendations for the management of rheumatoid arthritis with synthetic and biological disease-modifying antirheumatic drugs: 2013 update.

In this article, the 2010 European League against Rheumatism (EULAR) recommendations for the management of rheumatoid arthritis (RA) with synthetic and biological disease-modifying antirheumatic drugs (sDMARDs and bDMARDs, respectively) have been updated. The 2013 update has been developed by an international task force, which based its decisions mostly on evidence from three systematic literature reviews (one each on sDMARDs, including glucocorticoids, bDMARDs and safety aspects of DMARD therapy); treatment strategies were also covered by the searches. The evidence presented was discussed and summarised by the experts in the course of a consensus finding and voting process.

Outcome measures in rheumatoid arthritis randomised trials over the last 50 years.

Background: The development and application of standardised sets of outcomes to be measured and reported in clinical trials have the potential to increase the efficiency and value of research. One of the most notable of the current outcome sets began nearly 20 years ago: the World Health Organization and International League of Associations for Rheumatology core set of outcomes for rheumatoid arthritis clinical trials, originating from the OMERACT (Outcome Measures in Rheumatology) Initiative. This study assesses the use of this core outcome set by randomised trials in rheumatology.

Health economics in the field of osteoarthritis: an expert's consensus paper from the European Society for Clinical and Economic Aspects of Osteoporosis and Osteoarthritis (ESCEO).

Objectives: There is an important need to evaluate therapeutic approaches for osteoarthritis (OA) in terms of cost-effectiveness as well as efficacy.

Methods: The ESCEO expert working group met to discuss the epidemiological and economic evidence that justifies the increasing concern of the impact of this disease and reviewed the current state-of-the-art in health economic studies in this field.

Results: OA is a debilitating disease; it is increasing in frequency and is associated with a substantial and growing burden on society, in terms of both burden of illness and cost of illness.

EULAR evidence-based and consensus-based recommendations on the management of medium to high-dose glucocorticoid therapy in rheumatic diseases.

To develop recommendations for the management of medium to high-dose (ie, >7.5 mg but ≤100 mg prednisone equivalent daily) systemic glucocorticoid (GC) therapy in rheumatic diseases. A multidisciplinary EULAR task force was formed, including rheumatic patients.

Three trajectories of activity limitations in early symptomatic knee osteoarthritis: a 5-year follow-up study.

Objectives: Knee osteoarthritis (OA) is a leading cause of activity limitations. The knee OA population is likely to consist of subgroups. The aim of the present study was to identify homogeneous subgroups with distinct trajectories of activity limitations in patients with early symptomatic knee OA and to describe characteristics of these subgroups.

A non-inferiority trial of an attenuated combination strategy ('COBRA-light') compared to the original COBRA strategy: clinical results after 26 weeks.

Background: Early, intensive treatment of rheumatoid arthritis (RA) with the combination of (initially high dose) prednisolone, methotrexate and sulfasalazine (COBRA therapy) considerably lowers disease activity and suppresses radiological progression, but is infrequently prescribed in daily practice. Attenuating the COBRA regimen might lessen concerns about side effects, but the efficacy of such strategies is unknown.

Objective: To compare the 'COBRA-light' strategy with only two drugs, comprising a lower dose of prednisolone (starting at 30 mg/day, tapered to 7.

A simple model that suggests possible cost savings when modified-release prednisone 5 mg/day is added to current treatment in patients with active rheumatoid arthritis.

Objective: The effects of a 12-week treatment with modified-release prednisone (MR-pred) on the costs of drug treatment of RA were modelled.

Methods: With the results of a recent randomized trial as source data, we expressed the effect of treatment (MR-pred vs placebo) on the decrease in the proportion of RA patients meeting disease activity thresholds for reimbursement of biologic treatment.

Results: The results showed 11-13% more patients on MR-pred than on placebo dropped below reimbursement thresholds for The Netherlands, Belgium and the UK.

A delphi exercise to identify characteristic features of gout - opinions from patients and physicians, the first stage in developing new classification criteria.

Objective: To identify a comprehensive list of features that might discriminate between gout and other rheumatic musculoskeletal conditions, to be used subsequently for a case-control study to develop and test new classification criteria for gout.

Methods: Two Delphi exercises were conducted using Web-based questionnaires: one with physicians from several countries who had an interest in gout and one with patients from New Zealand who had gout. Physicians rated a list of potentially discriminating features that were identified by literature review and expert opinion, and patients rated a list of features that they generated themselves.