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To standardize outcome reporting in clinical trials of patients with nonspecific low back pain, an international multidisciplinary panel recommended physical functioning, pain intensity, and health-related quality of life (HRQoL) as core outcome domains. Given the lack of a consensus on measurement instruments for these 3 domains in patients with low back pain, this study aimed to generate such consensus. The measurement properties of 17 patient-reported outcome measures for physical functioning, 3 for pain intensity, and 5 for HRQoL were appraised in 3 systematic reviews following the COSMIN methodology. Researchers, clinicians, and patients (n = 207) were invited in a 2-round Delphi survey to generate consensus (≥67% agreement among participants) on which instruments to endorse. Response rates were 44% and 41%, respectively. In round 1, consensus was achieved on the Oswestry Disability Index version 2.1a for physical functioning (78% agreement) and the Numeric Rating Scale (NRS) for pain intensity (75% agreement). No consensus was achieved on any HRQoL instrument, although the Short Form 12 (SF12) approached the consensus threshold (64% agreement). In round 2, a consensus was reached on an NRS version with a 1-week recall period (96% agreement). Various participants requested 1 free-to-use instrument per domain. Considering all issues together, recommendations on core instruments were formulated: Oswestry Disability Index version 2.1a or 24-item Roland-Morris Disability Questionnaire for physical functioning, NRS for pain intensity, and SF12 or 10-item PROMIS Global Health form for HRQoL. Further studies need to fill the evidence gaps on the measurement properties of these and other instruments. [ABSTRACT FROM AUTHOR]

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. Methods An observational review was carried out of 350 randomised trials for the treatment of rheumatoid arthritis identified through the The Cochrane Library (up to and including September 2012 issue). Reports of these trials were evaluated to determine whether or not there were trends in the proportion of trials reporting on the full set of core outcomes over time. Researchers who conducted trials after the publication of the core set were contacted to assess their awareness of it and to collect reasons for non-inclusion of the full core set of outcomes in the study. Results Since the introduction of the core set of outcomes for rheumatoid arthritis, the consistency of measurement of the core set of outcomes has improved, although variation in the choice of measurement instrument remains. The majority of trialists who responded said that they would consider using the core outcome set in the design of a new trial. Conclusions This observational review suggests that a higher percentage of trialists conducting trials in rheumatoid arthritis are now measuring the rheumatoid arthritis core outcome set. Core outcome sets have the potential to improve the evidence base for health care, but consideration must be given to the methods for disseminating their availability amongst the relevant communities. [ABSTRACT FROM AUTHOR]

Abstract: Objective: To develop a simple system to assess benefit and harm of treatment on a single scale. Harm and benefit signals from trials need to be placed in the proper perspective to decide on the value of a treatment. Several systems have been developed for assessment, but few attempt to incorporate both benefit and risk in the same metric while retaining enough simplicity to aid patients and clinicians in their decision making. Study Design and Setting: We designed a very simple 3×3 table (Outcome Measures in Rheumatology [OMERACT] 3×3) that comprises three ranks for both beneficial and harm outcomes: for benefit, these are “none,” “substantial,” and “(near) remission”; for harm, these are “none,” “severe,” and “(near) death.” Patients are ranked both for benefit and harm and subsequently counted in a 3×3 table. Results: The system was feasible when applied to one trial dataset (patient-level information) and a meta-analysis. To become applicable as a tool, several issues need to be resolved in further development, especially the definitions and cutoffs for the ranks. Conclusion: A simple 3×3 table to rank both benefit and harm outcomes is feasible. For rheumatology this will be further developed in the context of the OMERACT initiative. [Copyright &y& Elsevier]

Objective To determine the effect of a multidisciplinary lifestyle program in patients with RA with low–moderate disease activity. Methods In the 'Plants for Joints' (PFJ) parallel-arm, assessor-blind randomized controlled trial, patients with RA and 28-joint DAS (DAS28) ≥2.6 and ≤5.1 were randomized to the PFJ or control group. The PFJ group followed a 16-week lifestyle program based on a whole-food plant-based diet, physical activity and stress management. The control group received usual care. Medication was kept stable 3 months before and during the trial whenever possible. We hypothesized that PFJ would lower disease activity (DAS28). Secondary outcomes included anthropometric, metabolic and patient-reported measures. An intention-to-treat analysis with a linear mixed model adjusted for baseline values was used to analyse between-group differences. Results Of the 83 people randomized, 77 completed the study. Participants were 92% female with mean (s. d.) age of 55 (12) years, BMI of 26 (4) kg/m2 and mean DAS28 of 3.8 (0.7). After 16 weeks the PFJ group had a mean 0.9-point greater improvement of DAS28 vs the control group (95% CI 0.4, 1.3; P  < 0.0001). The PFJ intervention led to greater decreases in body weight (difference –3.9 kg), fat mass (–2.8 kg), waist circumference (–3 cm), HbA1c (–1.3 mmol/mol) and low-density lipoprotein (–0.32 mmol/l), whereas patient-reported outcome measures, blood pressure, glucose and other lipids did not change. Conclusion The 16-week PFJ multidisciplinary lifestyle program substantially decreased disease activity and improved metabolic status in people with RA with low–moderate disease activity. Trial Registration International Clinical Trials Registry Platform; https://www.who.int/clinical-trials-registry-platform ; NL7800. [ABSTRACT FROM AUTHOR]

Summary: What is known: Currently, medication bottles with an electronic cap are frequently used to measure medication adherence. This system is termed medication event monitoring system (MEMS). To our knowledge, the optimal method to summarize data from MEMS has not yet been determined. Objective: Look for best practices on how to quantify adherence data from MEMS. Methods: Review of PubMed, Embase and Cochrane databases for the articles on medication adherence with MEMS. Results: Of 1493 identified articles, 207 were included in this review. The MEMS cap was used for a median of 3 months (IQR: 4; range: 1 week to 24 months) in various health conditions. Many different outcome measures were used. Most studies computed an adherence score, expressed as the percentage of days on which the correct dose of medication was taken. The threshold to mark people as adherent was most frequently, arbitrarily, set at 80% (range: 67%‐95%). We found no data to support a specific threshold. Discussion: Although the commonly used definition of adherence has face validity, we found no validation studies, and not all studies used the same cut‐off for adherence. Ideally, a cut‐off should be defined and validated in the context of the specific drug and its pharmacokinetic and dynamic characteristics, and perhaps other contextual factors, rather than generically. In addition, there was large heterogeneity in the definition of what "correct intake" of medication is. What is new and conclusion: Outcome measures for MEMS data lacked standardization, and no demonstrable effort to validate any definition against a relevant clinical outcome is available. Consensus on the definition of adherence is urgently needed. Outcome measures for Medication Event Monitoring System (MEMS) data lacked standardization and no demonstrable effort to validate any definition against a relevant clinical outcome is available. A consensus effort on the definition of adherence is urgently needed. [ABSTRACT FROM AUTHOR]

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. Methods An extensive pubmed-search was undertaken, identifying measurement instruments for patient perceived stiffness used in low disease activity. Eligible studies reported on 1) stiffness as an outcome in relation to other core set measures, 2) development of a patient reported tool to measure stiffness, or 3) comparison of two different tools to measure aspects of stiffness, all in low disease activity. Results Of 788 titles, only two studies report on validity of stiffness measures within low disease activity. Morning stiffness (MS) is reported in 44 to 80% of patients in low disease activity. A difference of 40 to 60 minutes in duration until maximum improvement is observed between active and inactive patients. Severity of MS might discriminate better between high and low disease activity compared to measurement of duration of MS. Conclusions There is insufficient data on measurement of stiffness in the spectrum of low disease activity or remission. [ABSTRACT FROM AUTHOR]

The use of nonstandardized and inadequately validated outcome measures in atopic eczema trials is a major obstacle to practising evidence-based dermatology. The Harmonising Outcome Measures for Eczema ( HOME) initiative is an international multiprofessional group dedicated to atopic eczema outcomes research. In June 2011, the HOME initiative conducted a consensus study involving 43 individuals from 10 countries, representing different stakeholders (patients, clinicians, methodologists, pharmaceutical industry) to determine core outcome domains for atopic eczema trials, to define quality criteria for atopic eczema outcome measures and to prioritize topics for atopic eczema outcomes research. Delegates were given evidence-based information, followed by structured group discussion and anonymous consensus voting. Consensus was achieved to include clinical signs, symptoms, long-term control of flares and quality of life into the core set of outcome domains for atopic eczema trials. The HOME initiative strongly recommends including and reporting these core outcome domains as primary or secondary endpoints in all future atopic eczema trials. Measures of these core outcome domains need to be valid, sensitive to change and feasible. Prioritized topics of the HOME initiative are the identification/development of the most appropriate instruments for the four core outcome domains. HOME is open to anyone with an interest in atopic eczema outcomes research. [ABSTRACT FROM AUTHOR]

Background: This masterclass introduces the topic of core outcome sets, describing rationale and methods for developing them, and providing some examples that are relevant for clinical research and practice. Method: A core outcome set is a minimum consensus-based set of outcomes that should be measured and reported in all clinical trials for a specific health condition and/or intervention. Issues surrounding outcome assessment, such as selective reporting and inconsistency across studies, can be addressed by the development of a core set. As suggested by key initiatives in this field (i.e. OMERACT and COMET), the development requires achieving consensus on: (1) core outcome domains and (2) core outcome measurement instruments. Different methods can be used to reach consensus, including: literature systematic reviews to inform the process, qualitative research with clinicians and patients, group discussions (e.g. nominal group technique), and structured surveys (e.g. Delphi technique). Various stakeholders should be involved in the process, with particular attention to patients. Results and conclusions: Several COSs have been developed for musculoskeletal conditions including a longstanding one for low back pain, IMMPACT recommendations on outcomes for chronic pain, and OMERACT COSs for hip, knee and hand osteoarthritis. There is a lack of COSs for neurological, geriatric, cardio-respiratory and pediatric conditions, therefore, future research could determine the value of developing COSs for these conditions. [ABSTRACT FROM AUTHOR]

Background: In cooperation with the Core Outcome Measures in Effectiveness Trials (COMET) initiative, the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) initiative aimed to develop a guideline on how to select outcome measurement instruments for outcomes (i.e., constructs or domains) included in a "Core Outcome Set" (COS). A COS is an agreed minimum set of outcomes that should be measured and reported in all clinical trials of a specific disease or trial population.Methods: Informed by a literature review to identify potentially relevant tasks on outcome measurement instrument selection, a Delphi study was performed among a panel of international experts, representing diverse stakeholders. In three consecutive rounds, panelists were asked to rate the importance of different tasks in the selection of outcome measurement instruments, to justify their choices, and to add other relevant tasks. Consensus was defined as being achieved when 70 % or more of the panelists agreed and when fewer than 15 % of the panelists disagreed.Results: Of the 481 invited experts, 120 agreed to participate of whom 95 (79 %) completed the first Delphi questionnaire. We reached consensus on four main steps in the selection of outcome measurement instruments for COS: Step 1, conceptual considerations; Step 2, finding existing outcome measurement instruments, by means of a systematic review and/or a literature search; Step 3, quality assessment of outcome measurement instruments, by means of the evaluation of the measurement properties and feasibility aspects of outcome measurement instruments; and Step 4, generic recommendations on the selection of outcome measurement instruments for outcomes included in a COS (consensus ranged from 70 to 99 %).Conclusions: This study resulted in a consensus-based guideline on the methods for selecting outcome measurement instruments for outcomes included in a COS. This guideline can be used by COS developers in defining how to measure core outcomes. [ABSTRACT FROM AUTHOR]

Objective To compare rates of sustained low and minimal disease activity and remission according to the American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) criteria during 3-year followup in rheumatoid arthritis (RA) patients treated with etanercept and adalimumab in routine care. Methods Four hundred seven RA patients previously unexposed to tumor necrosis factor antagonists were treated with etanercept (n = 203) or adalimumab (n = 204) and assessed at 3- and later 6-month intervals. Treatment allocation was at the discretion of the treating rheumatologist. Clinical parameters were measured at each time point, as were anti-adalimumab antibodies in adalimumab-treated patients. Achievement of clinical outcome was defined as the occurrence of sustained (at least 12 consecutive months) low disease activity (28-joint Disease Activity Score [DAS28] <3.2), minimal disease activity (DAS28 <2.6), or ACR/EULAR remission based on the Simplified Disease Activity Index (SDAI). Non-overlapping response rates were calculated. Results Among the adalimumab group, 13% reached sustained low disease activity but not sustained minimal disease activity, 15% reached sustained minimal disease activity but not sustained remission according to the SDAI, and 16% reached sustained ACR/EULAR remission. In the etanercept group the corresponding rates were 16%, 11%, and 12%, respectively ( P = 0.42, overall test for linear trend). Adalimumab-treated patients without anti-adalimumab antibodies (n = 150 [74%]) had the best outcomes, and adalimumab-treated patients with anti-adalimumab antibodies the worst, with outcomes in etanercept-treated patients in between ( P < 0.0001). Differences were most apparent in the sustained SDAI remission and sustained minimal disease activity categories. For example, 40% of anti-adalimumab antibody-negative patients, 23% of etanercept-treated patients, and 4% of anti-adalimumab antibody-positive patients achieved at least sustained minimal disease activity. Conclusion Overall, etanercept and adalimumab treatment appear similar in inducing a good long-term clinical outcome. However, in the case of adalimumab this is strongly dependent on the presence or absence of anti-adalimumab antibodies. [ABSTRACT FROM AUTHOR]

Content validity of patient-reported outcome measures (PROs) has been a focus of debate since the 2006 publication of the U.S. FDA Draft Guidance for Industry in Patient Reported Outcome Measurement. Under the auspices of the Patient Reported Outcomes Measurement Information System (PROMIS) initiative, a working meeting on content validity was convened with leading PRO measurement experts. Platform presentations and participant discussion highlighted key issues in the content validity debate, including inconsistency in the definition and evaluation of content validity, the need for empirical research to support methodological approaches to the evaluation of content validity, and concerns that continual re-evaluation of content validity slows the pace of science and leads to the proliferation of study-specific PROs. We advocate an approach to the evaluation of content validity, which includes meticulously documented qualitative and advanced quantitative methods. To advance the science of content validity in PROs, we recommend (1) development of a consensus definition of content validity; (2) development of content validity guidelines that delineate the role of qualitative and quantitative methods and the integration of multiple perspectives; (3) empirical evaluation of generalizability of content validity across applications; and (4) use of generic measures as the foundation for PROs assessment. [ABSTRACT FROM AUTHOR]

The author comments on getting patients involved in their rheumatology experience. Topics covered include the important role of clinicians in educating patients regarding how to effectively manage their health conditions. Also mentioned is the need for clinicians to help their patients understand the effects of inflammatory arthritis.